Guidelines

VIENNA – The use of immunosuppressive and antifibrotic drugs to treat skin and lung fibrosis leads updated recommendations from the European Alliance of Associations for Rheumatology (EULAR) for the treatment of systemic sclerosis.

“The most impactful new recommendation relates to the evidence for immunosuppressive agents and antifibrotics for the treatment of skin fibrosis and lung fibrosis,” said Francesco Del Galdo, MD, PhD, professor of experimental medicine, consultant rheumatologist, and scleroderma and connective tissue diseases specialist at Leeds Teaching Hospitals NHS Trust, Leeds, England. Dr. Del Galdo presented the update at the annual European Congress of Rheumatology.

“But there are also new recommendations, including a redefined target population for hematopoietic stem cell transplantation following cyclophosphamide, the upfront combination treatment at the time of diagnosis of pulmonary arterial hypertension [PAH], and a negative recommendation for the use of anticoagulants for pulmonary arterial hypertension,” noted Dr. Del Galdo, highlighting key updates in the 2024 recommendations.

Robert B.M. Landewé, MD, PhD, professor and rheumatologist at Amsterdam University Medical Center, Amsterdam, the Netherlands, and Zuyderland Medical Center, Heerlen, the Netherlands, co-moderated the session on EULAR recommendations. “The management of systemic sclerosis is a field in which a lot is happening,” he said. “The last update goes back to 2017, and in the meantime, many new approaches have seen the light, especially pertaining to skin fibrosis and interstitial lung disease. Six new recommendations have been coined, covering drugs like mycophenolate mofetil, nintedanib, rituximab, and tocilizumab. None of these therapies were present in the 2017 recommendations. It seems the field is now ready to further expand on targeted therapies for the management of musculoskeletal and gastrointestinal manifestations, calcinosis, and the local management of digital ulcers.”
 

‘Therapeutic Continuums’ Aid Disease Management

Dr. Del Galdo and his colleagues grouped the various interventions across what the recommendations label as evidence-backed “therapeutic continuums.” These span six of the eight different clinical manifestations of systemic sclerosis: Raynaud’s phenomenon, digital ulcers, pulmonary hypertension, musculoskeletal manifestations, skin fibrosis, interstitial lung disease (ILD), and gastrointestinal and renal crisis.

A slide showing the different strengths of evidence for various drugs across the eight manifestations illustrated the principle behind the therapeutic continuums. “These ‘therapeutic continuums’ suggest a common pathogenetic mechanism driving the various manifestations of disease,” said Dr. Del Galdo. For example, he noted, “If rituximab had a positive response in skin and in lung, it suggests that B cells play a role in the clinical manifestations of skin and lung in this disease.”

Dr. Del Galdo highlighted the new immunosuppression continuum and associated treatments for skin and lung fibrosis. “For skin involvement, the task force recommended mycophenolate, methotrexate, and rituximab, with tocilizumab having a lower level of evidence and lower recommendation strength; similarly, in interstitial lung disease, we have rituximab, mycophenolate, cyclophosphamide, and nintedanib, and these all have the highest strength of evidence. Tocilizumab is assigned one strength of evidence below the other drugs.”

He also cited the phosphodiesterase 5 inhibitor (PDE5i) drugs that are used across Raynaud’s phenomenon, digital ulcers, and pulmonary arterial hypertension, which together form a vascular therapeutic continuum.

The complexity of systemic sclerosis and multiple manifestations was a major determinant of the recommendations, Dr. Del Galdo pointed out. “The task force realized that since this is such a complex disease, we cannot recommend one treatment unconditionally. For example, with mycophenolate mofetil, what works for most patients for the skin and lung manifestations might not for someone who experiences severe diarrhea, in which mycophenolate is contraindicated. So, the highest degree of recommendation that the task force felt comfortable with was ‘should be considered.’ ”

Dr. Del Galdo stressed that the complex nature of systemic sclerosis means that “when thinking of treating one manifestation, you also always need to consider all the other clinical manifestations as experienced by the patient, and it is this multifaceted scenario that will ultimately lead to your final choice.”

Turning to new evidence around drug use, Dr. Del Galdo said that rituximab has the highest level of evidence across skin and lung manifestations, nintedanib is new in lung, and tocilizumab is new across both skin and lung.

To treat systemic sclerosis–pulmonary arterial hypertension (SSc-PAH), as long as there are no contraindications, the task force recommends using PDE5i and endothelin receptor antagonists (ERAs) at diagnosis. Data from phase 3 trials show a better outcome when the combination is established early.

The task force suggests avoiding the use of warfarin in PAH. “This is supported by a signal from two trials showing an increase in morbidity and mortality in these patients,” noted Dr. Del Galdo.

He also pointed out that selexipag and riociguat were new and important second-line additions for the treatment of PAH, and — consistent with the ERA approach — the EULAR recommendation supports frequent follow-up to establish a treat-to-target approach to maximizing clinical outcomes in SSc-PAH and SSc-ILD. “Specifically, for the first time, we recommend monitoring the effect of any chosen intervention selected within 3-6 months of starting. The evidence suggests there is a group of patients who respond and some who respond less well and who might benefit from a second-line intervention.”

For example, results of one trial support the approach of adding an antifibrotic agent to reduce progression in people with progressive lung fibrosis. “Similarly, for pulmonary hypertension, we recommend putting patients on dual treatment, and if this fails, place them on selexipag or switch the PDE5i to riociguat,” Dr. Del Galdo said.
 

Systemic Sclerosis Research Agenda and Recommendations Align

Dr. Del Galdo highlighted the value of therapeutic continuums in advancing disease understanding. “It is starting to teach us what we know and what we don’t and where do we need to build more evidence. Effectively, they determine where the gaps in therapy lie, and this starts to guide the research agenda.

“In fact, what is really interesting about this recommendation update — certainly from the perspective of disease understanding — is that we are starting to have a bird’s-eye view of the clinical manifestations of scleroderma that have so often been dealt with separately. Now we are starting to build a cumulative evidence map of this disease.”

In 2017, the research agenda largely advocated identifying immune-targeting drugs for skin and lung fibrosis, Dr. Del Galdo pointed out. “Now, we’ve done that — we’ve identified appropriate immunosuppressive drugs — and this is testimony to the importance of these recommendations because what prioritized the research agenda 10 years ago ended up informing the clinical trials and made it into the recommendations.”

“We definitely are one step forward compared to this 2017 recommendation and closer to what we would like to do,” he asserted.
 

Remission Elusive but Getting Closer

In some respects, according to Dr. Del Galdo, research and development is making relatively slow progress, especially compared with other rheumatologic diseases such as rheumatoid arthritis. “We cannot put patients with systemic sclerosis in remission yet. But I think we are one step ahead in that we’ve now established the treat-to-target approach to maximize the efficacy with which we can stall disease progression, but we cannot yet put these patients into remission,” he said. Systemic sclerosis has multiple manifestations, and fibrotic damage cannot be reversed. “Right now, the scar will remain there forever,” he noted.

Until remission is achievable, Dr. Del Galdo advises diagnosing and treating patients earlier to prevent fibrotic manifestations.

Dr. Del Galdo explained the three leading priorities on the systemic sclerosis research agenda. “There are three because it is such a complex disease. The first is considering the patient voice — this is the most important one, and the patients say they want a more holistic approach — so trialing and treating multiple manifestations together.”

Second, Dr. Del Galdo said, he would like to see a patient-reported measure developed that can capture the entire disease.

Third, from a physician’s point of view, Dr. Del Galdo said, “We want to send the patients into remission. We need to continue to further deconvolute the clinical manifestations and find the bottleneck at the beginning of the natural history of disease.

“If we can find a drug that is effective very early on, before the patients start getting the eight different manifestations with different levels of severity, then we will be on the right road, which we hope will end in remission.”

Dr. Del Galdo has served on the speakers bureau for AstraZeneca and Janssen; consulted for AstraZeneca, Boehringer Ingelheim, Capella, Chemomab, Janssen, and Mitsubishi-Tanabe; and received grant or research support from AbbVie, AstraZeneca, Boheringer Ingelheim, Capella, Chemomab, Kymab, Janssen, and Mitsubishi-Tanabe. Dr. Landewé had no relevant disclosures.

A version of this article first appeared on Medscape.com.

VIENNA – The use of immunosuppressive and antifibrotic drugs to treat skin and lung fibrosis leads updated recommendations from the European Alliance of Associations for Rheumatology (EULAR) for the treatment of systemic sclerosis.

“The most impactful new recommendation relates to the evidence for immunosuppressive agents and antifibrotics for the treatment of skin fibrosis and lung fibrosis,” said Francesco Del Galdo, MD, PhD, professor of experimental medicine, consultant rheumatologist, and scleroderma and connective tissue diseases specialist at Leeds Teaching Hospitals NHS Trust, Leeds, England. Dr. Del Galdo presented the update at the annual European Congress of Rheumatology.

“But there are also new recommendations, including a redefined target population for hematopoietic stem cell transplantation following cyclophosphamide, the upfront combination treatment at the time of diagnosis of pulmonary arterial hypertension [PAH], and a negative recommendation for the use of anticoagulants for pulmonary arterial hypertension,” noted Dr. Del Galdo, highlighting key updates in the 2024 recommendations.

Robert B.M. Landewé, MD, PhD, professor and rheumatologist at Amsterdam University Medical Center, Amsterdam, the Netherlands, and Zuyderland Medical Center, Heerlen, the Netherlands, co-moderated the session on EULAR recommendations. “The management of systemic sclerosis is a field in which a lot is happening,” he said. “The last update goes back to 2017, and in the meantime, many new approaches have seen the light, especially pertaining to skin fibrosis and interstitial lung disease. Six new recommendations have been coined, covering drugs like mycophenolate mofetil, nintedanib, rituximab, and tocilizumab. None of these therapies were present in the 2017 recommendations. It seems the field is now ready to further expand on targeted therapies for the management of musculoskeletal and gastrointestinal manifestations, calcinosis, and the local management of digital ulcers.”
 

‘Therapeutic Continuums’ Aid Disease Management

Dr. Del Galdo and his colleagues grouped the various interventions across what the recommendations label as evidence-backed “therapeutic continuums.” These span six of the eight different clinical manifestations of systemic sclerosis: Raynaud’s phenomenon, digital ulcers, pulmonary hypertension, musculoskeletal manifestations, skin fibrosis, interstitial lung disease (ILD), and gastrointestinal and renal crisis.

A slide showing the different strengths of evidence for various drugs across the eight manifestations illustrated the principle behind the therapeutic continuums. “These ‘therapeutic continuums’ suggest a common pathogenetic mechanism driving the various manifestations of disease,” said Dr. Del Galdo. For example, he noted, “If rituximab had a positive response in skin and in lung, it suggests that B cells play a role in the clinical manifestations of skin and lung in this disease.”

Dr. Del Galdo highlighted the new immunosuppression continuum and associated treatments for skin and lung fibrosis. “For skin involvement, the task force recommended mycophenolate, methotrexate, and rituximab, with tocilizumab having a lower level of evidence and lower recommendation strength; similarly, in interstitial lung disease, we have rituximab, mycophenolate, cyclophosphamide, and nintedanib, and these all have the highest strength of evidence. Tocilizumab is assigned one strength of evidence below the other drugs.”

He also cited the phosphodiesterase 5 inhibitor (PDE5i) drugs that are used across Raynaud’s phenomenon, digital ulcers, and pulmonary arterial hypertension, which together form a vascular therapeutic continuum.

The complexity of systemic sclerosis and multiple manifestations was a major determinant of the recommendations, Dr. Del Galdo pointed out. “The task force realized that since this is such a complex disease, we cannot recommend one treatment unconditionally. For example, with mycophenolate mofetil, what works for most patients for the skin and lung manifestations might not for someone who experiences severe diarrhea, in which mycophenolate is contraindicated. So, the highest degree of recommendation that the task force felt comfortable with was ‘should be considered.’ ”

Dr. Del Galdo stressed that the complex nature of systemic sclerosis means that “when thinking of treating one manifestation, you also always need to consider all the other clinical manifestations as experienced by the patient, and it is this multifaceted scenario that will ultimately lead to your final choice.”

Turning to new evidence around drug use, Dr. Del Galdo said that rituximab has the highest level of evidence across skin and lung manifestations, nintedanib is new in lung, and tocilizumab is new across both skin and lung.

To treat systemic sclerosis–pulmonary arterial hypertension (SSc-PAH), as long as there are no contraindications, the task force recommends using PDE5i and endothelin receptor antagonists (ERAs) at diagnosis. Data from phase 3 trials show a better outcome when the combination is established early.

The task force suggests avoiding the use of warfarin in PAH. “This is supported by a signal from two trials showing an increase in morbidity and mortality in these patients,” noted Dr. Del Galdo.

He also pointed out that selexipag and riociguat were new and important second-line additions for the treatment of PAH, and — consistent with the ERA approach — the EULAR recommendation supports frequent follow-up to establish a treat-to-target approach to maximizing clinical outcomes in SSc-PAH and SSc-ILD. “Specifically, for the first time, we recommend monitoring the effect of any chosen intervention selected within 3-6 months of starting. The evidence suggests there is a group of patients who respond and some who respond less well and who might benefit from a second-line intervention.”

For example, results of one trial support the approach of adding an antifibrotic agent to reduce progression in people with progressive lung fibrosis. “Similarly, for pulmonary hypertension, we recommend putting patients on dual treatment, and if this fails, place them on selexipag or switch the PDE5i to riociguat,” Dr. Del Galdo said.
 

Systemic Sclerosis Research Agenda and Recommendations Align

Dr. Del Galdo highlighted the value of therapeutic continuums in advancing disease understanding. “It is starting to teach us what we know and what we don’t and where do we need to build more evidence. Effectively, they determine where the gaps in therapy lie, and this starts to guide the research agenda.

“In fact, what is really interesting about this recommendation update — certainly from the perspective of disease understanding — is that we are starting to have a bird’s-eye view of the clinical manifestations of scleroderma that have so often been dealt with separately. Now we are starting to build a cumulative evidence map of this disease.”

In 2017, the research agenda largely advocated identifying immune-targeting drugs for skin and lung fibrosis, Dr. Del Galdo pointed out. “Now, we’ve done that — we’ve identified appropriate immunosuppressive drugs — and this is testimony to the importance of these recommendations because what prioritized the research agenda 10 years ago ended up informing the clinical trials and made it into the recommendations.”

“We definitely are one step forward compared to this 2017 recommendation and closer to what we would like to do,” he asserted.
 

Remission Elusive but Getting Closer

In some respects, according to Dr. Del Galdo, research and development is making relatively slow progress, especially compared with other rheumatologic diseases such as rheumatoid arthritis. “We cannot put patients with systemic sclerosis in remission yet. But I think we are one step ahead in that we’ve now established the treat-to-target approach to maximize the efficacy with which we can stall disease progression, but we cannot yet put these patients into remission,” he said. Systemic sclerosis has multiple manifestations, and fibrotic damage cannot be reversed. “Right now, the scar will remain there forever,” he noted.

Until remission is achievable, Dr. Del Galdo advises diagnosing and treating patients earlier to prevent fibrotic manifestations.

Dr. Del Galdo explained the three leading priorities on the systemic sclerosis research agenda. “There are three because it is such a complex disease. The first is considering the patient voice — this is the most important one, and the patients say they want a more holistic approach — so trialing and treating multiple manifestations together.”

Second, Dr. Del Galdo said, he would like to see a patient-reported measure developed that can capture the entire disease.

Third, from a physician’s point of view, Dr. Del Galdo said, “We want to send the patients into remission. We need to continue to further deconvolute the clinical manifestations and find the bottleneck at the beginning of the natural history of disease.

“If we can find a drug that is effective very early on, before the patients start getting the eight different manifestations with different levels of severity, then we will be on the right road, which we hope will end in remission.”

Dr. Del Galdo has served on the speakers bureau for AstraZeneca and Janssen; consulted for AstraZeneca, Boehringer Ingelheim, Capella, Chemomab, Janssen, and Mitsubishi-Tanabe; and received grant or research support from AbbVie, AstraZeneca, Boheringer Ingelheim, Capella, Chemomab, Kymab, Janssen, and Mitsubishi-Tanabe. Dr. Landewé had no relevant disclosures.

A version of this article first appeared on Medscape.com.

VIENNA – The use of immunosuppressive and antifibrotic drugs to treat skin and lung fibrosis leads updated recommendations from the European Alliance of Associations for Rheumatology (EULAR) for the treatment of systemic sclerosis.

“The most impactful new recommendation relates to the evidence for immunosuppressive agents and antifibrotics for the treatment of skin fibrosis and lung fibrosis,” said Francesco Del Galdo, MD, PhD, professor of experimental medicine, consultant rheumatologist, and scleroderma and connective tissue diseases specialist at Leeds Teaching Hospitals NHS Trust, Leeds, England. Dr. Del Galdo presented the update at the annual European Congress of Rheumatology.

“But there are also new recommendations, including a redefined target population for hematopoietic stem cell transplantation following cyclophosphamide, the upfront combination treatment at the time of diagnosis of pulmonary arterial hypertension [PAH], and a negative recommendation for the use of anticoagulants for pulmonary arterial hypertension,” noted Dr. Del Galdo, highlighting key updates in the 2024 recommendations.

Robert B.M. Landewé, MD, PhD, professor and rheumatologist at Amsterdam University Medical Center, Amsterdam, the Netherlands, and Zuyderland Medical Center, Heerlen, the Netherlands, co-moderated the session on EULAR recommendations. “The management of systemic sclerosis is a field in which a lot is happening,” he said. “The last update goes back to 2017, and in the meantime, many new approaches have seen the light, especially pertaining to skin fibrosis and interstitial lung disease. Six new recommendations have been coined, covering drugs like mycophenolate mofetil, nintedanib, rituximab, and tocilizumab. None of these therapies were present in the 2017 recommendations. It seems the field is now ready to further expand on targeted therapies for the management of musculoskeletal and gastrointestinal manifestations, calcinosis, and the local management of digital ulcers.”
 

‘Therapeutic Continuums’ Aid Disease Management

Dr. Del Galdo and his colleagues grouped the various interventions across what the recommendations label as evidence-backed “therapeutic continuums.” These span six of the eight different clinical manifestations of systemic sclerosis: Raynaud’s phenomenon, digital ulcers, pulmonary hypertension, musculoskeletal manifestations, skin fibrosis, interstitial lung disease (ILD), and gastrointestinal and renal crisis.

A slide showing the different strengths of evidence for various drugs across the eight manifestations illustrated the principle behind the therapeutic continuums. “These ‘therapeutic continuums’ suggest a common pathogenetic mechanism driving the various manifestations of disease,” said Dr. Del Galdo. For example, he noted, “If rituximab had a positive response in skin and in lung, it suggests that B cells play a role in the clinical manifestations of skin and lung in this disease.”

Dr. Del Galdo highlighted the new immunosuppression continuum and associated treatments for skin and lung fibrosis. “For skin involvement, the task force recommended mycophenolate, methotrexate, and rituximab, with tocilizumab having a lower level of evidence and lower recommendation strength; similarly, in interstitial lung disease, we have rituximab, mycophenolate, cyclophosphamide, and nintedanib, and these all have the highest strength of evidence. Tocilizumab is assigned one strength of evidence below the other drugs.”

He also cited the phosphodiesterase 5 inhibitor (PDE5i) drugs that are used across Raynaud’s phenomenon, digital ulcers, and pulmonary arterial hypertension, which together form a vascular therapeutic continuum.

The complexity of systemic sclerosis and multiple manifestations was a major determinant of the recommendations, Dr. Del Galdo pointed out. “The task force realized that since this is such a complex disease, we cannot recommend one treatment unconditionally. For example, with mycophenolate mofetil, what works for most patients for the skin and lung manifestations might not for someone who experiences severe diarrhea, in which mycophenolate is contraindicated. So, the highest degree of recommendation that the task force felt comfortable with was ‘should be considered.’ ”

Dr. Del Galdo stressed that the complex nature of systemic sclerosis means that “when thinking of treating one manifestation, you also always need to consider all the other clinical manifestations as experienced by the patient, and it is this multifaceted scenario that will ultimately lead to your final choice.”

Turning to new evidence around drug use, Dr. Del Galdo said that rituximab has the highest level of evidence across skin and lung manifestations, nintedanib is new in lung, and tocilizumab is new across both skin and lung.

To treat systemic sclerosis–pulmonary arterial hypertension (SSc-PAH), as long as there are no contraindications, the task force recommends using PDE5i and endothelin receptor antagonists (ERAs) at diagnosis. Data from phase 3 trials show a better outcome when the combination is established early.

The task force suggests avoiding the use of warfarin in PAH. “This is supported by a signal from two trials showing an increase in morbidity and mortality in these patients,” noted Dr. Del Galdo.

He also pointed out that selexipag and riociguat were new and important second-line additions for the treatment of PAH, and — consistent with the ERA approach — the EULAR recommendation supports frequent follow-up to establish a treat-to-target approach to maximizing clinical outcomes in SSc-PAH and SSc-ILD. “Specifically, for the first time, we recommend monitoring the effect of any chosen intervention selected within 3-6 months of starting. The evidence suggests there is a group of patients who respond and some who respond less well and who might benefit from a second-line intervention.”

For example, results of one trial support the approach of adding an antifibrotic agent to reduce progression in people with progressive lung fibrosis. “Similarly, for pulmonary hypertension, we recommend putting patients on dual treatment, and if this fails, place them on selexipag or switch the PDE5i to riociguat,” Dr. Del Galdo said.
 

Systemic Sclerosis Research Agenda and Recommendations Align

Dr. Del Galdo highlighted the value of therapeutic continuums in advancing disease understanding. “It is starting to teach us what we know and what we don’t and where do we need to build more evidence. Effectively, they determine where the gaps in therapy lie, and this starts to guide the research agenda.

“In fact, what is really interesting about this recommendation update — certainly from the perspective of disease understanding — is that we are starting to have a bird’s-eye view of the clinical manifestations of scleroderma that have so often been dealt with separately. Now we are starting to build a cumulative evidence map of this disease.”

In 2017, the research agenda largely advocated identifying immune-targeting drugs for skin and lung fibrosis, Dr. Del Galdo pointed out. “Now, we’ve done that — we’ve identified appropriate immunosuppressive drugs — and this is testimony to the importance of these recommendations because what prioritized the research agenda 10 years ago ended up informing the clinical trials and made it into the recommendations.”

“We definitely are one step forward compared to this 2017 recommendation and closer to what we would like to do,” he asserted.
 

