Clinician Reviews

UPDATED July 8, 2024 // Editor's note: This article has been revised to give a more nuanced view from Yael Klionsky, MD, about the need for more accurate and consistent gout management guidelines for busy primary care clinicians who often rely on them in clinical practice.

VIENNA — The first multicenter randomized trial in gout to compare treat-to-target (T2T) and treat for symptom avoidance (T2S) strategies has finally generated data to make the guideline-recommended practice of T2T evidence-based.

The T2T strategy may be guideline-endorsed, but it has never been validated, contended Anusha Moses, MSc, a researcher and PhD candidate at the University of Twente, Enschede, the Netherlands. She argued that this controlled trial fills an evidence gap.

T2T is defined as maintaining a serum uric acid (sUA) level below the physiologic threshold level of 36 mmol/L (< 6 mg/dL). T2S, in contrast, is a strategy of symptom control, typically basing therapy on suppression of symptoms independent of sUA, Dr. Moses explained. 

Ted Bosworth/Medscape Medical News

Both the American College of Rheumatology and European Alliance of Associations for Rheumatology (EULAR) have already endorsed T2T, but other organizations, such as the American College of Physicians (ACP), still accept symptom-based treatment in its gout clinical practice guideline, according to Dr. Moses.

The results of the trial were not surprising based on the pathophysiology of gout. Elevated sUA is considered the driver of both flares and the complications of gout. This well-established association led to endorsement of T2T in guidelines from organizations such as EULAR, but Dr. Moses said a controlled trial allows this to be declared as evidence based.

To provide proof that T2T is superior, 308 gout patients at eight centers were randomized to one of the two strategies in a trial called GO TEST OVERTURE. In the T2T arm, commonly used therapies, such as allopurinol, benzbromarone, and febuxostat were employed to achieve and maintain a target sUA of < 0.36 mmol/L. In the T2S comparator arm, the same drugs were offered to control symptoms and prevent recurrences, but sUA levels were not used to guide treatment. 

The 1-year results of a planned 2-year study were presented in an oral abstract session at the annual European Congress of Rheumatology. For this analysis, outcomes were compared in the last 6 months prior to the 1-year data analysis. When assessed at 2 years, the comparison will again be made in the prior 6 months of the study.

For the primary endpoint of flares defined by the validated Gallo criteria, the mean rates were 1.3 for T2T and 1.85 for T2S (P < .001), Dr. Moses reported.

The reduced risk for flares correlated with the greater proportion of patients with sUA < 0.36 mmol/L. These proportions were 72% and 26% (P < .001) for the T2T and T2S groups, respectively. The mean sUA levels were 0.31 mmol/L and 0.42 mmol/L (P < .001), respectively.

At the 1-year mark, none of the secondary endpoints reached statistical significance. These included mean numeric rating pain scale (2.46 vs 2.41), the proportion of patients in remission (8% vs 5.7%), and the mean Health Assessment Questionnaire-Disability Index score (0.65 vs 0.62), according to Dr. Moses, who said all of these endpoints will continue to be followed in the planned second year of the study.

At baseline, there were no differences in any of the variables evaluated, including age (about 62.5 years in both groups), proportion of patients with a body mass index > 30 kg/m2 (about 62%), sUA (about 0.5 mmol/L), or estimated glomerular filtration rate (about 70 mL/min/1.73 m2).
 

Nonspecialists Should Heed the Results

According to Yael Klionsky, MD, a clinical assistant professor of rheumatology and immunology at Wake Forest University School of Medicine, Winston-Salem, North Carolina, these data are not a surprise. 

Even before this trial was completed, the message for clinicians is that they “should be focused on maintaining serum uric acid levels below physiological levels to improve outcomes” in patients with recurrent flares, said Klionsky, citing the validated EULAR and ACR guidelines.

While the ACP does still consider T2S acceptable as a strategy for chronic gout management, Klionsky pointed out that those guidelines have not been updated recently. Specialists in the treatment of gout do not need any more evidence that the T2T approach leads to better outcomes.