Remission Elusive but Getting Closer

In some respects, according to Dr. Del Galdo, research and development is making relatively slow progress, especially compared with other rheumatologic diseases such as rheumatoid arthritis. “We cannot put patients with systemic sclerosis in remission yet. But I think we are one step ahead in that we’ve now established the treat-to-target approach to maximize the efficacy with which we can stall disease progression, but we cannot yet put these patients into remission,” he said. Systemic sclerosis has multiple manifestations, and fibrotic damage cannot be reversed. “Right now, the scar will remain there forever,” he noted.

Until remission is achievable, Dr. Del Galdo advises diagnosing and treating patients earlier to prevent fibrotic manifestations.

Dr. Del Galdo explained the three leading priorities on the systemic sclerosis research agenda. “There are three because it is such a complex disease. The first is considering the patient voice — this is the most important one, and the patients say they want a more holistic approach — so trialing and treating multiple manifestations together.”

Second, Dr. Del Galdo said, he would like to see a patient-reported measure developed that can capture the entire disease.

Third, from a physician’s point of view, Dr. Del Galdo said, “We want to send the patients into remission. We need to continue to further deconvolute the clinical manifestations and find the bottleneck at the beginning of the natural history of disease.

“If we can find a drug that is effective very early on, before the patients start getting the eight different manifestations with different levels of severity, then we will be on the right road, which we hope will end in remission.”

Dr. Del Galdo has served on the speakers bureau for AstraZeneca and Janssen; consulted for AstraZeneca, Boehringer Ingelheim, Capella, Chemomab, Janssen, and Mitsubishi-Tanabe; and received grant or research support from AbbVie, AstraZeneca, Boheringer Ingelheim, Capella, Chemomab, Kymab, Janssen, and Mitsubishi-Tanabe. Dr. Landewé had no relevant disclosures.

A version of this article first appeared on Medscape.com.

Type 2 diabetes (T2D) is the most common form of diabetes, representing more than 90% of all cases worldwide. The prevalence of T2D is increasing globally, mainly because of behavioral and social factors related to obesity, diet, and physical activity. The International Diabetes Federation estimated in its 2021 report that 537 million adults aged between 20 and 79 years have been diagnosed with diabetes worldwide. The organization predicts an increase to 643 million by 2030 and 743 million by 2045.

The main therapeutic goals for patients with T2D include adequate glycemic control and primary and secondary prevention of atherosclerotic cardiovascular and renal diseases, which represent nearly half of all deaths among adults with T2D. Despite the multiple treatment options available, 16% of adults with T2D have inadequate glycemic control, including hemoglobin A1c levels greater than 9%, even though glycemic control was the focus of the 2017 guidelines of the American College of Physicians.

Therefore, the ACP deemed it necessary to update the previous guidelines, considering new evidence on the efficacy and harms of new pharmacologic treatments in adults with T2D with the goal of reducing the risk for all-cause mortality, cardiovascular morbidity, and progression of chronic kidney disease (CKD) in these patients.
 

New Drugs

The pharmacologic treatments that the ACP considered while updating its guidelines include glucagon-like peptide 1 (GLP-1) receptor agonists (that is, dulaglutide, exenatide, liraglutide, lixisenatide, and semaglutide), a GLP-1 receptor agonist and a glucose-dependent insulinotropic polypeptide receptor agonist (that is, tirzepatide), sodium-glucose cotransporter 2 (SGLT-2) inhibitors (that is, canagliflozin, dapagliflozin, empagliflozin, and ertugliflozin), dipeptidyl peptidase 4 (DPP-4) inhibitors (that is, alogliptin, linagliptin, saxagliptin, and sitagliptin), and long-acting insulins (that is, insulin glargine and insulin degludec).

Recommendations

The ACP recommends adding an SGLT-2 inhibitor or a GLP-1 agonist to metformin and lifestyle modifications in adults with inadequately controlled T2D (strong recommendation, high certainty of evidence). Use an SGLT-2 inhibitor to reduce the risk for all-cause mortality, major adverse cardiovascular events (MACE), CKD progression, and hospitalization resulting from heart failure, according to the document. Use a GLP-1 agonist to reduce the risk for all-cause mortality, MACE, and strokes.

SGLT-2 inhibitors and GLP-1 agonists are the only newer pharmacological treatments for T2D that have reduced all-cause mortality than placebo or usual care. In indirect comparison, SGLT-2 inhibitors probably reduce the risk for hospitalization resulting from heart failure, while GLP-1 agonists probably reduce the risk for strokes.

Neither class of drugs causes severe hypoglycemia, but both are associated with various harms, as reported in specific warnings. Both classes of drugs lead to weight loss.

Compared with long-acting insulins, SGLT-2 inhibitors can reduce, and GLP-1 agonists probably reduce, all-cause mortality. Compared with DPP-4 inhibitors, GLP-1 agonists probably reduce all-cause mortality.

Compared with DPP-4 inhibitors, SGLT-2 inhibitors probably reduce MACE, as well as compared with sulfonylureas.

The ACP recommends against adding a DPP-4 inhibitor to metformin and lifestyle modifications in adults with inadequately controlled T2D to reduce morbidity and all-cause mortality (strong recommendation, high certainty of evidence).

Compared with usual therapy, DPP-4 inhibitors do not result in differences in all-cause mortality, MACE, myocardial infarction, stroke, hospitalization for chronic heart failure (CHF), CKD progression, or severe hypoglycemia. Compared with SGLT-2 inhibitors, DPP-4 inhibitors may increase hospitalization caused by CHF and probably increase the risk for MACE and CKD progression. Compared with GLP-1 agonists, they probably increase all-cause mortality and hospitalization caused by CHF and the risk for MACE. Metformin is the most common usual therapy in the studies considered.
 

Considerations for Practice

Metformin (unless contraindicated) and lifestyle modifications represent the first step in managing T2D in most patients, according to the ACP.

The choice of additional therapy requires a risk/benefit assessment and should be personalized on the basis of patient preferences, glycemic control goals, comorbidities, and the risk for hypoglycemia. SGLT-2 inhibitors can be added in patients with T2D and CHF or CKD, according to the ACP. GLP-1 agonists can be added in patients with T2D at increased risk for stroke or for whom total body weight loss is a significant therapeutic goal.

The A1c target should be considered between 7% and 8% in most adults with T2D, and de-escalation of pharmacologic treatments should be considered for A1c levels less than 6.5%. Self-monitoring of blood glucose may not be necessary in patients treated with metformin in combination with an SGLT-2 inhibitor or a GLP-1 agonist, according to the ACP.

The document also holds that, in cases of adequate glycemic control with the addition of an SGLT-2 inhibitor or a GLP-1 agonist, existing treatment with sulfonylureas or long-acting insulin should be reduced or stopped due to the increased risk for severe hypoglycemia.

This story was translated from Univadis Italy, which is part of the Medscape professional network, using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.

A version of this article appeared on Medscape.com.

Type 2 diabetes (T2D) is the most common form of diabetes, representing more than 90% of all cases worldwide. The prevalence of T2D is increasing globally, mainly because of behavioral and social factors related to obesity, diet, and physical activity. The International Diabetes Federation estimated in its 2021 report that 537 million adults aged between 20 and 79 years have been diagnosed with diabetes worldwide. The organization predicts an increase to 643 million by 2030 and 743 million by 2045.

The main therapeutic goals for patients with T2D include adequate glycemic control and primary and secondary prevention of atherosclerotic cardiovascular and renal diseases, which represent nearly half of all deaths among adults with T2D. Despite the multiple treatment options available, 16% of adults with T2D have inadequate glycemic control, including hemoglobin A1c levels greater than 9%, even though glycemic control was the focus of the 2017 guidelines of the American College of Physicians.

Therefore, the ACP deemed it necessary to update the previous guidelines, considering new evidence on the efficacy and harms of new pharmacologic treatments in adults with T2D with the goal of reducing the risk for all-cause mortality, cardiovascular morbidity, and progression of chronic kidney disease (CKD) in these patients.
 

New Drugs

The pharmacologic treatments that the ACP considered while updating its guidelines include glucagon-like peptide 1 (GLP-1) receptor agonists (that is, dulaglutide, exenatide, liraglutide, lixisenatide, and semaglutide), a GLP-1 receptor agonist and a glucose-dependent insulinotropic polypeptide receptor agonist (that is, tirzepatide), sodium-glucose cotransporter 2 (SGLT-2) inhibitors (that is, canagliflozin, dapagliflozin, empagliflozin, and ertugliflozin), dipeptidyl peptidase 4 (DPP-4) inhibitors (that is, alogliptin, linagliptin, saxagliptin, and sitagliptin), and long-acting insulins (that is, insulin glargine and insulin degludec).

Recommendations

The ACP recommends adding an SGLT-2 inhibitor or a GLP-1 agonist to metformin and lifestyle modifications in adults with inadequately controlled T2D (strong recommendation, high certainty of evidence). Use an SGLT-2 inhibitor to reduce the risk for all-cause mortality, major adverse cardiovascular events (MACE), CKD progression, and hospitalization resulting from heart failure, according to the document. Use a GLP-1 agonist to reduce the risk for all-cause mortality, MACE, and strokes.

SGLT-2 inhibitors and GLP-1 agonists are the only newer pharmacological treatments for T2D that have reduced all-cause mortality than placebo or usual care. In indirect comparison, SGLT-2 inhibitors probably reduce the risk for hospitalization resulting from heart failure, while GLP-1 agonists probably reduce the risk for strokes.

Neither class of drugs causes severe hypoglycemia, but both are associated with various harms, as reported in specific warnings. Both classes of drugs lead to weight loss.

Compared with long-acting insulins, SGLT-2 inhibitors can reduce, and GLP-1 agonists probably reduce, all-cause mortality. Compared with DPP-4 inhibitors, GLP-1 agonists probably reduce all-cause mortality.

Compared with DPP-4 inhibitors, SGLT-2 inhibitors probably reduce MACE, as well as compared with sulfonylureas.

The ACP recommends against adding a DPP-4 inhibitor to metformin and lifestyle modifications in adults with inadequately controlled T2D to reduce morbidity and all-cause mortality (strong recommendation, high certainty of evidence).

Compared with usual therapy, DPP-4 inhibitors do not result in differences in all-cause mortality, MACE, myocardial infarction, stroke, hospitalization for chronic heart failure (CHF), CKD progression, or severe hypoglycemia. Compared with SGLT-2 inhibitors, DPP-4 inhibitors may increase hospitalization caused by CHF and probably increase the risk for MACE and CKD progression. Compared with GLP-1 agonists, they probably increase all-cause mortality and hospitalization caused by CHF and the risk for MACE. Metformin is the most common usual therapy in the studies considered.
 

Considerations for Practice

Metformin (unless contraindicated) and lifestyle modifications represent the first step in managing T2D in most patients, according to the ACP.

The choice of additional therapy requires a risk/benefit assessment and should be personalized on the basis of patient preferences, glycemic control goals, comorbidities, and the risk for hypoglycemia. SGLT-2 inhibitors can be added in patients with T2D and CHF or CKD, according to the ACP. GLP-1 agonists can be added in patients with T2D at increased risk for stroke or for whom total body weight loss is a significant therapeutic goal.

The A1c target should be considered between 7% and 8% in most adults with T2D, and de-escalation of pharmacologic treatments should be considered for A1c levels less than 6.5%. Self-monitoring of blood glucose may not be necessary in patients treated with metformin in combination with an SGLT-2 inhibitor or a GLP-1 agonist, according to the ACP.

The document also holds that, in cases of adequate glycemic control with the addition of an SGLT-2 inhibitor or a GLP-1 agonist, existing treatment with sulfonylureas or long-acting insulin should be reduced or stopped due to the increased risk for severe hypoglycemia.

This story was translated from Univadis Italy, which is part of the Medscape professional network, using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.

A version of this article appeared on Medscape.com.

Type 2 diabetes (T2D) is the most common form of diabetes, representing more than 90% of all cases worldwide. The prevalence of T2D is increasing globally, mainly because of behavioral and social factors related to obesity, diet, and physical activity. The International Diabetes Federation estimated in its 2021 report that 537 million adults aged between 20 and 79 years have been diagnosed with diabetes worldwide. The organization predicts an increase to 643 million by 2030 and 743 million by 2045.

The main therapeutic goals for patients with T2D include adequate glycemic control and primary and secondary prevention of atherosclerotic cardiovascular and renal diseases, which represent nearly half of all deaths among adults with T2D. Despite the multiple treatment options available, 16% of adults with T2D have inadequate glycemic control, including hemoglobin A1c levels greater than 9%, even though glycemic control was the focus of the 2017 guidelines of the American College of Physicians.

Therefore, the ACP deemed it necessary to update the previous guidelines, considering new evidence on the efficacy and harms of new pharmacologic treatments in adults with T2D with the goal of reducing the risk for all-cause mortality, cardiovascular morbidity, and progression of chronic kidney disease (CKD) in these patients.
 

New Drugs

The pharmacologic treatments that the ACP considered while updating its guidelines include glucagon-like peptide 1 (GLP-1) receptor agonists (that is, dulaglutide, exenatide, liraglutide, lixisenatide, and semaglutide), a GLP-1 receptor agonist and a glucose-dependent insulinotropic polypeptide receptor agonist (that is, tirzepatide), sodium-glucose cotransporter 2 (SGLT-2) inhibitors (that is, canagliflozin, dapagliflozin, empagliflozin, and ertugliflozin), dipeptidyl peptidase 4 (DPP-4) inhibitors (that is, alogliptin, linagliptin, saxagliptin, and sitagliptin), and long-acting insulins (that is, insulin glargine and insulin degludec).

Recommendations

The ACP recommends adding an SGLT-2 inhibitor or a GLP-1 agonist to metformin and lifestyle modifications in adults with inadequately controlled T2D (strong recommendation, high certainty of evidence). Use an SGLT-2 inhibitor to reduce the risk for all-cause mortality, major adverse cardiovascular events (MACE), CKD progression, and hospitalization resulting from heart failure, according to the document. Use a GLP-1 agonist to reduce the risk for all-cause mortality, MACE, and strokes.

SGLT-2 inhibitors and GLP-1 agonists are the only newer pharmacological treatments for T2D that have reduced all-cause mortality than placebo or usual care. In indirect comparison, SGLT-2 inhibitors probably reduce the risk for hospitalization resulting from heart failure, while GLP-1 agonists probably reduce the risk for strokes.

Neither class of drugs causes severe hypoglycemia, but both are associated with various harms, as reported in specific warnings. Both classes of drugs lead to weight loss.

Compared with long-acting insulins, SGLT-2 inhibitors can reduce, and GLP-1 agonists probably reduce, all-cause mortality. Compared with DPP-4 inhibitors, GLP-1 agonists probably reduce all-cause mortality.

Compared with DPP-4 inhibitors, SGLT-2 inhibitors probably reduce MACE, as well as compared with sulfonylureas.

The ACP recommends against adding a DPP-4 inhibitor to metformin and lifestyle modifications in adults with inadequately controlled T2D to reduce morbidity and all-cause mortality (strong recommendation, high certainty of evidence).

Compared with usual therapy, DPP-4 inhibitors do not result in differences in all-cause mortality, MACE, myocardial infarction, stroke, hospitalization for chronic heart failure (CHF), CKD progression, or severe hypoglycemia. Compared with SGLT-2 inhibitors, DPP-4 inhibitors may increase hospitalization caused by CHF and probably increase the risk for MACE and CKD progression. Compared with GLP-1 agonists, they probably increase all-cause mortality and hospitalization caused by CHF and the risk for MACE. Metformin is the most common usual therapy in the studies considered.
 

Considerations for Practice

Metformin (unless contraindicated) and lifestyle modifications represent the first step in managing T2D in most patients, according to the ACP.

The choice of additional therapy requires a risk/benefit assessment and should be personalized on the basis of patient preferences, glycemic control goals, comorbidities, and the risk for hypoglycemia. SGLT-2 inhibitors can be added in patients with T2D and CHF or CKD, according to the ACP. GLP-1 agonists can be added in patients with T2D at increased risk for stroke or for whom total body weight loss is a significant therapeutic goal.

The A1c target should be considered between 7% and 8% in most adults with T2D, and de-escalation of pharmacologic treatments should be considered for A1c levels less than 6.5%. Self-monitoring of blood glucose may not be necessary in patients treated with metformin in combination with an SGLT-2 inhibitor or a GLP-1 agonist, according to the ACP.

The document also holds that, in cases of adequate glycemic control with the addition of an SGLT-2 inhibitor or a GLP-1 agonist, existing treatment with sulfonylureas or long-acting insulin should be reduced or stopped due to the increased risk for severe hypoglycemia.

This story was translated from Univadis Italy, which is part of the Medscape professional network, using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.

A version of this article appeared on Medscape.com.

This transcript has been edited for clarity. 

There is a helpful consensus from experts on the best management of patients with acute sore throat. This is a common problem in primary care, and one for which there is a lot of evidence, opinion, and ultimately overprescribing of antibiotics. This consensus presents a pragmatic clinical approach aimed at decreasing overprescribing, yet detecting which patients are likely to benefit from treatment with antibiotics. 

Let’s first go over the evidence that forms the basis for the recommendations, then the recommended approach. First, a sore throat can be caused by many different viruses, as well as group A streptococcus (GAS), the group C streptococcus S dysgalactiae, and fusobacterium. We sometimes think of throat cultures as telling us the definitive etiology of a sore throat. In fact, children commonly are colonized with GAS even when not infected — 35% of the time, when GAS is detected on throat swab in a child, GAS is not the cause of the sore throat. Very few adults are colonized with GAS.

Sore throats are usually self-limited, whether they are treated with antibiotics or not, but occasionally complications can occur. Suppurative complications include peritonsillar abscess, sinusitis and sepsis. Nonsuppurative complications are primarily glomerulonephritis and rheumatic fever, which can lead to rheumatic heart disease. 

Antibiotics. Antibiotics have three potential benefits in acute sore throat: to reduce the risk of developing rheumatic heart disease, reduce the duration and severity of symptoms, and treat suppurative complications. The risk for rheumatic heart disease has almost vanished in high-income countries, but not in low-income countries. Thus, antibiotic treatment of acute sore throat due to GAS may benefit those in living in, and those who recently emigrated from, low-income countries. 

Patients with suppurative complications should be identified because antibiotics are important for this group. Although antibiotics are prescribed primarily to prevent rheumatic fever in this population, they may be mildly helpful in reducing a patient’s symptoms. 

Testing. The sensitivity and specificity of high-quality point-of-care tests (POCTs) are on par with those of cultures, with the advantage that the results are available within minutes. Negative tests reduce unneeded antibiotic prescriptions.

Given this evidence, the authors recommend an approach that puts a lot of emphasis on two major things: the risk for rheumatic fever, and clinical assessment. On the basis of these factors, a decision is made about the utility of POCTs and treatment with antibiotics for GAS. The risk for rheumatic fever is based on epidemiology: If the patient is in a low-income country or has recently immigrated from one, then the risk is high, and if not, the risk is low.

Complicated vs uncomplicated? This is determined by clinical assessment of the severity of the patient’s illness, including general appearance. Uncomplicated sore throat means that the patient:

• Is not getting worse after 3 days of illness
• Has a duration of illness ≤ 5 days or is getting better after day 5
• Has mild to moderate symptom severity (bilateral throat pain, the ability to open the mouth fully, and absence of a sandpaper or scarlatiniform rash or strawberry tongue)

For patients with uncomplicated sore throat and low risk for rheumatic fever, the main goals are to reduce antibiotic use and provide symptomatic relief. For these patients, an assessment such as the Centor score can be done. Those with a low Centor score (0-2) can be treated with analgesics and there is no need for a POCT.

In patients with a higher Centor score, the consensus gives two choices: They can either be tested (and treated if the testing is positive), or it is reasonable to forgo testing and use a wait-and-see strategy, with reevaluation if they are getting worse after day 3 or not improving after day 5 days of their illness. Illnesses that last longer than 5 days with sore throat and fatigue should prompt consideration of alternative diagnoses, such as infectious mononucleosis. 

For patients with potentially complicated sore throat — including indicators such as worsening symptoms after 3 days or worsening after initiation of antibiotics, inability to open the mouth fully, unilateral neck pain or swelling, or rigors — should undergo a careful evaluation. The need for further testing in these patients, including labs and imaging, should be decided on a case-by-case basis. If the patient appears seriously ill, don’t rely solely on POCT for GAS, but think about other diagnoses. 

Rheumatic fever. The approach is very different in patients at high risk for rheumatic fever. POCT for GAS is recommended irrespective of their clinical score, and antibiotics should be prescribed if it’s positive for GAS. If a POCT is unavailable, then the consensus recommends prescribing antibiotics for all high-risk patients who have acute sore throat. 

This approach is sensible and puts a lot of emphasis on clinical evaluation, though it should be noted that this approach is considerably different from that in the 2012 Infectious Diseases Society of America guidelines. 
 

Dr. Skolnik, professor, Department of Family Medicine, Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, and associate director, Department of Family Medicine, Abington Jefferson Health, Abington, Pennsylvania, disclosed ties with AstraZeneca, Teva, Eli Lilly and Company, Boehringer Ingelheim, Sanofi, Sanofi Pasteur, GlaxoSmithKline, Merck, and Bayer.

A version of this article appeared on Medscape.com.

This transcript has been edited for clarity. 

There is a helpful consensus from experts on the best management of patients with acute sore throat. This is a common problem in primary care, and one for which there is a lot of evidence, opinion, and ultimately overprescribing of antibiotics. This consensus presents a pragmatic clinical approach aimed at decreasing overprescribing, yet detecting which patients are likely to benefit from treatment with antibiotics. 

Let’s first go over the evidence that forms the basis for the recommendations, then the recommended approach. First, a sore throat can be caused by many different viruses, as well as group A streptococcus (GAS), the group C streptococcus S dysgalactiae, and fusobacterium. We sometimes think of throat cultures as telling us the definitive etiology of a sore throat. In fact, children commonly are colonized with GAS even when not infected — 35% of the time, when GAS is detected on throat swab in a child, GAS is not the cause of the sore throat. Very few adults are colonized with GAS.

Sore throats are usually self-limited, whether they are treated with antibiotics or not, but occasionally complications can occur. Suppurative complications include peritonsillar abscess, sinusitis and sepsis. Nonsuppurative complications are primarily glomerulonephritis and rheumatic fever, which can lead to rheumatic heart disease. 

Antibiotics. Antibiotics have three potential benefits in acute sore throat: to reduce the risk of developing rheumatic heart disease, reduce the duration and severity of symptoms, and treat suppurative complications. The risk for rheumatic heart disease has almost vanished in high-income countries, but not in low-income countries. Thus, antibiotic treatment of acute sore throat due to GAS may benefit those in living in, and those who recently emigrated from, low-income countries. 

Patients with suppurative complications should be identified because antibiotics are important for this group. Although antibiotics are prescribed primarily to prevent rheumatic fever in this population, they may be mildly helpful in reducing a patient’s symptoms. 

Testing. The sensitivity and specificity of high-quality point-of-care tests (POCTs) are on par with those of cultures, with the advantage that the results are available within minutes. Negative tests reduce unneeded antibiotic prescriptions.

Given this evidence, the authors recommend an approach that puts a lot of emphasis on two major things: the risk for rheumatic fever, and clinical assessment. On the basis of these factors, a decision is made about the utility of POCTs and treatment with antibiotics for GAS. The risk for rheumatic fever is based on epidemiology: If the patient is in a low-income country or has recently immigrated from one, then the risk is high, and if not, the risk is low.

Complicated vs uncomplicated? This is determined by clinical assessment of the severity of the patient’s illness, including general appearance. Uncomplicated sore throat means that the patient:

• Is not getting worse after 3 days of illness
• Has a duration of illness ≤ 5 days or is getting better after day 5
• Has mild to moderate symptom severity (bilateral throat pain, the ability to open the mouth fully, and absence of a sandpaper or scarlatiniform rash or strawberry tongue)

For patients with uncomplicated sore throat and low risk for rheumatic fever, the main goals are to reduce antibiotic use and provide symptomatic relief. For these patients, an assessment such as the Centor score can be done. Those with a low Centor score (0-2) can be treated with analgesics and there is no need for a POCT.

In patients with a higher Centor score, the consensus gives two choices: They can either be tested (and treated if the testing is positive), or it is reasonable to forgo testing and use a wait-and-see strategy, with reevaluation if they are getting worse after day 3 or not improving after day 5 days of their illness. Illnesses that last longer than 5 days with sore throat and fatigue should prompt consideration of alternative diagnoses, such as infectious mononucleosis. 

For patients with potentially complicated sore throat — including indicators such as worsening symptoms after 3 days or worsening after initiation of antibiotics, inability to open the mouth fully, unilateral neck pain or swelling, or rigors — should undergo a careful evaluation. The need for further testing in these patients, including labs and imaging, should be decided on a case-by-case basis. If the patient appears seriously ill, don’t rely solely on POCT for GAS, but think about other diagnoses. 

Rheumatic fever. The approach is very different in patients at high risk for rheumatic fever. POCT for GAS is recommended irrespective of their clinical score, and antibiotics should be prescribed if it’s positive for GAS. If a POCT is unavailable, then the consensus recommends prescribing antibiotics for all high-risk patients who have acute sore throat. 

This approach is sensible and puts a lot of emphasis on clinical evaluation, though it should be noted that this approach is considerably different from that in the 2012 Infectious Diseases Society of America guidelines. 
 

Dr. Skolnik, professor, Department of Family Medicine, Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, and associate director, Department of Family Medicine, Abington Jefferson Health, Abington, Pennsylvania, disclosed ties with AstraZeneca, Teva, Eli Lilly and Company, Boehringer Ingelheim, Sanofi, Sanofi Pasteur, GlaxoSmithKline, Merck, and Bayer.

A version of this article appeared on Medscape.com.

This transcript has been edited for clarity. 

There is a helpful consensus from experts on the best management of patients with acute sore throat. This is a common problem in primary care, and one for which there is a lot of evidence, opinion, and ultimately overprescribing of antibiotics. This consensus presents a pragmatic clinical approach aimed at decreasing overprescribing, yet detecting which patients are likely to benefit from treatment with antibiotics. 

Let’s first go over the evidence that forms the basis for the recommendations, then the recommended approach. First, a sore throat can be caused by many different viruses, as well as group A streptococcus (GAS), the group C streptococcus S dysgalactiae, and fusobacterium. We sometimes think of throat cultures as telling us the definitive etiology of a sore throat. In fact, children commonly are colonized with GAS even when not infected — 35% of the time, when GAS is detected on throat swab in a child, GAS is not the cause of the sore throat. Very few adults are colonized with GAS.

Sore throats are usually self-limited, whether they are treated with antibiotics or not, but occasionally complications can occur. Suppurative complications include peritonsillar abscess, sinusitis and sepsis. Nonsuppurative complications are primarily glomerulonephritis and rheumatic fever, which can lead to rheumatic heart disease. 

Antibiotics. Antibiotics have three potential benefits in acute sore throat: to reduce the risk of developing rheumatic heart disease, reduce the duration and severity of symptoms, and treat suppurative complications. The risk for rheumatic heart disease has almost vanished in high-income countries, but not in low-income countries. Thus, antibiotic treatment of acute sore throat due to GAS may benefit those in living in, and those who recently emigrated from, low-income countries. 

Patients with suppurative complications should be identified because antibiotics are important for this group. Although antibiotics are prescribed primarily to prevent rheumatic fever in this population, they may be mildly helpful in reducing a patient’s symptoms. 

Testing. The sensitivity and specificity of high-quality point-of-care tests (POCTs) are on par with those of cultures, with the advantage that the results are available within minutes. Negative tests reduce unneeded antibiotic prescriptions.

Given this evidence, the authors recommend an approach that puts a lot of emphasis on two major things: the risk for rheumatic fever, and clinical assessment. On the basis of these factors, a decision is made about the utility of POCTs and treatment with antibiotics for GAS. The risk for rheumatic fever is based on epidemiology: If the patient is in a low-income country or has recently immigrated from one, then the risk is high, and if not, the risk is low.

Complicated vs uncomplicated? This is determined by clinical assessment of the severity of the patient’s illness, including general appearance. Uncomplicated sore throat means that the patient:

• Is not getting worse after 3 days of illness
• Has a duration of illness ≤ 5 days or is getting better after day 5
• Has mild to moderate symptom severity (bilateral throat pain, the ability to open the mouth fully, and absence of a sandpaper or scarlatiniform rash or strawberry tongue)

For patients with uncomplicated sore throat and low risk for rheumatic fever, the main goals are to reduce antibiotic use and provide symptomatic relief. For these patients, an assessment such as the Centor score can be done. Those with a low Centor score (0-2) can be treated with analgesics and there is no need for a POCT.

In patients with a higher Centor score, the consensus gives two choices: They can either be tested (and treated if the testing is positive), or it is reasonable to forgo testing and use a wait-and-see strategy, with reevaluation if they are getting worse after day 3 or not improving after day 5 days of their illness. Illnesses that last longer than 5 days with sore throat and fatigue should prompt consideration of alternative diagnoses, such as infectious mononucleosis. 

For patients with potentially complicated sore throat — including indicators such as worsening symptoms after 3 days or worsening after initiation of antibiotics, inability to open the mouth fully, unilateral neck pain or swelling, or rigors — should undergo a careful evaluation. The need for further testing in these patients, including labs and imaging, should be decided on a case-by-case basis. If the patient appears seriously ill, don’t rely solely on POCT for GAS, but think about other diagnoses. 

Rheumatic fever. The approach is very different in patients at high risk for rheumatic fever. POCT for GAS is recommended irrespective of their clinical score, and antibiotics should be prescribed if it’s positive for GAS. If a POCT is unavailable, then the consensus recommends prescribing antibiotics for all high-risk patients who have acute sore throat. 

This approach is sensible and puts a lot of emphasis on clinical evaluation, though it should be noted that this approach is considerably different from that in the 2012 Infectious Diseases Society of America guidelines. 
 

Dr. Skolnik, professor, Department of Family Medicine, Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, and associate director, Department of Family Medicine, Abington Jefferson Health, Abington, Pennsylvania, disclosed ties with AstraZeneca, Teva, Eli Lilly and Company, Boehringer Ingelheim, Sanofi, Sanofi Pasteur, GlaxoSmithKline, Merck, and Bayer.

A version of this article appeared on Medscape.com.

An influential US panel may largely reaffirm its current recommendation in favor of screening older women to prevent osteoporotic fractures, while also repeating its call for more research to try to determine whether men would benefit from this kind of routine testing.

The US Preventive Services Task Force (USPSTF) on June 11 released a draft update of its recommendations on osteoporosis screening. The task force will accept comments on the draft through July 8. Federal law gives the USPSTF recommendations extra clout, requiring insurers to cover — without co-pay — services that get top marks “A” or “B” from the task force.

The task force intends to maintain a “B” recommendation on screening of older women, indicating that the evidence gathered to date suggests a moderate net benefit. But the draft includes a shift in the approach to this screening.

The USPSTF proposed saying that it recommends screening for osteoporosis in both women aged 65 years and older and postmenopausal women younger than 65 years who are at an increased risk for an osteoporotic fracture. The current recommendation, finalized in 2018, advises “screening for osteoporosis with bone measurement testing [emphasis added]” for these groups.

The proposed change in language — dropping the phrase “with bone measurement testing” — is intended to expand flexibility for clinicians, Esa Davis, MD, MPH, a member of USPSTF and a professor at the University of Maryland School of Medicine, Baltimore, told this news organization.

kurastomokibridichuwronushutodretrovametrofreslowesuclolajuthushotruswespecishoswovigekagespouekisusovogilispuhaswichakubraclubregumunumiclapritreclitrithawajikofrejocreshivekiuopacosletacruprocruclijephibicriclagogabospetok

cutedostochastovavukowrawilacrocluhuresavemeswediwretiniwroclolonoswutresliwot

Insufficient Evidence

The USPSTF’s “I” grade is different from a “D” grade, which is what the task force uses to recommend against the use of a service.

A “D” grade means the USPSTF says there is moderate or high certainty that the service has no net benefit or that the harms outweigh the benefits. (The USPSTF makes it easy to search online for grades given to preventive services, including those that got a “D.”)

The USPSTF is calling for more studies on the benefits and harms of screening for osteoporosis to prevent fractures and related morbidity and mortality in men.

“Men do get osteoporosis,” Dr. Davis said. “But unfortunately, the evidence isn’t there” to allow USPSTF to make a recommendation on screening approaches.

“Any man who has concerns about bone health should certainly talk to his clinician and figure out what is the best form of screening” he might want to do, she said.

There’s been a growing interest in the question of whether to screen men for osteoporosis and bone health. For example, Osteoporosis Canada last year updated a guideline to emphasize the need to assess older patients of both sexes for the risk for fractures. But the Canadian Task Force on Preventive Health Care in 2023 came to a conclusion in line with the USPSTF draft.

The Canadian task force recommended against routine screening in men, while adding that clinicians should be alert to changes in health that may indicate the patient has experienced or is at a higher risk for fragility fracture.
 

Risk Factors, Concerns About Tests

The USPSTF said that risk factors associated with fragility fractures are similar in men and women. These include:

• Increasing age
• Low body mass index
• Excessive alcohol intake
• Current smoking
• Chronic corticosteroid use
• History of prior fractures, falls within the past year, cerebrovascular accident, and diabetes
• Hypogonadism

The process of updating the USPSTF recommendations can serve as a chance to expand public awareness about osteoporosis, as many men may not know to raise the question of their fracture risk during medical appointments, Dr. Davis said.

“Clinicians need to be aware of the risk factors and to be able to have conversations with men,” she said.

Dr. Davis also cautioned about the need to be aware of limitations with clinical risk assessment tools. In the draft recommendation statement, the USPSTF noted that some tools and approaches may be less likely to identify Black, Hispanic, and Asian people as high risk, and subsequently, clinicians may be less likely to offer treatment to them compared with White people of the same age, bone mineral density, and clinical risk profile.

Dr. Davis had no relevant financial relationships. Dr. Jain received research funding from the Amgen Foundation.
 

A version of this article appeared on Medscape.com.

An influential US panel may largely reaffirm its current recommendation in favor of screening older women to prevent osteoporotic fractures, while also repeating its call for more research to try to determine whether men would benefit from this kind of routine testing.

The US Preventive Services Task Force (USPSTF) on June 11 released a draft update of its recommendations on osteoporosis screening. The task force will accept comments on the draft through July 8. Federal law gives the USPSTF recommendations extra clout, requiring insurers to cover — without co-pay — services that get top marks “A” or “B” from the task force.

The task force intends to maintain a “B” recommendation on screening of older women, indicating that the evidence gathered to date suggests a moderate net benefit. But the draft includes a shift in the approach to this screening.

The USPSTF proposed saying that it recommends screening for osteoporosis in both women aged 65 years and older and postmenopausal women younger than 65 years who are at an increased risk for an osteoporotic fracture. The current recommendation, finalized in 2018, advises “screening for osteoporosis with bone measurement testing [emphasis added]” for these groups.

The proposed change in language — dropping the phrase “with bone measurement testing” — is intended to expand flexibility for clinicians, Esa Davis, MD, MPH, a member of USPSTF and a professor at the University of Maryland School of Medicine, Baltimore, told this news organization.

kurastomokibridichuwronushutodretrovametrofreslowesuclolajuthushotruswespecishoswovigekagespouekisusovogilispuhaswichakubraclubregumunumiclapritreclitrithawajikofrejocreshivekiuopacosletacruprocruclijephibicriclagogabospetok

cutedostochastovavukowrawilacrocluhuresavemeswediwretiniwroclolonoswutresliwot

Insufficient Evidence

The USPSTF’s “I” grade is different from a “D” grade, which is what the task force uses to recommend against the use of a service.

A “D” grade means the USPSTF says there is moderate or high certainty that the service has no net benefit or that the harms outweigh the benefits. (The USPSTF makes it easy to search online for grades given to preventive services, including those that got a “D.”)

The USPSTF is calling for more studies on the benefits and harms of screening for osteoporosis to prevent fractures and related morbidity and mortality in men.

“Men do get osteoporosis,” Dr. Davis said. “But unfortunately, the evidence isn’t there” to allow USPSTF to make a recommendation on screening approaches.

“Any man who has concerns about bone health should certainly talk to his clinician and figure out what is the best form of screening” he might want to do, she said.

There’s been a growing interest in the question of whether to screen men for osteoporosis and bone health. For example, Osteoporosis Canada last year updated a guideline to emphasize the need to assess older patients of both sexes for the risk for fractures. But the Canadian Task Force on Preventive Health Care in 2023 came to a conclusion in line with the USPSTF draft.

The Canadian task force recommended against routine screening in men, while adding that clinicians should be alert to changes in health that may indicate the patient has experienced or is at a higher risk for fragility fracture.
 

Risk Factors, Concerns About Tests

The USPSTF said that risk factors associated with fragility fractures are similar in men and women. These include:

• Increasing age
• Low body mass index
• Excessive alcohol intake
• Current smoking
• Chronic corticosteroid use
• History of prior fractures, falls within the past year, cerebrovascular accident, and diabetes
• Hypogonadism

The process of updating the USPSTF recommendations can serve as a chance to expand public awareness about osteoporosis, as many men may not know to raise the question of their fracture risk during medical appointments, Dr. Davis said.

“Clinicians need to be aware of the risk factors and to be able to have conversations with men,” she said.

Dr. Davis also cautioned about the need to be aware of limitations with clinical risk assessment tools. In the draft recommendation statement, the USPSTF noted that some tools and approaches may be less likely to identify Black, Hispanic, and Asian people as high risk, and subsequently, clinicians may be less likely to offer treatment to them compared with White people of the same age, bone mineral density, and clinical risk profile.

Dr. Davis had no relevant financial relationships. Dr. Jain received research funding from the Amgen Foundation.
 

A version of this article appeared on Medscape.com.

An influential US panel may largely reaffirm its current recommendation in favor of screening older women to prevent osteoporotic fractures, while also repeating its call for more research to try to determine whether men would benefit from this kind of routine testing.

The US Preventive Services Task Force (USPSTF) on June 11 released a draft update of its recommendations on osteoporosis screening. The task force will accept comments on the draft through July 8. Federal law gives the USPSTF recommendations extra clout, requiring insurers to cover — without co-pay — services that get top marks “A” or “B” from the task force.

The task force intends to maintain a “B” recommendation on screening of older women, indicating that the evidence gathered to date suggests a moderate net benefit. But the draft includes a shift in the approach to this screening.

The USPSTF proposed saying that it recommends screening for osteoporosis in both women aged 65 years and older and postmenopausal women younger than 65 years who are at an increased risk for an osteoporotic fracture. The current recommendation, finalized in 2018, advises “screening for osteoporosis with bone measurement testing [emphasis added]” for these groups.

The proposed change in language — dropping the phrase “with bone measurement testing” — is intended to expand flexibility for clinicians, Esa Davis, MD, MPH, a member of USPSTF and a professor at the University of Maryland School of Medicine, Baltimore, told this news organization.

kurastomokibridichuwronushutodretrovametrofreslowesuclolajuthushotruswespecishoswovigekagespouekisusovogilispuhaswichakubraclubregumunumiclapritreclitrithawajikofrejocreshivekiuopacosletacruprocruclijephibicriclagogabospetok

cutedostochastovavukowrawilacrocluhuresavemeswediwretiniwroclolonoswutresliwot

Insufficient Evidence

The USPSTF’s “I” grade is different from a “D” grade, which is what the task force uses to recommend against the use of a service.

A “D” grade means the USPSTF says there is moderate or high certainty that the service has no net benefit or that the harms outweigh the benefits. (The USPSTF makes it easy to search online for grades given to preventive services, including those that got a “D.”)

The USPSTF is calling for more studies on the benefits and harms of screening for osteoporosis to prevent fractures and related morbidity and mortality in men.

“Men do get osteoporosis,” Dr. Davis said. “But unfortunately, the evidence isn’t there” to allow USPSTF to make a recommendation on screening approaches.

“Any man who has concerns about bone health should certainly talk to his clinician and figure out what is the best form of screening” he might want to do, she said.

There’s been a growing interest in the question of whether to screen men for osteoporosis and bone health. For example, Osteoporosis Canada last year updated a guideline to emphasize the need to assess older patients of both sexes for the risk for fractures. But the Canadian Task Force on Preventive Health Care in 2023 came to a conclusion in line with the USPSTF draft.

The Canadian task force recommended against routine screening in men, while adding that clinicians should be alert to changes in health that may indicate the patient has experienced or is at a higher risk for fragility fracture.
 

Risk Factors, Concerns About Tests

The USPSTF said that risk factors associated with fragility fractures are similar in men and women. These include:

• Increasing age
• Low body mass index
• Excessive alcohol intake
• Current smoking
• Chronic corticosteroid use
• History of prior fractures, falls within the past year, cerebrovascular accident, and diabetes
• Hypogonadism

The process of updating the USPSTF recommendations can serve as a chance to expand public awareness about osteoporosis, as many men may not know to raise the question of their fracture risk during medical appointments, Dr. Davis said.

“Clinicians need to be aware of the risk factors and to be able to have conversations with men,” she said.

Dr. Davis also cautioned about the need to be aware of limitations with clinical risk assessment tools. In the draft recommendation statement, the USPSTF noted that some tools and approaches may be less likely to identify Black, Hispanic, and Asian people as high risk, and subsequently, clinicians may be less likely to offer treatment to them compared with White people of the same age, bone mineral density, and clinical risk profile.

Dr. Davis had no relevant financial relationships. Dr. Jain received research funding from the Amgen Foundation.
 

A version of this article appeared on Medscape.com.

Many adults are complacent about vaccinations, believing that annual COVID and flu shots aside, they had all the immunizations they need as children and teens. But adults need vaccines as well, especially if they have missed earlier doses. And older and health-compromised adults, in particular, can benefit from newer vaccines that were not part of the childhood schedule.

“The question is whether adults had the vaccinations they need in the first place,” Sandra Adamson Fryhofer, MD, an internist in Atlanta and the American Medical Association’s liaison to the Advisory Committee on Immunization Practices (ACIP) of the Centers for Disease Control and Prevention, said in an interview. “Many do not even have reliable records of vaccination.”

drisadrikadiradaslilivavabophothiduchimehispuhovadekibusiuiuachochocropacrodethoroviprejebrukestipruuochecejaphecromoshaspesta

Primary care physicians are ideally positioned to get adult patients to update their vaccination status on older vaccines and obtain newer ones as needed. “ACIP recommendations for adult vaccines are getting longer and more complicated and the way they’re administered is more complex, too, in that they’re not all given in the primary care office but sometimes in pharmacies,” Dr. Fryhofer said.

Not all adult patients want to update their vaccinations. “Vaccine hesitancy among many adults is accelerated by the several new vaccines that have been recommended in recent years,” Lauren Block, MD, MPH, an internist at Northwell Health and assistant professor in the Institute of Health System Science at the Feinstein Institutes for Medical Research in metropolitan New York City, said in an interview.

Physicians are rightly concerned about the lagging rates of adult vaccination, Dr. Block said. “Given the prevalence of conditions like pneumonia and shingles and the morbidity associated with them, healthcare providers should take every opportunity to discuss vaccination with patients, from well visits to hospital visits,” Dr. Block added. 

clushaclejuueshiprototiminiluspufrichoprathespeswuthocehilispapholufroswomadrichamuuoredipethesecacrastelospocrouemisogaslabrochuphasichasweshavojukibrovowrophojibebrecrorethelabemaswishanabuswatiphiwafruuaslesathudrocuchobostevechetreshidrures

She pointed to several obstacles to broader uptake, including product shortages, financial barriers, and, increasingly, the negative vocal messaging from media outlets and social media.
 

Current Recommendations

The main vaccines recommended for adults, besides flu and COVID shots, are for respiratory syncytial virus (RVS); shingles; pneumococcal disease; measles, mumps, and rubella (MMR); and tetanus, diphtheria, and pertussis (Tdap). Less commonly, booster vaccines for MM, and hepatitis are recommended when titers are proven to be low.

ACIP’s updated 2024 Adult Immunization Schedule can be downloaded from the website of the CDC.

The newest additions to the schedule include RSV vaccines, the mpox vaccine (Jynneos), a new MenACWY-MenB combo vaccine (Penbraya), and the new 2023-2024 formulation of updated COVID vaccines (both mRNA and protein-based adjuvanted versions).
 

1. Respiratory Syncytial Virus Vaccines

There are two licensed RSV vaccines, Arexvy and Abrysvo. The CDC schedule recommends a single-dose RSV vaccine for adults age 60 years and older, especially those at high risk of contracting the virus — but after shared decision-making based on a discussion of the risk-harm balance since this vaccine carries a small increased chance of developing the neurological symptoms of Guillain-Barré syndrome.

Chronic health conditions associated with a higher risk of severe RVS include cardiopulmonary disease, diabetes, and kidney, liver, and hematologic disorders, as well as compromised immunity, older age, and frailty.
 

2. Shingles Vaccines

This painful disease carries the potential complication of postherpetic neuralgia (PHN), which leads to long-term nerve pain in 10%-18% of patients, especially those over age 40. ACIP recommends two doses of the recombinant zoster vaccine (Shingrix) for individuals 50 years and older. Those 19 years and older with weakened immune systems due to disease or medical treatments should get two doses of the recombinant vaccine, as they have a higher risk of getting shingles and its complications, including neurological problems and skin and eye infections.

3 Pneumococcal Vaccines

There are three approved pneumococcal vaccines: PCV15 (Vaxneuvance), PCV20 (Prevnar20), and PPSV23 (Pneumovax23).