However, she agreed with the principle that non-rheumatologists need to be reached with better guidance in regard to gout management. While she expects the ACP to endorse T2T the next time their guidelines are updated, she hopes that primary care physicians recognize that T2T should now be a standard.

“In a 10- to 20-minute visit, managing multiple chronic conditions can be a challenge in primary care,” Klionsky said. “Many clinicians rely on guidelines so it is important to have consistent and accurate information.”

There is currently some distance between specialists and primary care physicians regarding the goals of gout management, according to a study that Klionsky presented at EULAR 2024. In a survey, nonspecialists and specialists did not perceive treatment priorities in the same way.

In this survey, which elicited responses from 151 rheumatologists, 150 nephrologists, and 102 primary care physicians, there was general agreement that preventing flares is a priority, but only 30% of primary care physicians and 35% of nephrologists vs 64% of rheumatologists identified the T2T target of < 0.36 mmol/L as a key step in reaching this goal.

Conversely, 58% of primary care physicians and 42% of nephrologists vs only 34% of rheumatologists considered absence of gout pain to be in the top three criteria.

In addition to the fact that primary care physicians differ from specialists in their goals for gout treatment, these data “highlight the need for the importance of a standardized definition of gout remission that includes serum uric acid control,” Dr. Klionsky said. She further thinks that this type of guidance should be disseminated to nonspecialists.

Dr. Moses reported no potential conflicts of interest. Dr. Klionsky reported financial relationships with Amgen, AstraZeneca, Eli Lilly, and MedIQ.

A version of this article appeared on Medscape.com.

UPDATED July 8, 2024 // Editor's note: This article has been revised to give a more nuanced view from Yael Klionsky, MD, about the need for more accurate and consistent gout management guidelines for busy primary care clinicians who often rely on them in clinical practice.

VIENNA — The first multicenter randomized trial in gout to compare treat-to-target (T2T) and treat for symptom avoidance (T2S) strategies has finally generated data to make the guideline-recommended practice of T2T evidence-based.

The T2T strategy may be guideline-endorsed, but it has never been validated, contended Anusha Moses, MSc, a researcher and PhD candidate at the University of Twente, Enschede, the Netherlands. She argued that this controlled trial fills an evidence gap.

T2T is defined as maintaining a serum uric acid (sUA) level below the physiologic threshold level of 36 mmol/L (< 6 mg/dL). T2S, in contrast, is a strategy of symptom control, typically basing therapy on suppression of symptoms independent of sUA, Dr. Moses explained. 

Ted Bosworth/Medscape Medical News

Both the American College of Rheumatology and European Alliance of Associations for Rheumatology (EULAR) have already endorsed T2T, but other organizations, such as the American College of Physicians (ACP), still accept symptom-based treatment in its gout clinical practice guideline, according to Dr. Moses.

The results of the trial were not surprising based on the pathophysiology of gout. Elevated sUA is considered the driver of both flares and the complications of gout. This well-established association led to endorsement of T2T in guidelines from organizations such as EULAR, but Dr. Moses said a controlled trial allows this to be declared as evidence based.

To provide proof that T2T is superior, 308 gout patients at eight centers were randomized to one of the two strategies in a trial called GO TEST OVERTURE. In the T2T arm, commonly used therapies, such as allopurinol, benzbromarone, and febuxostat were employed to achieve and maintain a target sUA of < 0.36 mmol/L. In the T2S comparator arm, the same drugs were offered to control symptoms and prevent recurrences, but sUA levels were not used to guide treatment. 

The 1-year results of a planned 2-year study were presented in an oral abstract session at the annual European Congress of Rheumatology. For this analysis, outcomes were compared in the last 6 months prior to the 1-year data analysis. When assessed at 2 years, the comparison will again be made in the prior 6 months of the study.

For the primary endpoint of flares defined by the validated Gallo criteria, the mean rates were 1.3 for T2T and 1.85 for T2S (P < .001), Dr. Moses reported.