“The pneumococcal vaccine schedule is the most complicated one as higher-valent products continue to become available,” Dr. Fryhofer said.

The two types are pneumococcal conjugate vaccines (PCVs, specifically PCV15 and PCV20) and the pneumococcal polysaccharide vaccine (PPSV23). “While PPSV23 covers 23 strains, it doesn’t give the long-term immunity of the conjugate vaccine,” said Dr. Fryhofer. “A patient may have completed their vaccination with the polysaccharide vaccine but 5 years out may no longer be protected. So we offer the option of getting a dose of PCV20 to round out the protection and confer greater immune memory.”

The ACIP schedule recommends immunization against the Streptococcus pneumoniae pathogen for all older and all at-risk adults. Routine administration of PCV15 or PCV20 is advised for those 65 years or older who have never received any pneumococcal conjugate vaccine or whose previous vaccination history is unknown. If PCV15 is used, it should be followed by PPSV23. Those 65 years or older should get PPSV23 even if they already had one or more doses of pneumococcal vaccine before turning 65.

Further vaccination is recommended for younger at-risk adults aged 19-64 years who have received both PCV13 and PPSV23 but have incomplete vaccination status. These individuals are advised to complete their pneumococcal series by receiving either a single dose of PCV20 at an interval of at least 5 years after the last pneumococcal vaccine dose or more than one dose of PPSV23.

See Pneumococcal Vaccination: Summary of Who and When to Vaccinate for CDC guidance on vaccination options for adults who have previously received a pneumococcal conjugate vaccine. Or, to sort out quickly who gets what and when based on their age, concurrent conditions, and vaccination history, the CDC offers a type-in app called the PneumoRecs VaxAdvisor.
 

4. Measles, Mumps, and Rubella, and Varicella Vaccines

The two approved MMR vaccines are M-M-R II and PRIORIX. A third vaccine, ProQuad, adds varicella.

Adults lacking presumptive evidence of immunity should get at least one dose of the MMR combination vaccine.

Those born before 1957 are deemed to be immune, Dr. Fryhofer noted.

Two doses are recommended for adults entering high-risk settings for measles or mumps transmission such as healthcare personnel, students away at college, and international travelers. The two doses should be separated by at least 28 days. It’s no secret that measles, though preventable, is making a comeback, with 146 reported cases (48 in adults) across 21 states as of May 31 — most linked to international travel.

Women who plan to get pregnant should be vaccinated before but not during each pregnancy. (The vaccine is safe during lactation.) And those of childbearing age with no presumptive evidence of immunity are advised to get at least one dose of the MMR vaccine.
 

5. Tetanus, Diphtheria, and Pertussis Vaccine

Adults with no previous Tdap vaccination should receive a single dose of Adacel or Boostrix followed by a booster every 10 years. Boostrix is recommended for adults over 64 years.

During every pregnancy, women should have a single dose of Tdap, preferably in gestational weeks 27 through 36.

As to the immediate postpartum period, Tdap is recommended only for mothers who did not receive it during their current pregnancy and never received a prior dose. If a woman did not receive Tdap during her current pregnancy but did receive a prior dose of Tdap, she does not need Tdap postpartum.
 

The Challenges

According to Dr. Fryhofer, widespread disinformation about the risks of immunization against vaccine-preventable diseases has brought us to a flashpoint. “It’s now more important than ever to keep telling patients that vaccination is one of the most effective tools for preventing individual illness and protecting public health.”

She recommends that doctors follow the National Institutes of Health’s AIMS method to broach the subject of adult vaccination and increase participation in an inquiring, reassuring, and low-pressure way. Standing for Announce, Inquire, Mirror, and Secure, AIMS structures a nonjudgmental, patient-friendly conversation around immunization to elicit and acknowledge the reasons for hesitancy while explaining the safety and efficacy of vaccines.

Dr. Fryhofer frequently uses AIMS to bring inoculation-averse patients around. “Keep the conversation open with reluctant patients but leave them where they are. They need to see you as a reliable source and nonjudgmental source of information,” she said.

Dr. Block recommends outlining the diseases that have been eliminated through vaccines, from polio to measles, as well as the dangers of vaccine refusal, as indicated by recent outbreaks of vaccine-preventable diseases in areas with low immunization rates. “This approach highlights the opportunity we all have to get vaccinated to protect ourselves and our communities,”  she said.

In Dr. Fryhofer’s view, the situation is urgent and doctors need to be proactive. “We’re now at a public-health tipping point where we may see a sliding back and a reversing of many years of progress.”

Dr. Fryhofer and Dr. Block disclosed no competing interests relevant to their comments.

Many adults are complacent about vaccinations, believing that annual COVID and flu shots aside, they had all the immunizations they need as children and teens. But adults need vaccines as well, especially if they have missed earlier doses. And older and health-compromised adults, in particular, can benefit from newer vaccines that were not part of the childhood schedule.

“The question is whether adults had the vaccinations they need in the first place,” Sandra Adamson Fryhofer, MD, an internist in Atlanta and the American Medical Association’s liaison to the Advisory Committee on Immunization Practices (ACIP) of the Centers for Disease Control and Prevention, said in an interview. “Many do not even have reliable records of vaccination.”

drisadrikadiradaslilivavabophothiduchimehispuhovadekibusiuiuachochocropacrodethoroviprejebrukestipruuochecejaphecromoshaspesta

Primary care physicians are ideally positioned to get adult patients to update their vaccination status on older vaccines and obtain newer ones as needed. “ACIP recommendations for adult vaccines are getting longer and more complicated and the way they’re administered is more complex, too, in that they’re not all given in the primary care office but sometimes in pharmacies,” Dr. Fryhofer said.

Not all adult patients want to update their vaccinations. “Vaccine hesitancy among many adults is accelerated by the several new vaccines that have been recommended in recent years,” Lauren Block, MD, MPH, an internist at Northwell Health and assistant professor in the Institute of Health System Science at the Feinstein Institutes for Medical Research in metropolitan New York City, said in an interview.

Physicians are rightly concerned about the lagging rates of adult vaccination, Dr. Block said. “Given the prevalence of conditions like pneumonia and shingles and the morbidity associated with them, healthcare providers should take every opportunity to discuss vaccination with patients, from well visits to hospital visits,” Dr. Block added. 

clushaclejuueshiprototiminiluspufrichoprathespeswuthocehilispapholufroswomadrichamuuoredipethesecacrastelospocrouemisogaslabrochuphasichasweshavojukibrovowrophojibebrecrorethelabemaswishanabuswatiphiwafruuaslesathudrocuchobostevechetreshidrures

She pointed to several obstacles to broader uptake, including product shortages, financial barriers, and, increasingly, the negative vocal messaging from media outlets and social media.
 

Current Recommendations

The main vaccines recommended for adults, besides flu and COVID shots, are for respiratory syncytial virus (RVS); shingles; pneumococcal disease; measles, mumps, and rubella (MMR); and tetanus, diphtheria, and pertussis (Tdap). Less commonly, booster vaccines for MM, and hepatitis are recommended when titers are proven to be low.

ACIP’s updated 2024 Adult Immunization Schedule can be downloaded from the website of the CDC.

The newest additions to the schedule include RSV vaccines, the mpox vaccine (Jynneos), a new MenACWY-MenB combo vaccine (Penbraya), and the new 2023-2024 formulation of updated COVID vaccines (both mRNA and protein-based adjuvanted versions).
 

1. Respiratory Syncytial Virus Vaccines

There are two licensed RSV vaccines, Arexvy and Abrysvo. The CDC schedule recommends a single-dose RSV vaccine for adults age 60 years and older, especially those at high risk of contracting the virus — but after shared decision-making based on a discussion of the risk-harm balance since this vaccine carries a small increased chance of developing the neurological symptoms of Guillain-Barré syndrome.

Chronic health conditions associated with a higher risk of severe RVS include cardiopulmonary disease, diabetes, and kidney, liver, and hematologic disorders, as well as compromised immunity, older age, and frailty.
 

2. Shingles Vaccines

This painful disease carries the potential complication of postherpetic neuralgia (PHN), which leads to long-term nerve pain in 10%-18% of patients, especially those over age 40. ACIP recommends two doses of the recombinant zoster vaccine (Shingrix) for individuals 50 years and older. Those 19 years and older with weakened immune systems due to disease or medical treatments should get two doses of the recombinant vaccine, as they have a higher risk of getting shingles and its complications, including neurological problems and skin and eye infections.

3 Pneumococcal Vaccines

There are three approved pneumococcal vaccines: PCV15 (Vaxneuvance), PCV20 (Prevnar20), and PPSV23 (Pneumovax23).

“The pneumococcal vaccine schedule is the most complicated one as higher-valent products continue to become available,” Dr. Fryhofer said.

The two types are pneumococcal conjugate vaccines (PCVs, specifically PCV15 and PCV20) and the pneumococcal polysaccharide vaccine (PPSV23). “While PPSV23 covers 23 strains, it doesn’t give the long-term immunity of the conjugate vaccine,” said Dr. Fryhofer. “A patient may have completed their vaccination with the polysaccharide vaccine but 5 years out may no longer be protected. So we offer the option of getting a dose of PCV20 to round out the protection and confer greater immune memory.”

The ACIP schedule recommends immunization against the Streptococcus pneumoniae pathogen for all older and all at-risk adults. Routine administration of PCV15 or PCV20 is advised for those 65 years or older who have never received any pneumococcal conjugate vaccine or whose previous vaccination history is unknown. If PCV15 is used, it should be followed by PPSV23. Those 65 years or older should get PPSV23 even if they already had one or more doses of pneumococcal vaccine before turning 65.

Further vaccination is recommended for younger at-risk adults aged 19-64 years who have received both PCV13 and PPSV23 but have incomplete vaccination status. These individuals are advised to complete their pneumococcal series by receiving either a single dose of PCV20 at an interval of at least 5 years after the last pneumococcal vaccine dose or more than one dose of PPSV23.

See Pneumococcal Vaccination: Summary of Who and When to Vaccinate for CDC guidance on vaccination options for adults who have previously received a pneumococcal conjugate vaccine. Or, to sort out quickly who gets what and when based on their age, concurrent conditions, and vaccination history, the CDC offers a type-in app called the PneumoRecs VaxAdvisor.
 

4. Measles, Mumps, and Rubella, and Varicella Vaccines

The two approved MMR vaccines are M-M-R II and PRIORIX. A third vaccine, ProQuad, adds varicella.

Adults lacking presumptive evidence of immunity should get at least one dose of the MMR combination vaccine.

Those born before 1957 are deemed to be immune, Dr. Fryhofer noted.

Two doses are recommended for adults entering high-risk settings for measles or mumps transmission such as healthcare personnel, students away at college, and international travelers. The two doses should be separated by at least 28 days. It’s no secret that measles, though preventable, is making a comeback, with 146 reported cases (48 in adults) across 21 states as of May 31 — most linked to international travel.

Women who plan to get pregnant should be vaccinated before but not during each pregnancy. (The vaccine is safe during lactation.) And those of childbearing age with no presumptive evidence of immunity are advised to get at least one dose of the MMR vaccine.
 

5. Tetanus, Diphtheria, and Pertussis Vaccine

Adults with no previous Tdap vaccination should receive a single dose of Adacel or Boostrix followed by a booster every 10 years. Boostrix is recommended for adults over 64 years.

During every pregnancy, women should have a single dose of Tdap, preferably in gestational weeks 27 through 36.

As to the immediate postpartum period, Tdap is recommended only for mothers who did not receive it during their current pregnancy and never received a prior dose. If a woman did not receive Tdap during her current pregnancy but did receive a prior dose of Tdap, she does not need Tdap postpartum.
 

The Challenges

According to Dr. Fryhofer, widespread disinformation about the risks of immunization against vaccine-preventable diseases has brought us to a flashpoint. “It’s now more important than ever to keep telling patients that vaccination is one of the most effective tools for preventing individual illness and protecting public health.”

She recommends that doctors follow the National Institutes of Health’s AIMS method to broach the subject of adult vaccination and increase participation in an inquiring, reassuring, and low-pressure way. Standing for Announce, Inquire, Mirror, and Secure, AIMS structures a nonjudgmental, patient-friendly conversation around immunization to elicit and acknowledge the reasons for hesitancy while explaining the safety and efficacy of vaccines.

Dr. Fryhofer frequently uses AIMS to bring inoculation-averse patients around. “Keep the conversation open with reluctant patients but leave them where they are. They need to see you as a reliable source and nonjudgmental source of information,” she said.

Dr. Block recommends outlining the diseases that have been eliminated through vaccines, from polio to measles, as well as the dangers of vaccine refusal, as indicated by recent outbreaks of vaccine-preventable diseases in areas with low immunization rates. “This approach highlights the opportunity we all have to get vaccinated to protect ourselves and our communities,”  she said.

In Dr. Fryhofer’s view, the situation is urgent and doctors need to be proactive. “We’re now at a public-health tipping point where we may see a sliding back and a reversing of many years of progress.”

Dr. Fryhofer and Dr. Block disclosed no competing interests relevant to their comments.

Many adults are complacent about vaccinations, believing that annual COVID and flu shots aside, they had all the immunizations they need as children and teens. But adults need vaccines as well, especially if they have missed earlier doses. And older and health-compromised adults, in particular, can benefit from newer vaccines that were not part of the childhood schedule.

“The question is whether adults had the vaccinations they need in the first place,” Sandra Adamson Fryhofer, MD, an internist in Atlanta and the American Medical Association’s liaison to the Advisory Committee on Immunization Practices (ACIP) of the Centers for Disease Control and Prevention, said in an interview. “Many do not even have reliable records of vaccination.”

drisadrikadiradaslilivavabophothiduchimehispuhovadekibusiuiuachochocropacrodethoroviprejebrukestipruuochecejaphecromoshaspesta

Primary care physicians are ideally positioned to get adult patients to update their vaccination status on older vaccines and obtain newer ones as needed. “ACIP recommendations for adult vaccines are getting longer and more complicated and the way they’re administered is more complex, too, in that they’re not all given in the primary care office but sometimes in pharmacies,” Dr. Fryhofer said.

Not all adult patients want to update their vaccinations. “Vaccine hesitancy among many adults is accelerated by the several new vaccines that have been recommended in recent years,” Lauren Block, MD, MPH, an internist at Northwell Health and assistant professor in the Institute of Health System Science at the Feinstein Institutes for Medical Research in metropolitan New York City, said in an interview.

Physicians are rightly concerned about the lagging rates of adult vaccination, Dr. Block said. “Given the prevalence of conditions like pneumonia and shingles and the morbidity associated with them, healthcare providers should take every opportunity to discuss vaccination with patients, from well visits to hospital visits,” Dr. Block added. 

clushaclejuueshiprototiminiluspufrichoprathespeswuthocehilispapholufroswomadrichamuuoredipethesecacrastelospocrouemisogaslabrochuphasichasweshavojukibrovowrophojibebrecrorethelabemaswishanabuswatiphiwafruuaslesathudrocuchobostevechetreshidrures

She pointed to several obstacles to broader uptake, including product shortages, financial barriers, and, increasingly, the negative vocal messaging from media outlets and social media.
 

Current Recommendations

The main vaccines recommended for adults, besides flu and COVID shots, are for respiratory syncytial virus (RVS); shingles; pneumococcal disease; measles, mumps, and rubella (MMR); and tetanus, diphtheria, and pertussis (Tdap). Less commonly, booster vaccines for MM, and hepatitis are recommended when titers are proven to be low.

ACIP’s updated 2024 Adult Immunization Schedule can be downloaded from the website of the CDC.

The newest additions to the schedule include RSV vaccines, the mpox vaccine (Jynneos), a new MenACWY-MenB combo vaccine (Penbraya), and the new 2023-2024 formulation of updated COVID vaccines (both mRNA and protein-based adjuvanted versions).
 

1. Respiratory Syncytial Virus Vaccines

There are two licensed RSV vaccines, Arexvy and Abrysvo. The CDC schedule recommends a single-dose RSV vaccine for adults age 60 years and older, especially those at high risk of contracting the virus — but after shared decision-making based on a discussion of the risk-harm balance since this vaccine carries a small increased chance of developing the neurological symptoms of Guillain-Barré syndrome.

Chronic health conditions associated with a higher risk of severe RVS include cardiopulmonary disease, diabetes, and kidney, liver, and hematologic disorders, as well as compromised immunity, older age, and frailty.
 

2. Shingles Vaccines

This painful disease carries the potential complication of postherpetic neuralgia (PHN), which leads to long-term nerve pain in 10%-18% of patients, especially those over age 40. ACIP recommends two doses of the recombinant zoster vaccine (Shingrix) for individuals 50 years and older. Those 19 years and older with weakened immune systems due to disease or medical treatments should get two doses of the recombinant vaccine, as they have a higher risk of getting shingles and its complications, including neurological problems and skin and eye infections.

3 Pneumococcal Vaccines

There are three approved pneumococcal vaccines: PCV15 (Vaxneuvance), PCV20 (Prevnar20), and PPSV23 (Pneumovax23).

“The pneumococcal vaccine schedule is the most complicated one as higher-valent products continue to become available,” Dr. Fryhofer said.

The two types are pneumococcal conjugate vaccines (PCVs, specifically PCV15 and PCV20) and the pneumococcal polysaccharide vaccine (PPSV23). “While PPSV23 covers 23 strains, it doesn’t give the long-term immunity of the conjugate vaccine,” said Dr. Fryhofer. “A patient may have completed their vaccination with the polysaccharide vaccine but 5 years out may no longer be protected. So we offer the option of getting a dose of PCV20 to round out the protection and confer greater immune memory.”

The ACIP schedule recommends immunization against the Streptococcus pneumoniae pathogen for all older and all at-risk adults. Routine administration of PCV15 or PCV20 is advised for those 65 years or older who have never received any pneumococcal conjugate vaccine or whose previous vaccination history is unknown. If PCV15 is used, it should be followed by PPSV23. Those 65 years or older should get PPSV23 even if they already had one or more doses of pneumococcal vaccine before turning 65.

Further vaccination is recommended for younger at-risk adults aged 19-64 years who have received both PCV13 and PPSV23 but have incomplete vaccination status. These individuals are advised to complete their pneumococcal series by receiving either a single dose of PCV20 at an interval of at least 5 years after the last pneumococcal vaccine dose or more than one dose of PPSV23.

See Pneumococcal Vaccination: Summary of Who and When to Vaccinate for CDC guidance on vaccination options for adults who have previously received a pneumococcal conjugate vaccine. Or, to sort out quickly who gets what and when based on their age, concurrent conditions, and vaccination history, the CDC offers a type-in app called the PneumoRecs VaxAdvisor.
 

4. Measles, Mumps, and Rubella, and Varicella Vaccines

The two approved MMR vaccines are M-M-R II and PRIORIX. A third vaccine, ProQuad, adds varicella.

Adults lacking presumptive evidence of immunity should get at least one dose of the MMR combination vaccine.

Those born before 1957 are deemed to be immune, Dr. Fryhofer noted.

Two doses are recommended for adults entering high-risk settings for measles or mumps transmission such as healthcare personnel, students away at college, and international travelers. The two doses should be separated by at least 28 days. It’s no secret that measles, though preventable, is making a comeback, with 146 reported cases (48 in adults) across 21 states as of May 31 — most linked to international travel.

Women who plan to get pregnant should be vaccinated before but not during each pregnancy. (The vaccine is safe during lactation.) And those of childbearing age with no presumptive evidence of immunity are advised to get at least one dose of the MMR vaccine.
 

5. Tetanus, Diphtheria, and Pertussis Vaccine

Adults with no previous Tdap vaccination should receive a single dose of Adacel or Boostrix followed by a booster every 10 years. Boostrix is recommended for adults over 64 years.

During every pregnancy, women should have a single dose of Tdap, preferably in gestational weeks 27 through 36.

As to the immediate postpartum period, Tdap is recommended only for mothers who did not receive it during their current pregnancy and never received a prior dose. If a woman did not receive Tdap during her current pregnancy but did receive a prior dose of Tdap, she does not need Tdap postpartum.
 

The Challenges

According to Dr. Fryhofer, widespread disinformation about the risks of immunization against vaccine-preventable diseases has brought us to a flashpoint. “It’s now more important than ever to keep telling patients that vaccination is one of the most effective tools for preventing individual illness and protecting public health.”

She recommends that doctors follow the National Institutes of Health’s AIMS method to broach the subject of adult vaccination and increase participation in an inquiring, reassuring, and low-pressure way. Standing for Announce, Inquire, Mirror, and Secure, AIMS structures a nonjudgmental, patient-friendly conversation around immunization to elicit and acknowledge the reasons for hesitancy while explaining the safety and efficacy of vaccines.

Dr. Fryhofer frequently uses AIMS to bring inoculation-averse patients around. “Keep the conversation open with reluctant patients but leave them where they are. They need to see you as a reliable source and nonjudgmental source of information,” she said.

Dr. Block recommends outlining the diseases that have been eliminated through vaccines, from polio to measles, as well as the dangers of vaccine refusal, as indicated by recent outbreaks of vaccine-preventable diseases in areas with low immunization rates. “This approach highlights the opportunity we all have to get vaccinated to protect ourselves and our communities,”  she said.

In Dr. Fryhofer’s view, the situation is urgent and doctors need to be proactive. “We’re now at a public-health tipping point where we may see a sliding back and a reversing of many years of progress.”

Dr. Fryhofer and Dr. Block disclosed no competing interests relevant to their comments.

BOSTON — New Endocrine Society guidelines call for limiting vitamin D supplementation beyond the daily recommended intake to specific risk groups and advises against routine 25-hydroxyvitamin D [25(OH)D] testing in healthy individuals. 

The evidence-based document was presented on June 3, 2024, at the Endocrine Society annual meeting, and simultaneously published in The Journal of Clinical Endocrinology and Metabolism. It advises that people who may benefit from vitamin D supplementation include: 

• Children aged 1-18 years to prevent rickets and to potentially lower the risk for respiratory tract infections
• Pregnant people to lower the risk for maternal and fetal or neonatal complications
• Adults older than 75 years to lower the risk for mortality
• Adults with prediabetes to lower the risk for type 2 diabetes

In those groups, the recommendation is for daily (rather than intermittent) empiric vitamin D supplementation of more than what was recommended in 2011 by the National Academy of Medicine (NAM), which was then called the Institute of Medicine (IOM): 600 IU/d for those aged 1-70 years and 800 IU/d for those older than 70 years. The document acknowledges that the optimal dose for these populations isn’t known, but it provides the dose ranges that were used in the trials cited as evidence for the recommendations. 

In contrast, the document advises against more vitamin D than the recommended daily intake for most healthier adults younger than 75 years and recommends against testing for blood vitamin D levels in the general population, including those with obesity or darker complexions. 

Guideline author Anastassios G. Pittas, MD, professor of medicine at Tufts University School of Medicine, Boston, told this news organization, “this guideline refers to people who are otherwise healthy, and there’s no clear indication for vitamin D, such as people with already established osteoporosis. This guideline is not relevant to them.”

Dr. Pittas also noted, “there’s no single question and single answer about the role of vitamin D in health and disease, which is what people often want to know. There are many questions, and we cannot answer all of them.”

Panel Chair Marie B. Demay, MD, professor of medicine at Harvard Medical School, Boston, told this news organization that indeed the panel was limited by lack of randomized clinical trial evidence to answer many important questions. “There is a paucity of data regarding definition of optimal levels and optimal intake of vitamin D for preventing specific diseases ... What we really need are large scale clinical trials and biomarkers so we can predict disease outcome before it happens.”

Overall, Dr. Demay said, “The recommendations are that populations adhere to the [NAM/IOM] dietary recommended intakes, and there are certain populations that will likely benefit from levels of intake above [those].” 

Asked to comment, session moderator Clifford J. Rosen, MD, director of Clinical and Translational Research and senior scientist at Maine Medical Center Research Institute, Scarborough, Maine, noted that screening for vitamin D is quite common in clinical practice, but the recommendation against doing so makes sense. 

“When clinicians measure vitamin D, then they’re forced to make a decision what to do about it. That’s where questions about the levels come in. And that’s a big problem. So what the panel’s saying is, don’t screen ... This really gets to the heart of the issue, because we have no data that there’s anything about screening that allows us to improve quality of life ... Screening is probably not worthwhile in any age group.”

Dr. Rosen, who was an author on the 2011 NAM/IOM dietary reference intakes, said that since then, new data have come out regarding the role of vitamin D in mortality in people older than 75 years, benefit in children with regard to respiratory illness, and the potential benefit of vitamin D in pregnancy. “Otherwise, I think we’re going over a lot of the same stuff that we’ve talked about since I was on the IOM panel 15 years ago ... But I think the level of evidence and rigor with which they did it is really impressive.”

However, Simeon I. Taylor, MD, professor of medicine at the University of Maryland, Baltimore, expressed disappointment that the document was limited to healthy people. “Although acknowledging challenges in managing vitamin D status in patients with several diseases, [such as] chronic kidney disease or inflammatory bowel disease, the new guidelines do not provide sufficient guidance for practicing physicians about how to manage these complex patients.”

In addition, Dr. Taylor said that the guidelines “do not explicitly consider the literature suggesting that alternative testing strategies may provide more relevant insights into vitamin D status. Just as variation in levels of thyroid-binding globulin have convinced endocrinologists not to rely on measurement of total thyroxine; interindividual variation in levels of vitamin D binding protein must be accounted for to interpret measurements of total levels of 25(OH)D. It would have been useful to explicitly consider the possible value of measuring vitamin D binding protein-independent indices of vitamin D status.”

Dr. Taylor also raised the same point as an audience member did during the Q&A period regarding patients with osteoporosis or osteopenia. “The value and utility of the new guidelines would be greatly strengthened by providing guidance for how to approach this important and very large group of individuals.”

Dr. Taylor did say that the document has “several strengths, including the fact that they acknowledge the major limitations of the quality of relevant evidence derived from clinical trials.” 

In an accompanying commentary, the guideline authors delve into the issues of skin pigmentation and race as they pertain to vitamin D metabolism, writing: 

The panel discovered that no randomized clinical trials have directly assessed vitamin D related patient-important outcomes based on participants’ skin pigmentation, although race and ethnicity often served as presumed proxies for skin pigmentation in the literature. In their deliberations, guideline panel members and selected Endocrine Society leaders underscored the critical need to distinguish between skin pigmentation as a biological variable and race and ethnicity as socially determined constructs. This differentiation is vital to maximize scientific rigor and, thus, the validity of resulting recommendations.

Dr. Pittas and Dr. Demay have no disclosures relevant to this clinical practice guideline. Dr. Rosen has no disclosures. Dr. Taylor serves as a consultant for Ionis Pharmaceuticals.
 

A version of this article appeared on Medscape.com.

BOSTON — New Endocrine Society guidelines call for limiting vitamin D supplementation beyond the daily recommended intake to specific risk groups and advises against routine 25-hydroxyvitamin D [25(OH)D] testing in healthy individuals. 

The evidence-based document was presented on June 3, 2024, at the Endocrine Society annual meeting, and simultaneously published in The Journal of Clinical Endocrinology and Metabolism. It advises that people who may benefit from vitamin D supplementation include: 

• Children aged 1-18 years to prevent rickets and to potentially lower the risk for respiratory tract infections
• Pregnant people to lower the risk for maternal and fetal or neonatal complications
• Adults older than 75 years to lower the risk for mortality
• Adults with prediabetes to lower the risk for type 2 diabetes

In those groups, the recommendation is for daily (rather than intermittent) empiric vitamin D supplementation of more than what was recommended in 2011 by the National Academy of Medicine (NAM), which was then called the Institute of Medicine (IOM): 600 IU/d for those aged 1-70 years and 800 IU/d for those older than 70 years. The document acknowledges that the optimal dose for these populations isn’t known, but it provides the dose ranges that were used in the trials cited as evidence for the recommendations. 

In contrast, the document advises against more vitamin D than the recommended daily intake for most healthier adults younger than 75 years and recommends against testing for blood vitamin D levels in the general population, including those with obesity or darker complexions. 

Guideline author Anastassios G. Pittas, MD, professor of medicine at Tufts University School of Medicine, Boston, told this news organization, “this guideline refers to people who are otherwise healthy, and there’s no clear indication for vitamin D, such as people with already established osteoporosis. This guideline is not relevant to them.”

Dr. Pittas also noted, “there’s no single question and single answer about the role of vitamin D in health and disease, which is what people often want to know. There are many questions, and we cannot answer all of them.”

Panel Chair Marie B. Demay, MD, professor of medicine at Harvard Medical School, Boston, told this news organization that indeed the panel was limited by lack of randomized clinical trial evidence to answer many important questions. “There is a paucity of data regarding definition of optimal levels and optimal intake of vitamin D for preventing specific diseases ... What we really need are large scale clinical trials and biomarkers so we can predict disease outcome before it happens.”

Overall, Dr. Demay said, “The recommendations are that populations adhere to the [NAM/IOM] dietary recommended intakes, and there are certain populations that will likely benefit from levels of intake above [those].” 

Asked to comment, session moderator Clifford J. Rosen, MD, director of Clinical and Translational Research and senior scientist at Maine Medical Center Research Institute, Scarborough, Maine, noted that screening for vitamin D is quite common in clinical practice, but the recommendation against doing so makes sense. 

“When clinicians measure vitamin D, then they’re forced to make a decision what to do about it. That’s where questions about the levels come in. And that’s a big problem. So what the panel’s saying is, don’t screen ... This really gets to the heart of the issue, because we have no data that there’s anything about screening that allows us to improve quality of life ... Screening is probably not worthwhile in any age group.”

Dr. Rosen, who was an author on the 2011 NAM/IOM dietary reference intakes, said that since then, new data have come out regarding the role of vitamin D in mortality in people older than 75 years, benefit in children with regard to respiratory illness, and the potential benefit of vitamin D in pregnancy. “Otherwise, I think we’re going over a lot of the same stuff that we’ve talked about since I was on the IOM panel 15 years ago ... But I think the level of evidence and rigor with which they did it is really impressive.”

However, Simeon I. Taylor, MD, professor of medicine at the University of Maryland, Baltimore, expressed disappointment that the document was limited to healthy people. “Although acknowledging challenges in managing vitamin D status in patients with several diseases, [such as] chronic kidney disease or inflammatory bowel disease, the new guidelines do not provide sufficient guidance for practicing physicians about how to manage these complex patients.”

In addition, Dr. Taylor said that the guidelines “do not explicitly consider the literature suggesting that alternative testing strategies may provide more relevant insights into vitamin D status. Just as variation in levels of thyroid-binding globulin have convinced endocrinologists not to rely on measurement of total thyroxine; interindividual variation in levels of vitamin D binding protein must be accounted for to interpret measurements of total levels of 25(OH)D. It would have been useful to explicitly consider the possible value of measuring vitamin D binding protein-independent indices of vitamin D status.”

Dr. Taylor also raised the same point as an audience member did during the Q&A period regarding patients with osteoporosis or osteopenia. “The value and utility of the new guidelines would be greatly strengthened by providing guidance for how to approach this important and very large group of individuals.”

Dr. Taylor did say that the document has “several strengths, including the fact that they acknowledge the major limitations of the quality of relevant evidence derived from clinical trials.” 

In an accompanying commentary, the guideline authors delve into the issues of skin pigmentation and race as they pertain to vitamin D metabolism, writing: 

The panel discovered that no randomized clinical trials have directly assessed vitamin D related patient-important outcomes based on participants’ skin pigmentation, although race and ethnicity often served as presumed proxies for skin pigmentation in the literature. In their deliberations, guideline panel members and selected Endocrine Society leaders underscored the critical need to distinguish between skin pigmentation as a biological variable and race and ethnicity as socially determined constructs. This differentiation is vital to maximize scientific rigor and, thus, the validity of resulting recommendations.

Dr. Pittas and Dr. Demay have no disclosures relevant to this clinical practice guideline. Dr. Rosen has no disclosures. Dr. Taylor serves as a consultant for Ionis Pharmaceuticals.
 

A version of this article appeared on Medscape.com.

BOSTON — New Endocrine Society guidelines call for limiting vitamin D supplementation beyond the daily recommended intake to specific risk groups and advises against routine 25-hydroxyvitamin D [25(OH)D] testing in healthy individuals. 

The evidence-based document was presented on June 3, 2024, at the Endocrine Society annual meeting, and simultaneously published in The Journal of Clinical Endocrinology and Metabolism. It advises that people who may benefit from vitamin D supplementation include: 

• Children aged 1-18 years to prevent rickets and to potentially lower the risk for respiratory tract infections
• Pregnant people to lower the risk for maternal and fetal or neonatal complications
• Adults older than 75 years to lower the risk for mortality
• Adults with prediabetes to lower the risk for type 2 diabetes

In those groups, the recommendation is for daily (rather than intermittent) empiric vitamin D supplementation of more than what was recommended in 2011 by the National Academy of Medicine (NAM), which was then called the Institute of Medicine (IOM): 600 IU/d for those aged 1-70 years and 800 IU/d for those older than 70 years. The document acknowledges that the optimal dose for these populations isn’t known, but it provides the dose ranges that were used in the trials cited as evidence for the recommendations. 

In contrast, the document advises against more vitamin D than the recommended daily intake for most healthier adults younger than 75 years and recommends against testing for blood vitamin D levels in the general population, including those with obesity or darker complexions. 

Guideline author Anastassios G. Pittas, MD, professor of medicine at Tufts University School of Medicine, Boston, told this news organization, “this guideline refers to people who are otherwise healthy, and there’s no clear indication for vitamin D, such as people with already established osteoporosis. This guideline is not relevant to them.”

Dr. Pittas also noted, “there’s no single question and single answer about the role of vitamin D in health and disease, which is what people often want to know. There are many questions, and we cannot answer all of them.”

Panel Chair Marie B. Demay, MD, professor of medicine at Harvard Medical School, Boston, told this news organization that indeed the panel was limited by lack of randomized clinical trial evidence to answer many important questions. “There is a paucity of data regarding definition of optimal levels and optimal intake of vitamin D for preventing specific diseases ... What we really need are large scale clinical trials and biomarkers so we can predict disease outcome before it happens.”

Overall, Dr. Demay said, “The recommendations are that populations adhere to the [NAM/IOM] dietary recommended intakes, and there are certain populations that will likely benefit from levels of intake above [those].” 

Asked to comment, session moderator Clifford J. Rosen, MD, director of Clinical and Translational Research and senior scientist at Maine Medical Center Research Institute, Scarborough, Maine, noted that screening for vitamin D is quite common in clinical practice, but the recommendation against doing so makes sense. 

“When clinicians measure vitamin D, then they’re forced to make a decision what to do about it. That’s where questions about the levels come in. And that’s a big problem. So what the panel’s saying is, don’t screen ... This really gets to the heart of the issue, because we have no data that there’s anything about screening that allows us to improve quality of life ... Screening is probably not worthwhile in any age group.”

Dr. Rosen, who was an author on the 2011 NAM/IOM dietary reference intakes, said that since then, new data have come out regarding the role of vitamin D in mortality in people older than 75 years, benefit in children with regard to respiratory illness, and the potential benefit of vitamin D in pregnancy. “Otherwise, I think we’re going over a lot of the same stuff that we’ve talked about since I was on the IOM panel 15 years ago ... But I think the level of evidence and rigor with which they did it is really impressive.”

However, Simeon I. Taylor, MD, professor of medicine at the University of Maryland, Baltimore, expressed disappointment that the document was limited to healthy people. “Although acknowledging challenges in managing vitamin D status in patients with several diseases, [such as] chronic kidney disease or inflammatory bowel disease, the new guidelines do not provide sufficient guidance for practicing physicians about how to manage these complex patients.”

In addition, Dr. Taylor said that the guidelines “do not explicitly consider the literature suggesting that alternative testing strategies may provide more relevant insights into vitamin D status. Just as variation in levels of thyroid-binding globulin have convinced endocrinologists not to rely on measurement of total thyroxine; interindividual variation in levels of vitamin D binding protein must be accounted for to interpret measurements of total levels of 25(OH)D. It would have been useful to explicitly consider the possible value of measuring vitamin D binding protein-independent indices of vitamin D status.”

Dr. Taylor also raised the same point as an audience member did during the Q&A period regarding patients with osteoporosis or osteopenia. “The value and utility of the new guidelines would be greatly strengthened by providing guidance for how to approach this important and very large group of individuals.”

Dr. Taylor did say that the document has “several strengths, including the fact that they acknowledge the major limitations of the quality of relevant evidence derived from clinical trials.” 

In an accompanying commentary, the guideline authors delve into the issues of skin pigmentation and race as they pertain to vitamin D metabolism, writing: 

The panel discovered that no randomized clinical trials have directly assessed vitamin D related patient-important outcomes based on participants’ skin pigmentation, although race and ethnicity often served as presumed proxies for skin pigmentation in the literature. In their deliberations, guideline panel members and selected Endocrine Society leaders underscored the critical need to distinguish between skin pigmentation as a biological variable and race and ethnicity as socially determined constructs. This differentiation is vital to maximize scientific rigor and, thus, the validity of resulting recommendations.

Dr. Pittas and Dr. Demay have no disclosures relevant to this clinical practice guideline. Dr. Rosen has no disclosures. Dr. Taylor serves as a consultant for Ionis Pharmaceuticals.
 

A version of this article appeared on Medscape.com.

New expert guidance to help clinicians manage the treatment of patients with epilepsy during pregnancy has been released.

Issued by the American Academy of Neurology, the American Epilepsy Society, and the Society for Maternal-Fetal Medicine, the new practice guideline covers the use of antiseizure medications (ASMs) and folic acid supplementation before conception and during pregnancy.

“Most children born to people with epilepsy are healthy, but there is a small risk of pregnancy-related problems, partly due to seizures and partly due to the effects of antiseizure medications,” the guidelines’ lead author Alison M. Pack, MD, MPH, professor of neurology and chief of the Epilepsy and Sleep Division, Columbia University, New York City, said in a news release.

“This guideline provides recommendations regarding the effects of antiseizure medications and folic acid supplementation on malformations at birth and the development of children during pregnancy, so that doctors and people with epilepsy can determine which treatments may be best for them,” she added. 

The guideline was published online in Neurology.
 

Why Now? 

The new guideline updates the 2009 guidance on epilepsy management during pregnancy. Since then, Dr. Pack told this news organization, there has been a wealth of new data on differential effects of different ASMs — notably, lamotrigine and levetiracetam — the most commonly prescribed medications in this population.

“In this guideline, we were able to assess differential effects of different ASMs on outcomes of interest, including major congenital malformations [MCMs], perinatal outcomes, and neurodevelopmental outcomes. In addition, we looked at the effect of folic acid supplementation on each of these outcomes,” she said.

The overarching goals of care for patients are to “optimize health outcomes both for individuals and their future offspring,” the authors wrote. Shared decision-making, they add, leads to better decision-making by providing a better understanding of the available treatment options and their potential risks, resulting in enhanced decision-making that aligns with personal values.

Clinicians should recommend ASMs that optimize seizure control and fetal outcomes, in the event of a pregnancy, at the earliest possible preconception time, the guideline authors note.

“Overall, treating clinicians need to balance treating the person with epilepsy to control convulsive seizures (generalized tonic-clonic seizures and focal-to-bilateral tonic-clonic seizures) to minimize potential risks to the birth parent and the possible risks of certain ASMs on the fetus if pregnancy occurs,” they wrote.

If a patient is already pregnant, the experts recommend that clinicians “exercise caution” in removing or replacing an ASM that controls convulsive seizures, even if it’s “not an optimal choice” for the fetus. 

In addition, they advise that ASM levels should be monitored throughout the pregnancy, guided by individual ASM pharmacokinetics and an individual patient’s clinical presentation. ASM dose, they note, should be adjusted during pregnancy in response to decreasing serum ASM levels or worsening seizure control.

The authors point out that there are limited data on “pregnancy-related outcomes with respect to acetazolamide, eslicarbazepine, ethosuximide, lacosamide, nitrazepam, perampanel, piracetam, pregabalin, rufinamide, stiripentol, tiagabine, and vigabatrin.”

Patients should be informed that the birth prevalence of any major congenital malformation in the general population ranges between 2.4% and 2.9%.
 

If Feasible, Avoid Valproic Acid 

“One of the most important take-home messages is that valproic acid has the highest unadjusted birth prevalence of all major congenital malformations — 9.7% — and the highest unadjusted birth prevalence of neural tube defects at 1.4%,” Dr. Pack said. As a result, the guideline authors advise against using valproic acid, if clinically feasible.

Valproic acid also has the highest prevalence of negative neurodevelopmental outcomes, including a reduction in global IQ and an increased prevalence of autism spectrum disorder (ASD). Patients should be counseled accordingly and advised of the increased risk for ASD and decreased IQ resulting from valproic acid.

Clinicians should consider using lamotrigine, levetiracetam, or oxcarbazepine when appropriate. Serum concentrations of most ASMs have a “defined therapeutic window” for effective seizure control and that concentration may decrease during pregnancy, particularly with lamotrigine and levetiracetam, the authors note.

Phenobarbital, topiramate, and valproic acid should because of the increased risk for cardiac malformations, oral clefts, and urogenital and renal malformations.

Fetal screening for major congenital malformations is recommended to enable early detection and timely intervention in patients treated with any ASM during pregnancy Patients receiving phenobarbital during pregnancy should also undergo fetal cardiac screenings.

Valproic acid and topiramate are also associated with children who are small for their gestational age. To enable early identification of fetal growth restriction, patients taking valproic acid or topiramate should be monitored. In addition, children exposed to these medications in utero should be monitored during childhood to ensure they are meeting age-appropriate developmental milestones. 

Folic acid taken during pregnancy can reduce the prevalence of negative neurodevelopment outcomes, but not major congenital malformations, Dr. Pack noted. 

“Due to limited available data, we were unable to define an optimal dose of folic acid supplementation beyond at least 0.4 mg/d,” Dr. Pack said. “Future studies, preferably randomized clinical trials, are needed to better define the optimal dose.”

She emphasized that epilepsy is one of the most common neurologic disorders, and 1 in 5 of those affected are people of childbearing potential. Understanding the effects of ASMs on pregnancy outcomes is critical for physicians who manage these patients.
 

Uncertainty Remains 

Commenting for this news organization, Kimford Meador, MD, a professor in the Department of Neurology and Neurological Sciences at Stanford University School of Medicine , Stanford Neuroscience Health Center, Palo Alto, California, noted that the new guidelines reflect the gains in knowledge since 2009 and that the recommendations are “reasonable, based on available data.”

However, “one very important point is how much remains unknown,” said Dr. Meador, who was not involved in writing the current guideline. “Many ASMs have no data, and several have estimates based on small samples or a single observational study.” Thus, “the risks for the majority of ASMs are uncertain.”

Given that randomized trials “are not possible in this population, and that all observational studies are subject to residual confounding, a reliable signal across multiple studies in humans is required to be certain of findings,” he stated.

This practice guideline was developed with financial support from the American Academy of Neurology. Dr. Pack serves on the editorial board for the journal Epilepsy Currents, receives royalties from UpToDate, receives funding from the National Institutes of Health for serving as coinvestigator and site principal investigator for the Maternal Outcomes and Neurodevelopmental Effects of Antiepileptic Drugs (MONEAD) study, and receives funding from Bayer for serving as a co-investigator on a study on women with epilepsy initiating a progestin intrauterine device. One of Dr. Pack’s immediate family members has received personal compensation for serving as an employee of REGENEXBIO. The other authors’ disclosures are listed on the original paper. Dr. Meador has received research support from the National Institutes of Health, Veterans Administration, Eisai, Inc, and Suno Medtronic Navigation, Inc, and the Epilepsy Study Consortium pays Dr. Meador’s university for his research on the Human Epilepsy Project and consultant time related to Eisai, UCB Pharma, and Xenon.

A version of this article first appeared on Medscape.com.

New expert guidance to help clinicians manage the treatment of patients with epilepsy during pregnancy has been released.

Issued by the American Academy of Neurology, the American Epilepsy Society, and the Society for Maternal-Fetal Medicine, the new practice guideline covers the use of antiseizure medications (ASMs) and folic acid supplementation before conception and during pregnancy.

“Most children born to people with epilepsy are healthy, but there is a small risk of pregnancy-related problems, partly due to seizures and partly due to the effects of antiseizure medications,” the guidelines’ lead author Alison M. Pack, MD, MPH, professor of neurology and chief of the Epilepsy and Sleep Division, Columbia University, New York City, said in a news release.

“This guideline provides recommendations regarding the effects of antiseizure medications and folic acid supplementation on malformations at birth and the development of children during pregnancy, so that doctors and people with epilepsy can determine which treatments may be best for them,” she added. 

The guideline was published online in Neurology.
 

Why Now? 

The new guideline updates the 2009 guidance on epilepsy management during pregnancy. Since then, Dr. Pack told this news organization, there has been a wealth of new data on differential effects of different ASMs — notably, lamotrigine and levetiracetam — the most commonly prescribed medications in this population.

“In this guideline, we were able to assess differential effects of different ASMs on outcomes of interest, including major congenital malformations [MCMs], perinatal outcomes, and neurodevelopmental outcomes. In addition, we looked at the effect of folic acid supplementation on each of these outcomes,” she said.