The reduced risk for flares correlated with the greater proportion of patients with sUA < 0.36 mmol/L. These proportions were 72% and 26% (P < .001) for the T2T and T2S groups, respectively. The mean sUA levels were 0.31 mmol/L and 0.42 mmol/L (P < .001), respectively.

At the 1-year mark, none of the secondary endpoints reached statistical significance. These included mean numeric rating pain scale (2.46 vs 2.41), the proportion of patients in remission (8% vs 5.7%), and the mean Health Assessment Questionnaire-Disability Index score (0.65 vs 0.62), according to Dr. Moses, who said all of these endpoints will continue to be followed in the planned second year of the study.

At baseline, there were no differences in any of the variables evaluated, including age (about 62.5 years in both groups), proportion of patients with a body mass index > 30 kg/m2 (about 62%), sUA (about 0.5 mmol/L), or estimated glomerular filtration rate (about 70 mL/min/1.73 m2).
 

Nonspecialists Should Heed the Results

According to Yael Klionsky, MD, a clinical assistant professor of rheumatology and immunology at Wake Forest University School of Medicine, Winston-Salem, North Carolina, these data are not a surprise. 

Even before this trial was completed, the message for clinicians is that they “should be focused on maintaining serum uric acid levels below physiological levels to improve outcomes” in patients with recurrent flares, said Klionsky, citing the validated EULAR and ACR guidelines.

While the ACP does still consider T2S acceptable as a strategy for chronic gout management, Klionsky pointed out that those guidelines have not been updated recently. Specialists in the treatment of gout do not need any more evidence that the T2T approach leads to better outcomes.

However, she agreed with the principle that non-rheumatologists need to be reached with better guidance in regard to gout management. While she expects the ACP to endorse T2T the next time their guidelines are updated, she hopes that primary care physicians recognize that T2T should now be a standard.

“In a 10- to 20-minute visit, managing multiple chronic conditions can be a challenge in primary care,” Klionsky said. “Many clinicians rely on guidelines so it is important to have consistent and accurate information.”

There is currently some distance between specialists and primary care physicians regarding the goals of gout management, according to a study that Klionsky presented at EULAR 2024. In a survey, nonspecialists and specialists did not perceive treatment priorities in the same way.

In this survey, which elicited responses from 151 rheumatologists, 150 nephrologists, and 102 primary care physicians, there was general agreement that preventing flares is a priority, but only 30% of primary care physicians and 35% of nephrologists vs 64% of rheumatologists identified the T2T target of < 0.36 mmol/L as a key step in reaching this goal.

Conversely, 58% of primary care physicians and 42% of nephrologists vs only 34% of rheumatologists considered absence of gout pain to be in the top three criteria.

In addition to the fact that primary care physicians differ from specialists in their goals for gout treatment, these data “highlight the need for the importance of a standardized definition of gout remission that includes serum uric acid control,” Dr. Klionsky said. She further thinks that this type of guidance should be disseminated to nonspecialists.

Dr. Moses reported no potential conflicts of interest. Dr. Klionsky reported financial relationships with Amgen, AstraZeneca, Eli Lilly, and MedIQ.

A version of this article appeared on Medscape.com.

UPDATED July 8, 2024 // Editor's note: This article has been revised to give a more nuanced view from Yael Klionsky, MD, about the need for more accurate and consistent gout management guidelines for busy primary care clinicians who often rely on them in clinical practice.

VIENNA — The first multicenter randomized trial in gout to compare treat-to-target (T2T) and treat for symptom avoidance (T2S) strategies has finally generated data to make the guideline-recommended practice of T2T evidence-based.

The T2T strategy may be guideline-endorsed, but it has never been validated, contended Anusha Moses, MSc, a researcher and PhD candidate at the University of Twente, Enschede, the Netherlands. She argued that this controlled trial fills an evidence gap.