The overarching goals of care for patients are to “optimize health outcomes both for individuals and their future offspring,” the authors wrote. Shared decision-making, they add, leads to better decision-making by providing a better understanding of the available treatment options and their potential risks, resulting in enhanced decision-making that aligns with personal values.

Clinicians should recommend ASMs that optimize seizure control and fetal outcomes, in the event of a pregnancy, at the earliest possible preconception time, the guideline authors note.

“Overall, treating clinicians need to balance treating the person with epilepsy to control convulsive seizures (generalized tonic-clonic seizures and focal-to-bilateral tonic-clonic seizures) to minimize potential risks to the birth parent and the possible risks of certain ASMs on the fetus if pregnancy occurs,” they wrote.

If a patient is already pregnant, the experts recommend that clinicians “exercise caution” in removing or replacing an ASM that controls convulsive seizures, even if it’s “not an optimal choice” for the fetus. 

In addition, they advise that ASM levels should be monitored throughout the pregnancy, guided by individual ASM pharmacokinetics and an individual patient’s clinical presentation. ASM dose, they note, should be adjusted during pregnancy in response to decreasing serum ASM levels or worsening seizure control.

The authors point out that there are limited data on “pregnancy-related outcomes with respect to acetazolamide, eslicarbazepine, ethosuximide, lacosamide, nitrazepam, perampanel, piracetam, pregabalin, rufinamide, stiripentol, tiagabine, and vigabatrin.”

Patients should be informed that the birth prevalence of any major congenital malformation in the general population ranges between 2.4% and 2.9%.
 

If Feasible, Avoid Valproic Acid 

“One of the most important take-home messages is that valproic acid has the highest unadjusted birth prevalence of all major congenital malformations — 9.7% — and the highest unadjusted birth prevalence of neural tube defects at 1.4%,” Dr. Pack said. As a result, the guideline authors advise against using valproic acid, if clinically feasible.

Valproic acid also has the highest prevalence of negative neurodevelopmental outcomes, including a reduction in global IQ and an increased prevalence of autism spectrum disorder (ASD). Patients should be counseled accordingly and advised of the increased risk for ASD and decreased IQ resulting from valproic acid.

Clinicians should consider using lamotrigine, levetiracetam, or oxcarbazepine when appropriate. Serum concentrations of most ASMs have a “defined therapeutic window” for effective seizure control and that concentration may decrease during pregnancy, particularly with lamotrigine and levetiracetam, the authors note.

Phenobarbital, topiramate, and valproic acid should because of the increased risk for cardiac malformations, oral clefts, and urogenital and renal malformations.

Fetal screening for major congenital malformations is recommended to enable early detection and timely intervention in patients treated with any ASM during pregnancy Patients receiving phenobarbital during pregnancy should also undergo fetal cardiac screenings.

Valproic acid and topiramate are also associated with children who are small for their gestational age. To enable early identification of fetal growth restriction, patients taking valproic acid or topiramate should be monitored. In addition, children exposed to these medications in utero should be monitored during childhood to ensure they are meeting age-appropriate developmental milestones. 

Folic acid taken during pregnancy can reduce the prevalence of negative neurodevelopment outcomes, but not major congenital malformations, Dr. Pack noted. 

“Due to limited available data, we were unable to define an optimal dose of folic acid supplementation beyond at least 0.4 mg/d,” Dr. Pack said. “Future studies, preferably randomized clinical trials, are needed to better define the optimal dose.”

She emphasized that epilepsy is one of the most common neurologic disorders, and 1 in 5 of those affected are people of childbearing potential. Understanding the effects of ASMs on pregnancy outcomes is critical for physicians who manage these patients.
 

Uncertainty Remains 

Commenting for this news organization, Kimford Meador, MD, a professor in the Department of Neurology and Neurological Sciences at Stanford University School of Medicine , Stanford Neuroscience Health Center, Palo Alto, California, noted that the new guidelines reflect the gains in knowledge since 2009 and that the recommendations are “reasonable, based on available data.”

However, “one very important point is how much remains unknown,” said Dr. Meador, who was not involved in writing the current guideline. “Many ASMs have no data, and several have estimates based on small samples or a single observational study.” Thus, “the risks for the majority of ASMs are uncertain.”

Given that randomized trials “are not possible in this population, and that all observational studies are subject to residual confounding, a reliable signal across multiple studies in humans is required to be certain of findings,” he stated.

This practice guideline was developed with financial support from the American Academy of Neurology. Dr. Pack serves on the editorial board for the journal Epilepsy Currents, receives royalties from UpToDate, receives funding from the National Institutes of Health for serving as coinvestigator and site principal investigator for the Maternal Outcomes and Neurodevelopmental Effects of Antiepileptic Drugs (MONEAD) study, and receives funding from Bayer for serving as a co-investigator on a study on women with epilepsy initiating a progestin intrauterine device. One of Dr. Pack’s immediate family members has received personal compensation for serving as an employee of REGENEXBIO. The other authors’ disclosures are listed on the original paper. Dr. Meador has received research support from the National Institutes of Health, Veterans Administration, Eisai, Inc, and Suno Medtronic Navigation, Inc, and the Epilepsy Study Consortium pays Dr. Meador’s university for his research on the Human Epilepsy Project and consultant time related to Eisai, UCB Pharma, and Xenon.

A version of this article first appeared on Medscape.com.

New expert guidance to help clinicians manage the treatment of patients with epilepsy during pregnancy has been released.

Issued by the American Academy of Neurology, the American Epilepsy Society, and the Society for Maternal-Fetal Medicine, the new practice guideline covers the use of antiseizure medications (ASMs) and folic acid supplementation before conception and during pregnancy.

“Most children born to people with epilepsy are healthy, but there is a small risk of pregnancy-related problems, partly due to seizures and partly due to the effects of antiseizure medications,” the guidelines’ lead author Alison M. Pack, MD, MPH, professor of neurology and chief of the Epilepsy and Sleep Division, Columbia University, New York City, said in a news release.

“This guideline provides recommendations regarding the effects of antiseizure medications and folic acid supplementation on malformations at birth and the development of children during pregnancy, so that doctors and people with epilepsy can determine which treatments may be best for them,” she added. 

The guideline was published online in Neurology.
 

Why Now? 

The new guideline updates the 2009 guidance on epilepsy management during pregnancy. Since then, Dr. Pack told this news organization, there has been a wealth of new data on differential effects of different ASMs — notably, lamotrigine and levetiracetam — the most commonly prescribed medications in this population.

“In this guideline, we were able to assess differential effects of different ASMs on outcomes of interest, including major congenital malformations [MCMs], perinatal outcomes, and neurodevelopmental outcomes. In addition, we looked at the effect of folic acid supplementation on each of these outcomes,” she said.

The overarching goals of care for patients are to “optimize health outcomes both for individuals and their future offspring,” the authors wrote. Shared decision-making, they add, leads to better decision-making by providing a better understanding of the available treatment options and their potential risks, resulting in enhanced decision-making that aligns with personal values.

Clinicians should recommend ASMs that optimize seizure control and fetal outcomes, in the event of a pregnancy, at the earliest possible preconception time, the guideline authors note.

“Overall, treating clinicians need to balance treating the person with epilepsy to control convulsive seizures (generalized tonic-clonic seizures and focal-to-bilateral tonic-clonic seizures) to minimize potential risks to the birth parent and the possible risks of certain ASMs on the fetus if pregnancy occurs,” they wrote.

If a patient is already pregnant, the experts recommend that clinicians “exercise caution” in removing or replacing an ASM that controls convulsive seizures, even if it’s “not an optimal choice” for the fetus. 

In addition, they advise that ASM levels should be monitored throughout the pregnancy, guided by individual ASM pharmacokinetics and an individual patient’s clinical presentation. ASM dose, they note, should be adjusted during pregnancy in response to decreasing serum ASM levels or worsening seizure control.

The authors point out that there are limited data on “pregnancy-related outcomes with respect to acetazolamide, eslicarbazepine, ethosuximide, lacosamide, nitrazepam, perampanel, piracetam, pregabalin, rufinamide, stiripentol, tiagabine, and vigabatrin.”

Patients should be informed that the birth prevalence of any major congenital malformation in the general population ranges between 2.4% and 2.9%.
 

If Feasible, Avoid Valproic Acid 

“One of the most important take-home messages is that valproic acid has the highest unadjusted birth prevalence of all major congenital malformations — 9.7% — and the highest unadjusted birth prevalence of neural tube defects at 1.4%,” Dr. Pack said. As a result, the guideline authors advise against using valproic acid, if clinically feasible.

Valproic acid also has the highest prevalence of negative neurodevelopmental outcomes, including a reduction in global IQ and an increased prevalence of autism spectrum disorder (ASD). Patients should be counseled accordingly and advised of the increased risk for ASD and decreased IQ resulting from valproic acid.

Clinicians should consider using lamotrigine, levetiracetam, or oxcarbazepine when appropriate. Serum concentrations of most ASMs have a “defined therapeutic window” for effective seizure control and that concentration may decrease during pregnancy, particularly with lamotrigine and levetiracetam, the authors note.

Phenobarbital, topiramate, and valproic acid should because of the increased risk for cardiac malformations, oral clefts, and urogenital and renal malformations.

Fetal screening for major congenital malformations is recommended to enable early detection and timely intervention in patients treated with any ASM during pregnancy Patients receiving phenobarbital during pregnancy should also undergo fetal cardiac screenings.

Valproic acid and topiramate are also associated with children who are small for their gestational age. To enable early identification of fetal growth restriction, patients taking valproic acid or topiramate should be monitored. In addition, children exposed to these medications in utero should be monitored during childhood to ensure they are meeting age-appropriate developmental milestones. 

Folic acid taken during pregnancy can reduce the prevalence of negative neurodevelopment outcomes, but not major congenital malformations, Dr. Pack noted. 

“Due to limited available data, we were unable to define an optimal dose of folic acid supplementation beyond at least 0.4 mg/d,” Dr. Pack said. “Future studies, preferably randomized clinical trials, are needed to better define the optimal dose.”

She emphasized that epilepsy is one of the most common neurologic disorders, and 1 in 5 of those affected are people of childbearing potential. Understanding the effects of ASMs on pregnancy outcomes is critical for physicians who manage these patients.
 

Uncertainty Remains 

Commenting for this news organization, Kimford Meador, MD, a professor in the Department of Neurology and Neurological Sciences at Stanford University School of Medicine , Stanford Neuroscience Health Center, Palo Alto, California, noted that the new guidelines reflect the gains in knowledge since 2009 and that the recommendations are “reasonable, based on available data.”

However, “one very important point is how much remains unknown,” said Dr. Meador, who was not involved in writing the current guideline. “Many ASMs have no data, and several have estimates based on small samples or a single observational study.” Thus, “the risks for the majority of ASMs are uncertain.”

Given that randomized trials “are not possible in this population, and that all observational studies are subject to residual confounding, a reliable signal across multiple studies in humans is required to be certain of findings,” he stated.

This practice guideline was developed with financial support from the American Academy of Neurology. Dr. Pack serves on the editorial board for the journal Epilepsy Currents, receives royalties from UpToDate, receives funding from the National Institutes of Health for serving as coinvestigator and site principal investigator for the Maternal Outcomes and Neurodevelopmental Effects of Antiepileptic Drugs (MONEAD) study, and receives funding from Bayer for serving as a co-investigator on a study on women with epilepsy initiating a progestin intrauterine device. One of Dr. Pack’s immediate family members has received personal compensation for serving as an employee of REGENEXBIO. The other authors’ disclosures are listed on the original paper. Dr. Meador has received research support from the National Institutes of Health, Veterans Administration, Eisai, Inc, and Suno Medtronic Navigation, Inc, and the Epilepsy Study Consortium pays Dr. Meador’s university for his research on the Human Epilepsy Project and consultant time related to Eisai, UCB Pharma, and Xenon.

A version of this article first appeared on Medscape.com.

New guidelines to manage peripheral neuropathy related to Sjögren disease have been developed by a multidisciplinary team of physicians from across medicine.

The guidelines will provide an evidence-based resource for the assessment, diagnosis, and treatment of various peripheral neuropathies related to the disorder.

Up until now, the field has been “haphazard and chaotic,” lead author George Sarka, MD, DrPH, MPH, director of the CME Committee for MemorialCare, Saddleback Medical Center, Laguna Hills, California, and member of the Sjögren Foundation PNS Guidelines Topic Review Group (TRG), told this news organization.

Dr. Sarka discussed the initiative at the American Academy of Neurology 2024 annual meeting.


 

Severe, Complex Illness

Sjögren disease is the second most common autoimmune rheumatic disorder after rheumatoid arthritis, affecting an estimated 4 million Americans. Women make up most of the patient population at a ratio of 9:1.

The condition typically affects the mucous membranes and moisture-secreting glands of the eyes and mouth, resulting in decreased tears and saliva. But peripheral nervous system (PNS) manifestations often precede these symptoms and can occur in up to 60% of Sjögren disease cases.

“Traditionally, Sjögren’s was looked at as a dry eye and dry mouth disease, but we realize now that it’s so much broader than that,” said Dr. Sarka. “It’s a severe, systemic, and complex illness that can affect any body organ or system, and the nervous system is frequently affected.”

PNS manifestations cause more than mere discomfort; they can lead to diagnostic and management challenges, costly treatments, and diminished quality of life.

Getting a proper diagnosis goes a long way toward improving the quality of life for these patients, Steven Mandel, MD, clinical professor of neurology at the Zucker School of Medicine at Hofstra-Northwell, adjunct clinical professor of medicine at NY Medical College, New York City, and member of the TRG, told this news organization.

The problem is, doctors don’t always think an autoimmune disorder is causing the symptoms, said Dr. Sarka. “There’s an old adage in neurology that if you don’t think about it, you’re going to miss it; you have to ask, and that’s what we’re trying to get people to do.”

The condition often accompanies other immune system disorders such as rheumatoid arthritis and lupus. But as patients are referred back and forth between ophthalmologists, rheumatologists, and neurologists, the condition is often missed. “It could be 4 or 5 years before a definitive diagnosis of Sjögren’s is made,” said Dr. Sarka.

He believes the education system is partly to blame. “Medical schools have been very deficient in teaching people about recognizing Sjögren disease.”

That leaves many physicians at a loss about “what to do with these patients when they walk in the door,” said Dr. Mandel. “They don’t know how to manage them; they don’t know how to diagnose them; and they don’t know how to treat them.”

Developing guidelines with multispecialty collaboration was “absolutely critical” in addressing this knowledge gap, Dr. Mandel added. That process involved “a very rigorous and transparent methodology so that it would be accepted by all the professionals involved in Sjögren’s,” he said.

The process took 3 years and involved amassing and grading the evidence, getting consensus from committee members, developing recommendations, and getting feedback and external review.
 

Scant Evidence

An early literature search revealed very little evidence on PNS manifestations in patients with Sjögren disease, so the guideline committee “leaned very heavily on expert opinion” to develop recommendations, Kathy Hammitt, MA, vice president of Medical and Scientific Affairs, Sjögren’s Foundation, told this news organization.

The literature search also showed different terms are used to describe PNS, “which is where the chaos comes in,” said Dr. Sarka.

Experts from different specialties worked together to define and align nomenclature used by various specialists. They developed definitions for seven PNS categories including mononeuropathy, large fiber neuropathy, small fiber neuropathy, demyelinating polyradiculoneuropathy, ganglionopathy, vasculitis neuropathy, and autoimmune nervous system neuropathy.

The guidelines pertaining to PNS manifestations encompass a spectrum of neurologic abnormalities, including cranial neuropathies (trigeminal neuropathy or acute facial neuropathy), polyneuropathies (large fiber neuropathy, small fiber neuropathy, demyelinating polyradiculoneuropathy, vasculitis neuropathy, or ganglionopathy), and autonomic nervous system (ANS) neuropathies (postural tachycardia, orthostatic hypotension, or autonomic dysfunction).
 

Key Steps

The guidelines address two key steps for each PNS manifestation — the workup and evaluation of patients with suspected ANS manifestation including standard evaluations, diagnostic tests, and treatment. The experts developed 31 best practices for diagnosis and workup and 20 treatment recommendations.

Initial assessment of potential ANS involvement includes asking patients about orthostatic postural lightheadedness and difficulties with digestion, urination, sweating, and sexual function.

Treatment of autoimmune diseases typically focuses on relieving symptoms and can include steroids, the anticonvulsant gabapentin, the monoclonal antibody rituximab, and intravenous immunoglobulin. “The type of neuropathy will mandate or suggest certain therapies over others,” said Dr. Sarka, adding that a patient can have more than one neuropathy.

Therapeutics for Sjögren disease is another example of an area that has been “very haphazard,” he added.

The guidelines are aimed not just at specialists but also at general practitioners who treat many of these patients. But Dr. Hammitt emphasized that neurologists can be “instrumental” in identifying Sjögren disease in patients with PNS symptoms.

“Our hope is that specialists — in this case, neurologists — will recognize the potential for this condition in their PNS patients and ensure referral to a rheumatologist or knowledgeable family practitioner to manage overall care.”

The committee will soon submit its manuscript to the AAN for publication.

“Once published, we will have a robust dissemination strategy to ensure that providers, patients, and policymakers are aware of, and use, this very valuable resource,” said Dr. Hammitt.

No conflicts of interest were reported.

A version of this article appeared on Medscape.com.

New guidelines to manage peripheral neuropathy related to Sjögren disease have been developed by a multidisciplinary team of physicians from across medicine.

The guidelines will provide an evidence-based resource for the assessment, diagnosis, and treatment of various peripheral neuropathies related to the disorder.

Up until now, the field has been “haphazard and chaotic,” lead author George Sarka, MD, DrPH, MPH, director of the CME Committee for MemorialCare, Saddleback Medical Center, Laguna Hills, California, and member of the Sjögren Foundation PNS Guidelines Topic Review Group (TRG), told this news organization.

Dr. Sarka discussed the initiative at the American Academy of Neurology 2024 annual meeting.


 

Severe, Complex Illness

Sjögren disease is the second most common autoimmune rheumatic disorder after rheumatoid arthritis, affecting an estimated 4 million Americans. Women make up most of the patient population at a ratio of 9:1.

The condition typically affects the mucous membranes and moisture-secreting glands of the eyes and mouth, resulting in decreased tears and saliva. But peripheral nervous system (PNS) manifestations often precede these symptoms and can occur in up to 60% of Sjögren disease cases.

“Traditionally, Sjögren’s was looked at as a dry eye and dry mouth disease, but we realize now that it’s so much broader than that,” said Dr. Sarka. “It’s a severe, systemic, and complex illness that can affect any body organ or system, and the nervous system is frequently affected.”

PNS manifestations cause more than mere discomfort; they can lead to diagnostic and management challenges, costly treatments, and diminished quality of life.

Getting a proper diagnosis goes a long way toward improving the quality of life for these patients, Steven Mandel, MD, clinical professor of neurology at the Zucker School of Medicine at Hofstra-Northwell, adjunct clinical professor of medicine at NY Medical College, New York City, and member of the TRG, told this news organization.

The problem is, doctors don’t always think an autoimmune disorder is causing the symptoms, said Dr. Sarka. “There’s an old adage in neurology that if you don’t think about it, you’re going to miss it; you have to ask, and that’s what we’re trying to get people to do.”

The condition often accompanies other immune system disorders such as rheumatoid arthritis and lupus. But as patients are referred back and forth between ophthalmologists, rheumatologists, and neurologists, the condition is often missed. “It could be 4 or 5 years before a definitive diagnosis of Sjögren’s is made,” said Dr. Sarka.

He believes the education system is partly to blame. “Medical schools have been very deficient in teaching people about recognizing Sjögren disease.”

That leaves many physicians at a loss about “what to do with these patients when they walk in the door,” said Dr. Mandel. “They don’t know how to manage them; they don’t know how to diagnose them; and they don’t know how to treat them.”

Developing guidelines with multispecialty collaboration was “absolutely critical” in addressing this knowledge gap, Dr. Mandel added. That process involved “a very rigorous and transparent methodology so that it would be accepted by all the professionals involved in Sjögren’s,” he said.

The process took 3 years and involved amassing and grading the evidence, getting consensus from committee members, developing recommendations, and getting feedback and external review.
 

Scant Evidence

An early literature search revealed very little evidence on PNS manifestations in patients with Sjögren disease, so the guideline committee “leaned very heavily on expert opinion” to develop recommendations, Kathy Hammitt, MA, vice president of Medical and Scientific Affairs, Sjögren’s Foundation, told this news organization.

The literature search also showed different terms are used to describe PNS, “which is where the chaos comes in,” said Dr. Sarka.

Experts from different specialties worked together to define and align nomenclature used by various specialists. They developed definitions for seven PNS categories including mononeuropathy, large fiber neuropathy, small fiber neuropathy, demyelinating polyradiculoneuropathy, ganglionopathy, vasculitis neuropathy, and autoimmune nervous system neuropathy.

The guidelines pertaining to PNS manifestations encompass a spectrum of neurologic abnormalities, including cranial neuropathies (trigeminal neuropathy or acute facial neuropathy), polyneuropathies (large fiber neuropathy, small fiber neuropathy, demyelinating polyradiculoneuropathy, vasculitis neuropathy, or ganglionopathy), and autonomic nervous system (ANS) neuropathies (postural tachycardia, orthostatic hypotension, or autonomic dysfunction).
 

Key Steps

The guidelines address two key steps for each PNS manifestation — the workup and evaluation of patients with suspected ANS manifestation including standard evaluations, diagnostic tests, and treatment. The experts developed 31 best practices for diagnosis and workup and 20 treatment recommendations.

Initial assessment of potential ANS involvement includes asking patients about orthostatic postural lightheadedness and difficulties with digestion, urination, sweating, and sexual function.

Treatment of autoimmune diseases typically focuses on relieving symptoms and can include steroids, the anticonvulsant gabapentin, the monoclonal antibody rituximab, and intravenous immunoglobulin. “The type of neuropathy will mandate or suggest certain therapies over others,” said Dr. Sarka, adding that a patient can have more than one neuropathy.

Therapeutics for Sjögren disease is another example of an area that has been “very haphazard,” he added.

The guidelines are aimed not just at specialists but also at general practitioners who treat many of these patients. But Dr. Hammitt emphasized that neurologists can be “instrumental” in identifying Sjögren disease in patients with PNS symptoms.

“Our hope is that specialists — in this case, neurologists — will recognize the potential for this condition in their PNS patients and ensure referral to a rheumatologist or knowledgeable family practitioner to manage overall care.”

The committee will soon submit its manuscript to the AAN for publication.

“Once published, we will have a robust dissemination strategy to ensure that providers, patients, and policymakers are aware of, and use, this very valuable resource,” said Dr. Hammitt.

No conflicts of interest were reported.

A version of this article appeared on Medscape.com.