T2T is defined as maintaining a serum uric acid (sUA) level below the physiologic threshold level of 36 mmol/L (< 6 mg/dL). T2S, in contrast, is a strategy of symptom control, typically basing therapy on suppression of symptoms independent of sUA, Dr. Moses explained. 

Ted Bosworth/Medscape Medical News

Both the American College of Rheumatology and European Alliance of Associations for Rheumatology (EULAR) have already endorsed T2T, but other organizations, such as the American College of Physicians (ACP), still accept symptom-based treatment in its gout clinical practice guideline, according to Dr. Moses.

The results of the trial were not surprising based on the pathophysiology of gout. Elevated sUA is considered the driver of both flares and the complications of gout. This well-established association led to endorsement of T2T in guidelines from organizations such as EULAR, but Dr. Moses said a controlled trial allows this to be declared as evidence based.

To provide proof that T2T is superior, 308 gout patients at eight centers were randomized to one of the two strategies in a trial called GO TEST OVERTURE. In the T2T arm, commonly used therapies, such as allopurinol, benzbromarone, and febuxostat were employed to achieve and maintain a target sUA of < 0.36 mmol/L. In the T2S comparator arm, the same drugs were offered to control symptoms and prevent recurrences, but sUA levels were not used to guide treatment. 

The 1-year results of a planned 2-year study were presented in an oral abstract session at the annual European Congress of Rheumatology. For this analysis, outcomes were compared in the last 6 months prior to the 1-year data analysis. When assessed at 2 years, the comparison will again be made in the prior 6 months of the study.

For the primary endpoint of flares defined by the validated Gallo criteria, the mean rates were 1.3 for T2T and 1.85 for T2S (P < .001), Dr. Moses reported.

The reduced risk for flares correlated with the greater proportion of patients with sUA < 0.36 mmol/L. These proportions were 72% and 26% (P < .001) for the T2T and T2S groups, respectively. The mean sUA levels were 0.31 mmol/L and 0.42 mmol/L (P < .001), respectively.

At the 1-year mark, none of the secondary endpoints reached statistical significance. These included mean numeric rating pain scale (2.46 vs 2.41), the proportion of patients in remission (8% vs 5.7%), and the mean Health Assessment Questionnaire-Disability Index score (0.65 vs 0.62), according to Dr. Moses, who said all of these endpoints will continue to be followed in the planned second year of the study.

At baseline, there were no differences in any of the variables evaluated, including age (about 62.5 years in both groups), proportion of patients with a body mass index > 30 kg/m2 (about 62%), sUA (about 0.5 mmol/L), or estimated glomerular filtration rate (about 70 mL/min/1.73 m2).
 

Nonspecialists Should Heed the Results

According to Yael Klionsky, MD, a clinical assistant professor of rheumatology and immunology at Wake Forest University School of Medicine, Winston-Salem, North Carolina, these data are not a surprise. 

Even before this trial was completed, the message for clinicians is that they “should be focused on maintaining serum uric acid levels below physiological levels to improve outcomes” in patients with recurrent flares, said Klionsky, citing the validated EULAR and ACR guidelines.

While the ACP does still consider T2S acceptable as a strategy for chronic gout management, Klionsky pointed out that those guidelines have not been updated recently. Specialists in the treatment of gout do not need any more evidence that the T2T approach leads to better outcomes.

However, she agreed with the principle that non-rheumatologists need to be reached with better guidance in regard to gout management. While she expects the ACP to endorse T2T the next time their guidelines are updated, she hopes that primary care physicians recognize that T2T should now be a standard.

“In a 10- to 20-minute visit, managing multiple chronic conditions can be a challenge in primary care,” Klionsky said. “Many clinicians rely on guidelines so it is important to have consistent and accurate information.”

There is currently some distance between specialists and primary care physicians regarding the goals of gout management, according to a study that Klionsky presented at EULAR 2024. In a survey, nonspecialists and specialists did not perceive treatment priorities in the same way.

In this survey, which elicited responses from 151 rheumatologists, 150 nephrologists, and 102 primary care physicians, there was general agreement that preventing flares is a priority, but only 30% of primary care physicians and 35% of nephrologists vs 64% of rheumatologists identified the T2T target of < 0.36 mmol/L as a key step in reaching this goal.

Conversely, 58% of primary care physicians and 42% of nephrologists vs only 34% of rheumatologists considered absence of gout pain to be in the top three criteria.

In addition to the fact that primary care physicians differ from specialists in their goals for gout treatment, these data “highlight the need for the importance of a standardized definition of gout remission that includes serum uric acid control,” Dr. Klionsky said. She further thinks that this type of guidance should be disseminated to nonspecialists.

Dr. Moses reported no potential conflicts of interest. Dr. Klionsky reported financial relationships with Amgen, AstraZeneca, Eli Lilly, and MedIQ.

A version of this article appeared on Medscape.com.

CHICAGO — One of the more recent developments in sunscreen technology is the addition of iron oxide to mineral sunscreens.

Iron oxide is “an excellent pigment” that absorbs and blocks visible light, which is particularly important in individuals with Fitzpatrick skin types III-VI, Zoe D. Draelos, MD, consulting professor of dermatology at Duke University, Durham, North Carolina, said at the Pigmentation Disorders Exchange symposium.

Susan C. Taylor, MD, professor of dermatology at the University of Pennsylvania, Philadelphia, who spoke at the conference, also recommended tinted sunscreen with iron oxide for patients with skin of color. “It still needs to be broad spectrum,” she said, “and at least an SPF [Sun Protection Factor] 30.”

When blended with mineral sunscreens, iron oxide can reduce transmission of visible light by 90% and can protect patients from hyperpigmentation. Iron oxide comes in different colors blended together for various degrees of tinting.

Dr. Taylor noted that iron oxide is listed under the inactive ingredients. “The literature indicates a 3% concentration to aim for, but we don’t know the concentration in most of the products,” she added.

During her presentation, Dr. Draelos noted that inorganic sunscreens, such as zinc oxide and titanium oxides, are highly effective but make the skin white and pasty. To address this issue, many companies are now grinding these materials into such small particles that they are transparent.

“That’s great, except the smaller the particle is, the less UV [ultraviolet] radiation it reflects and that lowers the [SPF],” she said.

In addition to providing photoprotection, sunscreens in general provide protection from nanoparticles in tobacco and combustion, such as traffic exhaust, which can harm skin over time. “Moisturizers and sunscreens are the best way to protect against pollution and tobacco nanoparticle damage, which can contribute to inflammation,” she noted. They create a film over the skin and trap the nanoparticles.
 

Start the Patient Visit With a Photoprotection Talk

At the meeting, Dr. Taylor recommended that for all patients with hypopigmentation and hyperpigmentation disorders, “treatment really begins with photoprotection.”

She acknowledged that photoprotection discussions, including the basics of seeking shade, wearing protective clothing, and avoiding midday sun, often come at the end of the patient visit but she urged dermatologists to make that the first topic instead.

Dr. Taylor said a question often asked of patients of color about prolonged sun exposure — whether their skin turns bright red after too much sun — may get a negative reply. The better question is whether the patient has experienced tender skin after too much sun — which can signify a sunburn, she said.

Dr. Draelos reported no relevant financial relationships. Dr. Taylor reported financial relationships and grant support from multiple pharmaceutical companies.

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

CHICAGO — One of the more recent developments in sunscreen technology is the addition of iron oxide to mineral sunscreens.

Iron oxide is “an excellent pigment” that absorbs and blocks visible light, which is particularly important in individuals with Fitzpatrick skin types III-VI, Zoe D. Draelos, MD, consulting professor of dermatology at Duke University, Durham, North Carolina, said at the Pigmentation Disorders Exchange symposium.

Susan C. Taylor, MD, professor of dermatology at the University of Pennsylvania, Philadelphia, who spoke at the conference, also recommended tinted sunscreen with iron oxide for patients with skin of color. “It still needs to be broad spectrum,” she said, “and at least an SPF [Sun Protection Factor] 30.”