New guidelines to manage peripheral neuropathy related to Sjögren disease have been developed by a multidisciplinary team of physicians from across medicine.

The guidelines will provide an evidence-based resource for the assessment, diagnosis, and treatment of various peripheral neuropathies related to the disorder.

Up until now, the field has been “haphazard and chaotic,” lead author George Sarka, MD, DrPH, MPH, director of the CME Committee for MemorialCare, Saddleback Medical Center, Laguna Hills, California, and member of the Sjögren Foundation PNS Guidelines Topic Review Group (TRG), told this news organization.

Dr. Sarka discussed the initiative at the American Academy of Neurology 2024 annual meeting.


 

Severe, Complex Illness

Sjögren disease is the second most common autoimmune rheumatic disorder after rheumatoid arthritis, affecting an estimated 4 million Americans. Women make up most of the patient population at a ratio of 9:1.

The condition typically affects the mucous membranes and moisture-secreting glands of the eyes and mouth, resulting in decreased tears and saliva. But peripheral nervous system (PNS) manifestations often precede these symptoms and can occur in up to 60% of Sjögren disease cases.

“Traditionally, Sjögren’s was looked at as a dry eye and dry mouth disease, but we realize now that it’s so much broader than that,” said Dr. Sarka. “It’s a severe, systemic, and complex illness that can affect any body organ or system, and the nervous system is frequently affected.”

PNS manifestations cause more than mere discomfort; they can lead to diagnostic and management challenges, costly treatments, and diminished quality of life.

Getting a proper diagnosis goes a long way toward improving the quality of life for these patients, Steven Mandel, MD, clinical professor of neurology at the Zucker School of Medicine at Hofstra-Northwell, adjunct clinical professor of medicine at NY Medical College, New York City, and member of the TRG, told this news organization.

The problem is, doctors don’t always think an autoimmune disorder is causing the symptoms, said Dr. Sarka. “There’s an old adage in neurology that if you don’t think about it, you’re going to miss it; you have to ask, and that’s what we’re trying to get people to do.”

The condition often accompanies other immune system disorders such as rheumatoid arthritis and lupus. But as patients are referred back and forth between ophthalmologists, rheumatologists, and neurologists, the condition is often missed. “It could be 4 or 5 years before a definitive diagnosis of Sjögren’s is made,” said Dr. Sarka.

He believes the education system is partly to blame. “Medical schools have been very deficient in teaching people about recognizing Sjögren disease.”

That leaves many physicians at a loss about “what to do with these patients when they walk in the door,” said Dr. Mandel. “They don’t know how to manage them; they don’t know how to diagnose them; and they don’t know how to treat them.”

Developing guidelines with multispecialty collaboration was “absolutely critical” in addressing this knowledge gap, Dr. Mandel added. That process involved “a very rigorous and transparent methodology so that it would be accepted by all the professionals involved in Sjögren’s,” he said.

The process took 3 years and involved amassing and grading the evidence, getting consensus from committee members, developing recommendations, and getting feedback and external review.
 

Scant Evidence

An early literature search revealed very little evidence on PNS manifestations in patients with Sjögren disease, so the guideline committee “leaned very heavily on expert opinion” to develop recommendations, Kathy Hammitt, MA, vice president of Medical and Scientific Affairs, Sjögren’s Foundation, told this news organization.

The literature search also showed different terms are used to describe PNS, “which is where the chaos comes in,” said Dr. Sarka.

Experts from different specialties worked together to define and align nomenclature used by various specialists. They developed definitions for seven PNS categories including mononeuropathy, large fiber neuropathy, small fiber neuropathy, demyelinating polyradiculoneuropathy, ganglionopathy, vasculitis neuropathy, and autoimmune nervous system neuropathy.

The guidelines pertaining to PNS manifestations encompass a spectrum of neurologic abnormalities, including cranial neuropathies (trigeminal neuropathy or acute facial neuropathy), polyneuropathies (large fiber neuropathy, small fiber neuropathy, demyelinating polyradiculoneuropathy, vasculitis neuropathy, or ganglionopathy), and autonomic nervous system (ANS) neuropathies (postural tachycardia, orthostatic hypotension, or autonomic dysfunction).
 

Key Steps

The guidelines address two key steps for each PNS manifestation — the workup and evaluation of patients with suspected ANS manifestation including standard evaluations, diagnostic tests, and treatment. The experts developed 31 best practices for diagnosis and workup and 20 treatment recommendations.

Initial assessment of potential ANS involvement includes asking patients about orthostatic postural lightheadedness and difficulties with digestion, urination, sweating, and sexual function.

Treatment of autoimmune diseases typically focuses on relieving symptoms and can include steroids, the anticonvulsant gabapentin, the monoclonal antibody rituximab, and intravenous immunoglobulin. “The type of neuropathy will mandate or suggest certain therapies over others,” said Dr. Sarka, adding that a patient can have more than one neuropathy.

Therapeutics for Sjögren disease is another example of an area that has been “very haphazard,” he added.

The guidelines are aimed not just at specialists but also at general practitioners who treat many of these patients. But Dr. Hammitt emphasized that neurologists can be “instrumental” in identifying Sjögren disease in patients with PNS symptoms.

“Our hope is that specialists — in this case, neurologists — will recognize the potential for this condition in their PNS patients and ensure referral to a rheumatologist or knowledgeable family practitioner to manage overall care.”

The committee will soon submit its manuscript to the AAN for publication.

“Once published, we will have a robust dissemination strategy to ensure that providers, patients, and policymakers are aware of, and use, this very valuable resource,” said Dr. Hammitt.

No conflicts of interest were reported.

A version of this article appeared on Medscape.com.

LIVERPOOL, ENGLAND — An updated guideline from the British Society for Rheumatology (BSR) on the management of Sjögren disease asks rheumatologists and other clinicians caring for patients with the condition to “do the little things well” rather than overly focusing on rheumatologic treatments. The guideline’s new format provides recommendations for specific clinical questions and now also includes recommendations for managing the disease in children and adolescents. 

“The original guideline was published in 2017, and things move on very rapidly,” consultant rheumatologist Elizabeth Price, MBBCh, PhD, said ahead of her presentation of the updated guideline at the annual meeting of the British Society for Rheumatology.

167930_Price_Elizabeth_web.jpg

What’s in a Name?

The BSR guideline on the management of adult and juvenile onset Sjögren disease is published in Rheumatology and is available via the BSR website, where it is accompanied by a short summary sheet. 

The most notable change perhaps is the name the guideline now uses, Dr. Price said at BSR 2024. “We have been bold and called it Sjögren disease.” Previously, the guideline used the term primary Sjögren’s syndrome, but there has been a “move away from using eponymous syndromes and dropping s’s and apostrophes,” she explained. 

Another significant change is that advice on managing Sjögren disease in children and adolescents is now included where appropriate, meaning that the British guideline is now the first to cover Sjögren disease “across the ages,” Dr. Price said.

A pediatric/adolescent rheumatologist joined the guideline working group, which already consisted of several adult rheumatologists, ophthalmologists, and a dentistry consultant. The group now comprises 22 members total, including a general practitioner, an oncologist, a renal physician, an occupational therapist, two patients with Sjögren disease, and a librarian.
 

Confirming the Diagnosis

The first questions asked to help form the new recommendations were around confirming a diagnosis of Sjögren disease, such as what is the diagnostic accuracy of antinuclear antibodies (ANAs), extractable nuclear antigens (ENAs), and other novel antigens in Sjögren disease? And what is the diagnostic accuracy of salivary gland ultrasound, imaging in general, and salivary gland or lacrimal gland biopsies? 

The resulting recommendations advised not to measure ANAs in the absence of clinical indicators of Sjögren disease or any other connective tissue disease but to use it to screen if there was a clinical suspicion. And ENAs should be measured even if the ANAs were negative and there is a high index of suspicion. 

In terms of imaging, ultrasound of the salivary glands was thought to be useful, but other imaging was not recommended for routine practice at the current time. Minor lip but not lacrimal gland biopsies were recommended if clinical and serologic features were not enough to make a diagnosis.
 

Lymphoma Worries

The 2017 version of the guideline did not include information about lymphoma, but this is the thing that most patients with Sjögren disease will worry about, Dr. Price said. “They all look it up on YouTube, they all come back and tell me that they are really worried they’ll develop it.”

The question that was therefore posed was whether there were any measurable biomarkers that could predict the development of lymphoma in adults and children. Seven predictors were found, the strongest being a low level of complement C4 alone or together with low levels of C3. Other predictors were salivary gland enlargement, lymphadenopathy, anti-Ro/La and rheumatoid factor autoantibodies, cryoglobulinemia, monoclonal gammopathy, and a high focus score. 

All of these predictors put someone in a higher risk category for lymphoma. If two or fewer of those features are present, the lifetime risk is “probably below 2%,” Dr. Price said. However, if all seven are present, the lifetime risk is “approaching 100%.”

The recommendation made on the basis of these findings is that people with Sjögren disease need to be offered early further investigation if they present with any new salivary gland swelling or other symptoms that might suggest the development of lymphoma. In this regard, a labial salivary gland biopsy might provide additional prognostic information.
 

‘Do the Little Things Well’

“You have to do the little things well,” Dr. Price said. “Many of the patients [who] come to see me for a second opinion have not been prescribed the right eye drops, have not been given advice on dental care,” with their management taking “too much on the rheumatological treatments.”

Rheumatologists are of course not trained or expected to be experts in ophthalmology or dentistry, but “you need to look at their mouth and you do need to examine their eyes, and you do need to give them some advice,” Dr. Price advised.

Thankfully, that is where the updated guidelines should help, with a recommendation that people with Sjögren disease should use preservative-free eye drops every 2-3 hours.

“It’s vital you avoid preservatives, because preservatives flatten the corneal surface and reduce the surface area and can cause inflammation in their own right,” Dr. Price cautioned, adding that there are plenty of suitable eye drop formulations available. 

In regard to helping with dry mouth symptoms, the recommendation is to use a saliva substitute for symptomatic relief. For vaginal dryness, the recommendation is to consider advising topical nonhormonal vaginal moisturizers plus estrogen creams or pessaries in peri- or postmenopausal women with significant vaginal dryness. 

“Very important, however, is to maintain a neutral pH, an alkaline environment in the mouth because acid damages dental enamel,” Dr. Price said. Conversely, an acidic vaginal moisturizer is needed to treat vaginal dryness. 

Dental hygiene is important. Regular brushing with a fluoride-based toothpaste is advised. The use of xylitol-containing products has been shown to reduce bacteria known to increase the risk for dental decay. Telling patients not to eat between meals is also simple but important advice. 

“We do recommend that patients are assessed holistically,” Dr. Price said, noting that they should be offered access to cognitive-behavioral therapy and exercise therapies to help with the symptoms of fatigue and joint pain.
 

Watch Out for Comorbidities

Sjögren disease is associated with many comorbidities, some of which might be predicted from the age and demographics of the people who are normally affected. 

“This is on the whole an older, female population, so you see a lot of osteoarthritis, gastroesophageal reflux, and hypertension,” Dr. Price said. “However, you may not be aware that 1 in 5 of these patients develop thyroid disease,” and there is a higher rate of celiac disease and primary biliary cholangitis than is seen in the general background population. 

The recommendation, therefore, is to “be aware of and consider screening for commonly associated conditions, as guided by age and/or clinical presentation.” As such, it’s recommended that baseline and repeated investigations that look for signs of comorbidity are performed, such as thyroid function assessment and liver function tests, to name two.
 

Treatment Recommendations

As in the original guideline, the treatment of systemic disease is discussed, but the advice has been overhauled with the availability of new data. 

The updated guidance notes that a trial of hydroxychloroquine for 6-12 months is the recommended treatment approach for people with fatigue and systemic symptoms. 

Systemic steroids may be used in the short-term for specific indications but should not be offered routinely. 

Conventional immunosuppressive or biologic drugs and immunoglobulins are not currently recommended outside of managing specific systemic complications.

In juvenile cases, the treatment of recurrent swollen parotid glands that are not due to infection or stone disease should include a short course of a nonsteroidal anti-inflammatory drug or a short course of oral steroids. This should be combined with massage followed by washouts with saline or steroids. In refractory cases, escalation to anti–B-cell–targeted therapies may be considered in select situations.
 

View on Updates

Patient advocate Bridget Crampton, who leads the helpline team at Sjögren’s UK (formerly the British Sjögren’s Syndrome Association), commented on the importance of the guidelines during a roundtable held by the BSR. 

“I think it will help [patients] make better use of their own appointments. So, they’ll know what treatments might be offered. They’ll know what they want to talk about at their appointments,” she said. 

Ms. Crampton, who has lived with Sjögren disease herself for the past 20 years, added: “I think it’s important for patients that we have guidelines like this. It means that all clinicians can easily access information. My hope is that it might standardize care across the UK a little bit more.” 

No specific funding was received to create the guidelines, be that from any bodies in the public, commercial, or not-for-profit sectors. No conflicts of interests were expressed by any of the experts quoted in this article.

A version of this article appeared on Medscape.com.

LIVERPOOL, ENGLAND — An updated guideline from the British Society for Rheumatology (BSR) on the management of Sjögren disease asks rheumatologists and other clinicians caring for patients with the condition to “do the little things well” rather than overly focusing on rheumatologic treatments. The guideline’s new format provides recommendations for specific clinical questions and now also includes recommendations for managing the disease in children and adolescents. 

“The original guideline was published in 2017, and things move on very rapidly,” consultant rheumatologist Elizabeth Price, MBBCh, PhD, said ahead of her presentation of the updated guideline at the annual meeting of the British Society for Rheumatology.

167930_Price_Elizabeth_web.jpg

What’s in a Name?

The BSR guideline on the management of adult and juvenile onset Sjögren disease is published in Rheumatology and is available via the BSR website, where it is accompanied by a short summary sheet. 

The most notable change perhaps is the name the guideline now uses, Dr. Price said at BSR 2024. “We have been bold and called it Sjögren disease.” Previously, the guideline used the term primary Sjögren’s syndrome, but there has been a “move away from using eponymous syndromes and dropping s’s and apostrophes,” she explained. 

Another significant change is that advice on managing Sjögren disease in children and adolescents is now included where appropriate, meaning that the British guideline is now the first to cover Sjögren disease “across the ages,” Dr. Price said.

A pediatric/adolescent rheumatologist joined the guideline working group, which already consisted of several adult rheumatologists, ophthalmologists, and a dentistry consultant. The group now comprises 22 members total, including a general practitioner, an oncologist, a renal physician, an occupational therapist, two patients with Sjögren disease, and a librarian.
 

Confirming the Diagnosis

The first questions asked to help form the new recommendations were around confirming a diagnosis of Sjögren disease, such as what is the diagnostic accuracy of antinuclear antibodies (ANAs), extractable nuclear antigens (ENAs), and other novel antigens in Sjögren disease? And what is the diagnostic accuracy of salivary gland ultrasound, imaging in general, and salivary gland or lacrimal gland biopsies? 

The resulting recommendations advised not to measure ANAs in the absence of clinical indicators of Sjögren disease or any other connective tissue disease but to use it to screen if there was a clinical suspicion. And ENAs should be measured even if the ANAs were negative and there is a high index of suspicion. 

In terms of imaging, ultrasound of the salivary glands was thought to be useful, but other imaging was not recommended for routine practice at the current time. Minor lip but not lacrimal gland biopsies were recommended if clinical and serologic features were not enough to make a diagnosis.
 

Lymphoma Worries

The 2017 version of the guideline did not include information about lymphoma, but this is the thing that most patients with Sjögren disease will worry about, Dr. Price said. “They all look it up on YouTube, they all come back and tell me that they are really worried they’ll develop it.”

The question that was therefore posed was whether there were any measurable biomarkers that could predict the development of lymphoma in adults and children. Seven predictors were found, the strongest being a low level of complement C4 alone or together with low levels of C3. Other predictors were salivary gland enlargement, lymphadenopathy, anti-Ro/La and rheumatoid factor autoantibodies, cryoglobulinemia, monoclonal gammopathy, and a high focus score. 

All of these predictors put someone in a higher risk category for lymphoma. If two or fewer of those features are present, the lifetime risk is “probably below 2%,” Dr. Price said. However, if all seven are present, the lifetime risk is “approaching 100%.”

The recommendation made on the basis of these findings is that people with Sjögren disease need to be offered early further investigation if they present with any new salivary gland swelling or other symptoms that might suggest the development of lymphoma. In this regard, a labial salivary gland biopsy might provide additional prognostic information.
 

‘Do the Little Things Well’

“You have to do the little things well,” Dr. Price said. “Many of the patients [who] come to see me for a second opinion have not been prescribed the right eye drops, have not been given advice on dental care,” with their management taking “too much on the rheumatological treatments.”

Rheumatologists are of course not trained or expected to be experts in ophthalmology or dentistry, but “you need to look at their mouth and you do need to examine their eyes, and you do need to give them some advice,” Dr. Price advised.

Thankfully, that is where the updated guidelines should help, with a recommendation that people with Sjögren disease should use preservative-free eye drops every 2-3 hours.

“It’s vital you avoid preservatives, because preservatives flatten the corneal surface and reduce the surface area and can cause inflammation in their own right,” Dr. Price cautioned, adding that there are plenty of suitable eye drop formulations available. 

In regard to helping with dry mouth symptoms, the recommendation is to use a saliva substitute for symptomatic relief. For vaginal dryness, the recommendation is to consider advising topical nonhormonal vaginal moisturizers plus estrogen creams or pessaries in peri- or postmenopausal women with significant vaginal dryness. 

“Very important, however, is to maintain a neutral pH, an alkaline environment in the mouth because acid damages dental enamel,” Dr. Price said. Conversely, an acidic vaginal moisturizer is needed to treat vaginal dryness. 

Dental hygiene is important. Regular brushing with a fluoride-based toothpaste is advised. The use of xylitol-containing products has been shown to reduce bacteria known to increase the risk for dental decay. Telling patients not to eat between meals is also simple but important advice. 

“We do recommend that patients are assessed holistically,” Dr. Price said, noting that they should be offered access to cognitive-behavioral therapy and exercise therapies to help with the symptoms of fatigue and joint pain.
 

Watch Out for Comorbidities

Sjögren disease is associated with many comorbidities, some of which might be predicted from the age and demographics of the people who are normally affected. 

“This is on the whole an older, female population, so you see a lot of osteoarthritis, gastroesophageal reflux, and hypertension,” Dr. Price said. “However, you may not be aware that 1 in 5 of these patients develop thyroid disease,” and there is a higher rate of celiac disease and primary biliary cholangitis than is seen in the general background population. 

The recommendation, therefore, is to “be aware of and consider screening for commonly associated conditions, as guided by age and/or clinical presentation.” As such, it’s recommended that baseline and repeated investigations that look for signs of comorbidity are performed, such as thyroid function assessment and liver function tests, to name two.
 

Treatment Recommendations

As in the original guideline, the treatment of systemic disease is discussed, but the advice has been overhauled with the availability of new data. 

The updated guidance notes that a trial of hydroxychloroquine for 6-12 months is the recommended treatment approach for people with fatigue and systemic symptoms. 

Systemic steroids may be used in the short-term for specific indications but should not be offered routinely. 

Conventional immunosuppressive or biologic drugs and immunoglobulins are not currently recommended outside of managing specific systemic complications.

In juvenile cases, the treatment of recurrent swollen parotid glands that are not due to infection or stone disease should include a short course of a nonsteroidal anti-inflammatory drug or a short course of oral steroids. This should be combined with massage followed by washouts with saline or steroids. In refractory cases, escalation to anti–B-cell–targeted therapies may be considered in select situations.
 

View on Updates

Patient advocate Bridget Crampton, who leads the helpline team at Sjögren’s UK (formerly the British Sjögren’s Syndrome Association), commented on the importance of the guidelines during a roundtable held by the BSR. 

“I think it will help [patients] make better use of their own appointments. So, they’ll know what treatments might be offered. They’ll know what they want to talk about at their appointments,” she said. 

Ms. Crampton, who has lived with Sjögren disease herself for the past 20 years, added: “I think it’s important for patients that we have guidelines like this. It means that all clinicians can easily access information. My hope is that it might standardize care across the UK a little bit more.” 

No specific funding was received to create the guidelines, be that from any bodies in the public, commercial, or not-for-profit sectors. No conflicts of interests were expressed by any of the experts quoted in this article.

A version of this article appeared on Medscape.com.

LIVERPOOL, ENGLAND — An updated guideline from the British Society for Rheumatology (BSR) on the management of Sjögren disease asks rheumatologists and other clinicians caring for patients with the condition to “do the little things well” rather than overly focusing on rheumatologic treatments. The guideline’s new format provides recommendations for specific clinical questions and now also includes recommendations for managing the disease in children and adolescents. 

“The original guideline was published in 2017, and things move on very rapidly,” consultant rheumatologist Elizabeth Price, MBBCh, PhD, said ahead of her presentation of the updated guideline at the annual meeting of the British Society for Rheumatology.

167930_Price_Elizabeth_web.jpg

What’s in a Name?

The BSR guideline on the management of adult and juvenile onset Sjögren disease is published in Rheumatology and is available via the BSR website, where it is accompanied by a short summary sheet. 

The most notable change perhaps is the name the guideline now uses, Dr. Price said at BSR 2024. “We have been bold and called it Sjögren disease.” Previously, the guideline used the term primary Sjögren’s syndrome, but there has been a “move away from using eponymous syndromes and dropping s’s and apostrophes,” she explained. 

Another significant change is that advice on managing Sjögren disease in children and adolescents is now included where appropriate, meaning that the British guideline is now the first to cover Sjögren disease “across the ages,” Dr. Price said.

A pediatric/adolescent rheumatologist joined the guideline working group, which already consisted of several adult rheumatologists, ophthalmologists, and a dentistry consultant. The group now comprises 22 members total, including a general practitioner, an oncologist, a renal physician, an occupational therapist, two patients with Sjögren disease, and a librarian.
 

Confirming the Diagnosis

The first questions asked to help form the new recommendations were around confirming a diagnosis of Sjögren disease, such as what is the diagnostic accuracy of antinuclear antibodies (ANAs), extractable nuclear antigens (ENAs), and other novel antigens in Sjögren disease? And what is the diagnostic accuracy of salivary gland ultrasound, imaging in general, and salivary gland or lacrimal gland biopsies? 

The resulting recommendations advised not to measure ANAs in the absence of clinical indicators of Sjögren disease or any other connective tissue disease but to use it to screen if there was a clinical suspicion. And ENAs should be measured even if the ANAs were negative and there is a high index of suspicion. 

In terms of imaging, ultrasound of the salivary glands was thought to be useful, but other imaging was not recommended for routine practice at the current time. Minor lip but not lacrimal gland biopsies were recommended if clinical and serologic features were not enough to make a diagnosis.
 