When blended with mineral sunscreens, iron oxide can reduce transmission of visible light by 90% and can protect patients from hyperpigmentation. Iron oxide comes in different colors blended together for various degrees of tinting.

Dr. Taylor noted that iron oxide is listed under the inactive ingredients. “The literature indicates a 3% concentration to aim for, but we don’t know the concentration in most of the products,” she added.

During her presentation, Dr. Draelos noted that inorganic sunscreens, such as zinc oxide and titanium oxides, are highly effective but make the skin white and pasty. To address this issue, many companies are now grinding these materials into such small particles that they are transparent.

“That’s great, except the smaller the particle is, the less UV [ultraviolet] radiation it reflects and that lowers the [SPF],” she said.

In addition to providing photoprotection, sunscreens in general provide protection from nanoparticles in tobacco and combustion, such as traffic exhaust, which can harm skin over time. “Moisturizers and sunscreens are the best way to protect against pollution and tobacco nanoparticle damage, which can contribute to inflammation,” she noted. They create a film over the skin and trap the nanoparticles.
 

Start the Patient Visit With a Photoprotection Talk

At the meeting, Dr. Taylor recommended that for all patients with hypopigmentation and hyperpigmentation disorders, “treatment really begins with photoprotection.”

She acknowledged that photoprotection discussions, including the basics of seeking shade, wearing protective clothing, and avoiding midday sun, often come at the end of the patient visit but she urged dermatologists to make that the first topic instead.

Dr. Taylor said a question often asked of patients of color about prolonged sun exposure — whether their skin turns bright red after too much sun — may get a negative reply. The better question is whether the patient has experienced tender skin after too much sun — which can signify a sunburn, she said.

Dr. Draelos reported no relevant financial relationships. Dr. Taylor reported financial relationships and grant support from multiple pharmaceutical companies.

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

CHICAGO — One of the more recent developments in sunscreen technology is the addition of iron oxide to mineral sunscreens.

Iron oxide is “an excellent pigment” that absorbs and blocks visible light, which is particularly important in individuals with Fitzpatrick skin types III-VI, Zoe D. Draelos, MD, consulting professor of dermatology at Duke University, Durham, North Carolina, said at the Pigmentation Disorders Exchange symposium.

Susan C. Taylor, MD, professor of dermatology at the University of Pennsylvania, Philadelphia, who spoke at the conference, also recommended tinted sunscreen with iron oxide for patients with skin of color. “It still needs to be broad spectrum,” she said, “and at least an SPF [Sun Protection Factor] 30.”

When blended with mineral sunscreens, iron oxide can reduce transmission of visible light by 90% and can protect patients from hyperpigmentation. Iron oxide comes in different colors blended together for various degrees of tinting.

Dr. Taylor noted that iron oxide is listed under the inactive ingredients. “The literature indicates a 3% concentration to aim for, but we don’t know the concentration in most of the products,” she added.

During her presentation, Dr. Draelos noted that inorganic sunscreens, such as zinc oxide and titanium oxides, are highly effective but make the skin white and pasty. To address this issue, many companies are now grinding these materials into such small particles that they are transparent.

“That’s great, except the smaller the particle is, the less UV [ultraviolet] radiation it reflects and that lowers the [SPF],” she said.

In addition to providing photoprotection, sunscreens in general provide protection from nanoparticles in tobacco and combustion, such as traffic exhaust, which can harm skin over time. “Moisturizers and sunscreens are the best way to protect against pollution and tobacco nanoparticle damage, which can contribute to inflammation,” she noted. They create a film over the skin and trap the nanoparticles.
 

Start the Patient Visit With a Photoprotection Talk

At the meeting, Dr. Taylor recommended that for all patients with hypopigmentation and hyperpigmentation disorders, “treatment really begins with photoprotection.”

She acknowledged that photoprotection discussions, including the basics of seeking shade, wearing protective clothing, and avoiding midday sun, often come at the end of the patient visit but she urged dermatologists to make that the first topic instead.

Dr. Taylor said a question often asked of patients of color about prolonged sun exposure — whether their skin turns bright red after too much sun — may get a negative reply. The better question is whether the patient has experienced tender skin after too much sun — which can signify a sunburn, she said.

Dr. Draelos reported no relevant financial relationships. Dr. Taylor reported financial relationships and grant support from multiple pharmaceutical companies.

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

We recently shared a clinical case drawn from a family medicine practice about the effect of adverse childhood experiences (ACEs) on health. The widespread epidemiology and significant health consequences require a focus on the prevention and management of ACEs. 
 

The Centers for Disease Control and Prevention published an important monograph on ACEs in 2019. Although it is evidence based, most of the interventions recommended to reduce ACEs and their sequelae are larger policy and public health efforts that go well beyond the clinician’s office. Important highlights from these recommended strategies to reduce ACEs include:

• Strengthen economic support for families through policies such as the earned income tax credit and child tax credit.
• Establish routine parental work/shift times to optimize cognitive outcomes in children.
• Promote social norms for healthy families through public health campaigns and legislative efforts to reduce corporal punishment of children. Bystander training that targets boys and men has also proven effective in reducing sexual violence.
• Facilitate early in-home visitation for at-risk families as well as high-quality childcare.
• Employ social-emotional learning approaches for children and adolescents, which can improve aggressive or violent behavior, rates of substance use, and academic success.
• Connect youth to after-school programs featuring caring adults.

But clinicians still play a vital role in the prevention and management of ACEs among their patients. Akin to gathering a patient’s past medical history or family history is initiating universal ACE screening in practice and exploring related topics in conversation.

The ACEs Aware initiative in California provides a comprehensive ACE screening clinical workflow to help implement these conversations in practice, including the assessment of associated health conditions and their appropriate clinical follow-up. While it is encouraged to universally screen patients, the key screenings to prioritize for the pediatric population are “parental depression, severe stress, unhealthy drug use, domestic violence, harsh punishment, [and] food insecurity.” Moreover, a systematic review by Steen and colleagues shared insight into newer interpretations of ACE screening which relate trauma to “[...] community violence, poverty, housing instability, structural racism, environmental blight, and climate change.” 

These exposures are now being investigated for a connection to the toxic stress response. In the long term, this genetic regulatory mechanism can be affected by “high doses of cumulative adversity experienced during critical and sensitive periods of early life development — without the buffering protections of trusted, nurturing caregivers and safe, stable environments.” This micro and macro lens fosters a deeper clinician understanding of a patient’s trauma origin and can better guide appropriate clinical follow-up. 

ACE-associated health conditions can be neurologic, endocrine, metabolic, or immune system–related. Early diagnosis and treatment of these conditions can help prevent long-term health care complications, costly for both patient and the health care system. 

After the initial clinical assessment, physicians can educate patients about the ways that ACE-associated health conditions are a consequence of toxic stress exposure. From there, physicians should rely on a broader integrated health team, within the health system and the community, to offer clinical interventions and services to mitigate patients’ toxic stress. The ACEs Aware Stress Buster wheel highlights seven targets to strategize stress regulation. This wheel can be used to identify existing protective factors for patients and track treatment progress, which may buffer the negative impact of stressors and contribute to health and resilience. 

The burden of universal screenings in primary care is high. Without ACE screening, however, the opportunity to address downstream health effects from toxic stress may be lost. Dubowitz and colleagues suggest ways to successfully incorporate ACE screenings in clinical workflow:

• Utilize technology to implement a streamlined referral processing/tracking system.
• Train clinicians to respond competently to positive ACE screens.
• Gather in-network and community-based resources for patients.

In addition, prioritize screening for families with children younger than 6 years of age to begin interventions as early as possible. Primary care clinicians have the unique opportunity to provide appropriate intervention over continual care. An intervention as simple as encouraging pediatric patient involvement in after-school programs may mitigate toxic stress and prevent the development of an ACE-associated health condition. 