Lymphoma Worries

The 2017 version of the guideline did not include information about lymphoma, but this is the thing that most patients with Sjögren disease will worry about, Dr. Price said. “They all look it up on YouTube, they all come back and tell me that they are really worried they’ll develop it.”

The question that was therefore posed was whether there were any measurable biomarkers that could predict the development of lymphoma in adults and children. Seven predictors were found, the strongest being a low level of complement C4 alone or together with low levels of C3. Other predictors were salivary gland enlargement, lymphadenopathy, anti-Ro/La and rheumatoid factor autoantibodies, cryoglobulinemia, monoclonal gammopathy, and a high focus score. 

All of these predictors put someone in a higher risk category for lymphoma. If two or fewer of those features are present, the lifetime risk is “probably below 2%,” Dr. Price said. However, if all seven are present, the lifetime risk is “approaching 100%.”

The recommendation made on the basis of these findings is that people with Sjögren disease need to be offered early further investigation if they present with any new salivary gland swelling or other symptoms that might suggest the development of lymphoma. In this regard, a labial salivary gland biopsy might provide additional prognostic information.
 

‘Do the Little Things Well’

“You have to do the little things well,” Dr. Price said. “Many of the patients [who] come to see me for a second opinion have not been prescribed the right eye drops, have not been given advice on dental care,” with their management taking “too much on the rheumatological treatments.”

Rheumatologists are of course not trained or expected to be experts in ophthalmology or dentistry, but “you need to look at their mouth and you do need to examine their eyes, and you do need to give them some advice,” Dr. Price advised.

Thankfully, that is where the updated guidelines should help, with a recommendation that people with Sjögren disease should use preservative-free eye drops every 2-3 hours.

“It’s vital you avoid preservatives, because preservatives flatten the corneal surface and reduce the surface area and can cause inflammation in their own right,” Dr. Price cautioned, adding that there are plenty of suitable eye drop formulations available. 

In regard to helping with dry mouth symptoms, the recommendation is to use a saliva substitute for symptomatic relief. For vaginal dryness, the recommendation is to consider advising topical nonhormonal vaginal moisturizers plus estrogen creams or pessaries in peri- or postmenopausal women with significant vaginal dryness. 

“Very important, however, is to maintain a neutral pH, an alkaline environment in the mouth because acid damages dental enamel,” Dr. Price said. Conversely, an acidic vaginal moisturizer is needed to treat vaginal dryness. 

Dental hygiene is important. Regular brushing with a fluoride-based toothpaste is advised. The use of xylitol-containing products has been shown to reduce bacteria known to increase the risk for dental decay. Telling patients not to eat between meals is also simple but important advice. 

“We do recommend that patients are assessed holistically,” Dr. Price said, noting that they should be offered access to cognitive-behavioral therapy and exercise therapies to help with the symptoms of fatigue and joint pain.
 

Watch Out for Comorbidities

Sjögren disease is associated with many comorbidities, some of which might be predicted from the age and demographics of the people who are normally affected. 

“This is on the whole an older, female population, so you see a lot of osteoarthritis, gastroesophageal reflux, and hypertension,” Dr. Price said. “However, you may not be aware that 1 in 5 of these patients develop thyroid disease,” and there is a higher rate of celiac disease and primary biliary cholangitis than is seen in the general background population. 

The recommendation, therefore, is to “be aware of and consider screening for commonly associated conditions, as guided by age and/or clinical presentation.” As such, it’s recommended that baseline and repeated investigations that look for signs of comorbidity are performed, such as thyroid function assessment and liver function tests, to name two.
 

Treatment Recommendations

As in the original guideline, the treatment of systemic disease is discussed, but the advice has been overhauled with the availability of new data. 

The updated guidance notes that a trial of hydroxychloroquine for 6-12 months is the recommended treatment approach for people with fatigue and systemic symptoms. 

Systemic steroids may be used in the short-term for specific indications but should not be offered routinely. 

Conventional immunosuppressive or biologic drugs and immunoglobulins are not currently recommended outside of managing specific systemic complications.

In juvenile cases, the treatment of recurrent swollen parotid glands that are not due to infection or stone disease should include a short course of a nonsteroidal anti-inflammatory drug or a short course of oral steroids. This should be combined with massage followed by washouts with saline or steroids. In refractory cases, escalation to anti–B-cell–targeted therapies may be considered in select situations.
 

View on Updates

Patient advocate Bridget Crampton, who leads the helpline team at Sjögren’s UK (formerly the British Sjögren’s Syndrome Association), commented on the importance of the guidelines during a roundtable held by the BSR. 

“I think it will help [patients] make better use of their own appointments. So, they’ll know what treatments might be offered. They’ll know what they want to talk about at their appointments,” she said. 

Ms. Crampton, who has lived with Sjögren disease herself for the past 20 years, added: “I think it’s important for patients that we have guidelines like this. It means that all clinicians can easily access information. My hope is that it might standardize care across the UK a little bit more.” 

No specific funding was received to create the guidelines, be that from any bodies in the public, commercial, or not-for-profit sectors. No conflicts of interests were expressed by any of the experts quoted in this article.

A version of this article appeared on Medscape.com.

LIVERPOOL, ENGLAND — The British Society for Rheumatology (BSR) and the British Association of Dermatologists (BAD) have joined forces for the first time to develop the first British guidelines for the management of people living with Behçet’s disease.

The guidelines will also be the first “living guidelines” produced by either society, which means they will be regularly revised and updated when new evidence emerges that warrants inclusion.

With more than 90 recommendations being made, the new guidelines promise to be the most comprehensive and most up-to-date yet for what is regarded as a rare disease. Robert Moots, MBBS, PhD, provided a “sneak peek” of the guidelines at the annual meeting of the British Society for Rheumatology.

Dr. Moots, professor of rheumatology at the University of Liverpool and a consultant rheumatologist for Liverpool University Hospitals NHS Foundation Trust in England, noted that while the European Alliance of Associations for Rheumatology has produced a guideline for Behçet’s disease, this was last updated in 2018 and is not specific for the population for patients that is seen in the United Kingdom.

The British recommendations will cover all possible manifestations of Behçet’s disease and give practical advice on how to manage everything from the most common presentations such as skin lesions, mouth ulcers, and genital ulcers, as well as the potentially more serious eye, neurological, and vascular involvement.

167862_Moots_Robert_web.jpg

Importance of Raising Awareness

“Joint and musculoskeletal problems are actually one of the least complained of symptoms in people with Behçet’s, and they often can’t understand why a rheumatologist is seeing them,” Dr. Moot said. “But of course, people do get joint problems, they can get enthesitis and arthralgia.”

Dr. Moots has been leading one of the three National Health Service (NHS) Centres of Excellence for Behçet’s Syndrome in England for more than a decade and told this news organization that diagnosing patients could be challenging. It can take up to 10 years from the first symptoms appearing to getting a diagnosis, so part of the job of the NHS Centres of Excellence is to raise awareness among both the healthcare profession and the general public.

“It’s a condition that people learn about at medical school. Most doctors will have come across it, but because it was thought to be really rare in the UK, nobody perhaps really expects to see it,” Dr. Moot said.

“But we all have these patients,” he added. “In Liverpool, we’re commissioned to be looking after an anticipated 150 people with Behçet’s — we’ve got 700. With more awareness, there’s more diagnoses being made, and people are being looked after better.”
 

Patient Perspective

Tony Thornburn, OBE, chair of the patient advocacy group Behçet’s UK, agreed in a separate interview that raising awareness of the syndrome was key to improving its management.

“Patients have said that it is a bit like having arthritis, lupus, MS [multiple sclerosis], and Crohn’s [disease] all at once,” Mr. Thorburn said. “So what we need is a guideline to ensure that people know what they’re looking at.”

Mr. Thorburn added, “Guidelines are important for raising awareness but also providing the detailed information that clinicians and GPs [general practitioners] need to have to treat a patient when they come in with this multifaceted condition.”
 

Multifaceted Means Multidisciplinary Management

Because there can be so many different aspects to managing someone with Behçet’s disease, a multispecialty team that was convened to develop the guidelines agreed that multidisciplinary management should be an overarching theme.

“The guideline development group consisted of all the specialties that you would need for a complex multisystem disease like Behçet’s,” Dr. Moot said. He highlighted that working alongside the consultants in adult and pediatric rheumatology were specialists in dermatology, gastroenterology, neurology, ophthalmology, obstetrics and gynecology, and psychology.

“We’re actually looking at psychological interactions and their impact for the first time,” Dr. Moot said, noting that clinicians needed to “take it seriously, and ask about it.”
 

Management of Manifestations

One of the general principles of the guidelines is to assess the involvement of each organ system and target treatment accordingly.

“One of the problems is that the evidence base to tell us what to do is pretty low,” Dr. Moots acknowledged. There have been few good quality randomized trials, so “treatment tends to be eminence-based rather than evidence-based.”

The recommendation wording bears this in mind, stating whether a treatment should or should not be offered, or just considered if there is no strong evidence to back up its use.

With regard to musculoskeletal manifestations, the recommendations say that colchicine should be offered, perhaps as a first-line option, or an intraarticular steroid injection in the case of monoarthritis. An intramuscular depot steroid may also be appropriate to offer, and there was good evidence to offer azathioprine or, as an alternative in refractory cases, a tumor necrosis factor (TNF) inhibitor. Nonsteroidal anti-inflammatory drugs, methotrexate, apremilast, secukinumab, and referral to a physiotherapist could only be considered, however, based on weaker levels of evidence for their use.

To treat mucocutaneous disease, the guidelines advise offering topical steroids in the form of ointment for genital ulcers or mouthwash or ointment for oral ulcers. For skin lesions, it is recommended to offer colchicine, azathioprine, mycophenolate mofetil, or TNF inhibitor and to consider the use of apremilast, secukinumab, or dapsone.
 

Future Work and Revision

“One of the key things we would like to see developing is a national registry,” Dr. Moots said. This would include biobanking samples for future research and possible genomic and phenotyping studies.

More work needs to be done in conducting clinical trials in children and young people with Behçet’s disease, studies to find prognostic factors for neurological disease, and clinical trials of potential new drug approaches such as Janus kinase inhibitors. Importantly, an auditing process needs to be set up to see what effect, if any, the guidelines will actually have onpatient management.

“It’s taken 5 years to today” to develop the guidelines, Dr. Moot said. What form the process of updating them will take still has to be decided, he said in the interview. It is likely that the necessary literature searches will be performed every 6 months or so, but it will be a compromise between the ideal situation and having the staffing time to do it.

“It’s a big ask,” Dr. Moot acknowledged, adding that even if updates were only once a year, it would still be much faster than the 5- or 6-year cycle that it traditionally takes for most guidelines to be updated.

The BSR and BAD’s processes for developing guidelines are accredited by the National Institute for Health and Care Excellence in England. Dr. Moots is the chief investigator for the Secukinumab in Behçet’s trial, which is sponsored by the Liverpool University Hospitals NHS Foundation Trust via grant funding from Novartis.
 

A version of this article appeared on Medscape.com.

LIVERPOOL, ENGLAND — The British Society for Rheumatology (BSR) and the British Association of Dermatologists (BAD) have joined forces for the first time to develop the first British guidelines for the management of people living with Behçet’s disease.

The guidelines will also be the first “living guidelines” produced by either society, which means they will be regularly revised and updated when new evidence emerges that warrants inclusion.

With more than 90 recommendations being made, the new guidelines promise to be the most comprehensive and most up-to-date yet for what is regarded as a rare disease. Robert Moots, MBBS, PhD, provided a “sneak peek” of the guidelines at the annual meeting of the British Society for Rheumatology.

Dr. Moots, professor of rheumatology at the University of Liverpool and a consultant rheumatologist for Liverpool University Hospitals NHS Foundation Trust in England, noted that while the European Alliance of Associations for Rheumatology has produced a guideline for Behçet’s disease, this was last updated in 2018 and is not specific for the population for patients that is seen in the United Kingdom.

The British recommendations will cover all possible manifestations of Behçet’s disease and give practical advice on how to manage everything from the most common presentations such as skin lesions, mouth ulcers, and genital ulcers, as well as the potentially more serious eye, neurological, and vascular involvement.

167862_Moots_Robert_web.jpg

Importance of Raising Awareness

“Joint and musculoskeletal problems are actually one of the least complained of symptoms in people with Behçet’s, and they often can’t understand why a rheumatologist is seeing them,” Dr. Moot said. “But of course, people do get joint problems, they can get enthesitis and arthralgia.”

Dr. Moots has been leading one of the three National Health Service (NHS) Centres of Excellence for Behçet’s Syndrome in England for more than a decade and told this news organization that diagnosing patients could be challenging. It can take up to 10 years from the first symptoms appearing to getting a diagnosis, so part of the job of the NHS Centres of Excellence is to raise awareness among both the healthcare profession and the general public.

“It’s a condition that people learn about at medical school. Most doctors will have come across it, but because it was thought to be really rare in the UK, nobody perhaps really expects to see it,” Dr. Moot said.

“But we all have these patients,” he added. “In Liverpool, we’re commissioned to be looking after an anticipated 150 people with Behçet’s — we’ve got 700. With more awareness, there’s more diagnoses being made, and people are being looked after better.”
 

Patient Perspective

Tony Thornburn, OBE, chair of the patient advocacy group Behçet’s UK, agreed in a separate interview that raising awareness of the syndrome was key to improving its management.

“Patients have said that it is a bit like having arthritis, lupus, MS [multiple sclerosis], and Crohn’s [disease] all at once,” Mr. Thorburn said. “So what we need is a guideline to ensure that people know what they’re looking at.”

Mr. Thorburn added, “Guidelines are important for raising awareness but also providing the detailed information that clinicians and GPs [general practitioners] need to have to treat a patient when they come in with this multifaceted condition.”
 

Multifaceted Means Multidisciplinary Management

Because there can be so many different aspects to managing someone with Behçet’s disease, a multispecialty team that was convened to develop the guidelines agreed that multidisciplinary management should be an overarching theme.

“The guideline development group consisted of all the specialties that you would need for a complex multisystem disease like Behçet’s,” Dr. Moot said. He highlighted that working alongside the consultants in adult and pediatric rheumatology were specialists in dermatology, gastroenterology, neurology, ophthalmology, obstetrics and gynecology, and psychology.

“We’re actually looking at psychological interactions and their impact for the first time,” Dr. Moot said, noting that clinicians needed to “take it seriously, and ask about it.”
 

Management of Manifestations

One of the general principles of the guidelines is to assess the involvement of each organ system and target treatment accordingly.

“One of the problems is that the evidence base to tell us what to do is pretty low,” Dr. Moots acknowledged. There have been few good quality randomized trials, so “treatment tends to be eminence-based rather than evidence-based.”

The recommendation wording bears this in mind, stating whether a treatment should or should not be offered, or just considered if there is no strong evidence to back up its use.

With regard to musculoskeletal manifestations, the recommendations say that colchicine should be offered, perhaps as a first-line option, or an intraarticular steroid injection in the case of monoarthritis. An intramuscular depot steroid may also be appropriate to offer, and there was good evidence to offer azathioprine or, as an alternative in refractory cases, a tumor necrosis factor (TNF) inhibitor. Nonsteroidal anti-inflammatory drugs, methotrexate, apremilast, secukinumab, and referral to a physiotherapist could only be considered, however, based on weaker levels of evidence for their use.

To treat mucocutaneous disease, the guidelines advise offering topical steroids in the form of ointment for genital ulcers or mouthwash or ointment for oral ulcers. For skin lesions, it is recommended to offer colchicine, azathioprine, mycophenolate mofetil, or TNF inhibitor and to consider the use of apremilast, secukinumab, or dapsone.
 

Future Work and Revision

“One of the key things we would like to see developing is a national registry,” Dr. Moots said. This would include biobanking samples for future research and possible genomic and phenotyping studies.

More work needs to be done in conducting clinical trials in children and young people with Behçet’s disease, studies to find prognostic factors for neurological disease, and clinical trials of potential new drug approaches such as Janus kinase inhibitors. Importantly, an auditing process needs to be set up to see what effect, if any, the guidelines will actually have onpatient management.

“It’s taken 5 years to today” to develop the guidelines, Dr. Moot said. What form the process of updating them will take still has to be decided, he said in the interview. It is likely that the necessary literature searches will be performed every 6 months or so, but it will be a compromise between the ideal situation and having the staffing time to do it.

“It’s a big ask,” Dr. Moot acknowledged, adding that even if updates were only once a year, it would still be much faster than the 5- or 6-year cycle that it traditionally takes for most guidelines to be updated.

The BSR and BAD’s processes for developing guidelines are accredited by the National Institute for Health and Care Excellence in England. Dr. Moots is the chief investigator for the Secukinumab in Behçet’s trial, which is sponsored by the Liverpool University Hospitals NHS Foundation Trust via grant funding from Novartis.
 

A version of this article appeared on Medscape.com.

LIVERPOOL, ENGLAND — The British Society for Rheumatology (BSR) and the British Association of Dermatologists (BAD) have joined forces for the first time to develop the first British guidelines for the management of people living with Behçet’s disease.

The guidelines will also be the first “living guidelines” produced by either society, which means they will be regularly revised and updated when new evidence emerges that warrants inclusion.

With more than 90 recommendations being made, the new guidelines promise to be the most comprehensive and most up-to-date yet for what is regarded as a rare disease. Robert Moots, MBBS, PhD, provided a “sneak peek” of the guidelines at the annual meeting of the British Society for Rheumatology.

Dr. Moots, professor of rheumatology at the University of Liverpool and a consultant rheumatologist for Liverpool University Hospitals NHS Foundation Trust in England, noted that while the European Alliance of Associations for Rheumatology has produced a guideline for Behçet’s disease, this was last updated in 2018 and is not specific for the population for patients that is seen in the United Kingdom.

The British recommendations will cover all possible manifestations of Behçet’s disease and give practical advice on how to manage everything from the most common presentations such as skin lesions, mouth ulcers, and genital ulcers, as well as the potentially more serious eye, neurological, and vascular involvement.

167862_Moots_Robert_web.jpg

Importance of Raising Awareness

“Joint and musculoskeletal problems are actually one of the least complained of symptoms in people with Behçet’s, and they often can’t understand why a rheumatologist is seeing them,” Dr. Moot said. “But of course, people do get joint problems, they can get enthesitis and arthralgia.”

Dr. Moots has been leading one of the three National Health Service (NHS) Centres of Excellence for Behçet’s Syndrome in England for more than a decade and told this news organization that diagnosing patients could be challenging. It can take up to 10 years from the first symptoms appearing to getting a diagnosis, so part of the job of the NHS Centres of Excellence is to raise awareness among both the healthcare profession and the general public.

“It’s a condition that people learn about at medical school. Most doctors will have come across it, but because it was thought to be really rare in the UK, nobody perhaps really expects to see it,” Dr. Moot said.

“But we all have these patients,” he added. “In Liverpool, we’re commissioned to be looking after an anticipated 150 people with Behçet’s — we’ve got 700. With more awareness, there’s more diagnoses being made, and people are being looked after better.”
 

Patient Perspective

Tony Thornburn, OBE, chair of the patient advocacy group Behçet’s UK, agreed in a separate interview that raising awareness of the syndrome was key to improving its management.

“Patients have said that it is a bit like having arthritis, lupus, MS [multiple sclerosis], and Crohn’s [disease] all at once,” Mr. Thorburn said. “So what we need is a guideline to ensure that people know what they’re looking at.”

Mr. Thorburn added, “Guidelines are important for raising awareness but also providing the detailed information that clinicians and GPs [general practitioners] need to have to treat a patient when they come in with this multifaceted condition.”
 

Multifaceted Means Multidisciplinary Management

Because there can be so many different aspects to managing someone with Behçet’s disease, a multispecialty team that was convened to develop the guidelines agreed that multidisciplinary management should be an overarching theme.

“The guideline development group consisted of all the specialties that you would need for a complex multisystem disease like Behçet’s,” Dr. Moot said. He highlighted that working alongside the consultants in adult and pediatric rheumatology were specialists in dermatology, gastroenterology, neurology, ophthalmology, obstetrics and gynecology, and psychology.

“We’re actually looking at psychological interactions and their impact for the first time,” Dr. Moot said, noting that clinicians needed to “take it seriously, and ask about it.”
 

Management of Manifestations

One of the general principles of the guidelines is to assess the involvement of each organ system and target treatment accordingly.

“One of the problems is that the evidence base to tell us what to do is pretty low,” Dr. Moots acknowledged. There have been few good quality randomized trials, so “treatment tends to be eminence-based rather than evidence-based.”

The recommendation wording bears this in mind, stating whether a treatment should or should not be offered, or just considered if there is no strong evidence to back up its use.

With regard to musculoskeletal manifestations, the recommendations say that colchicine should be offered, perhaps as a first-line option, or an intraarticular steroid injection in the case of monoarthritis. An intramuscular depot steroid may also be appropriate to offer, and there was good evidence to offer azathioprine or, as an alternative in refractory cases, a tumor necrosis factor (TNF) inhibitor. Nonsteroidal anti-inflammatory drugs, methotrexate, apremilast, secukinumab, and referral to a physiotherapist could only be considered, however, based on weaker levels of evidence for their use.

To treat mucocutaneous disease, the guidelines advise offering topical steroids in the form of ointment for genital ulcers or mouthwash or ointment for oral ulcers. For skin lesions, it is recommended to offer colchicine, azathioprine, mycophenolate mofetil, or TNF inhibitor and to consider the use of apremilast, secukinumab, or dapsone.
 

Future Work and Revision

“One of the key things we would like to see developing is a national registry,” Dr. Moots said. This would include biobanking samples for future research and possible genomic and phenotyping studies.

More work needs to be done in conducting clinical trials in children and young people with Behçet’s disease, studies to find prognostic factors for neurological disease, and clinical trials of potential new drug approaches such as Janus kinase inhibitors. Importantly, an auditing process needs to be set up to see what effect, if any, the guidelines will actually have onpatient management.

“It’s taken 5 years to today” to develop the guidelines, Dr. Moot said. What form the process of updating them will take still has to be decided, he said in the interview. It is likely that the necessary literature searches will be performed every 6 months or so, but it will be a compromise between the ideal situation and having the staffing time to do it.

“It’s a big ask,” Dr. Moot acknowledged, adding that even if updates were only once a year, it would still be much faster than the 5- or 6-year cycle that it traditionally takes for most guidelines to be updated.

The BSR and BAD’s processes for developing guidelines are accredited by the National Institute for Health and Care Excellence in England. Dr. Moots is the chief investigator for the Secukinumab in Behçet’s trial, which is sponsored by the Liverpool University Hospitals NHS Foundation Trust via grant funding from Novartis.
 

A version of this article appeared on Medscape.com.