Dr. Vega, Health Sciences Clinical Professor, Family Medicine, University of California, Irvine, disclosed ties with McNeil Pharmaceuticals. Alejandra Hurtado, MD candidate, University of California, Irvine School of Medicine, has disclosed no relevant financial relationships.

A version of this article appeared on Medscape.com.

We recently shared a clinical case drawn from a family medicine practice about the effect of adverse childhood experiences (ACEs) on health. The widespread epidemiology and significant health consequences require a focus on the prevention and management of ACEs. 
 

The Centers for Disease Control and Prevention published an important monograph on ACEs in 2019. Although it is evidence based, most of the interventions recommended to reduce ACEs and their sequelae are larger policy and public health efforts that go well beyond the clinician’s office. Important highlights from these recommended strategies to reduce ACEs include:

• Strengthen economic support for families through policies such as the earned income tax credit and child tax credit.
• Establish routine parental work/shift times to optimize cognitive outcomes in children.
• Promote social norms for healthy families through public health campaigns and legislative efforts to reduce corporal punishment of children. Bystander training that targets boys and men has also proven effective in reducing sexual violence.
• Facilitate early in-home visitation for at-risk families as well as high-quality childcare.
• Employ social-emotional learning approaches for children and adolescents, which can improve aggressive or violent behavior, rates of substance use, and academic success.
• Connect youth to after-school programs featuring caring adults.

But clinicians still play a vital role in the prevention and management of ACEs among their patients. Akin to gathering a patient’s past medical history or family history is initiating universal ACE screening in practice and exploring related topics in conversation.

The ACEs Aware initiative in California provides a comprehensive ACE screening clinical workflow to help implement these conversations in practice, including the assessment of associated health conditions and their appropriate clinical follow-up. While it is encouraged to universally screen patients, the key screenings to prioritize for the pediatric population are “parental depression, severe stress, unhealthy drug use, domestic violence, harsh punishment, [and] food insecurity.” Moreover, a systematic review by Steen and colleagues shared insight into newer interpretations of ACE screening which relate trauma to “[...] community violence, poverty, housing instability, structural racism, environmental blight, and climate change.” 

These exposures are now being investigated for a connection to the toxic stress response. In the long term, this genetic regulatory mechanism can be affected by “high doses of cumulative adversity experienced during critical and sensitive periods of early life development — without the buffering protections of trusted, nurturing caregivers and safe, stable environments.” This micro and macro lens fosters a deeper clinician understanding of a patient’s trauma origin and can better guide appropriate clinical follow-up. 

ACE-associated health conditions can be neurologic, endocrine, metabolic, or immune system–related. Early diagnosis and treatment of these conditions can help prevent long-term health care complications, costly for both patient and the health care system. 

After the initial clinical assessment, physicians can educate patients about the ways that ACE-associated health conditions are a consequence of toxic stress exposure. From there, physicians should rely on a broader integrated health team, within the health system and the community, to offer clinical interventions and services to mitigate patients’ toxic stress. The ACEs Aware Stress Buster wheel highlights seven targets to strategize stress regulation. This wheel can be used to identify existing protective factors for patients and track treatment progress, which may buffer the negative impact of stressors and contribute to health and resilience. 

The burden of universal screenings in primary care is high. Without ACE screening, however, the opportunity to address downstream health effects from toxic stress may be lost. Dubowitz and colleagues suggest ways to successfully incorporate ACE screenings in clinical workflow:

• Utilize technology to implement a streamlined referral processing/tracking system.
• Train clinicians to respond competently to positive ACE screens.
• Gather in-network and community-based resources for patients.

In addition, prioritize screening for families with children younger than 6 years of age to begin interventions as early as possible. Primary care clinicians have the unique opportunity to provide appropriate intervention over continual care. An intervention as simple as encouraging pediatric patient involvement in after-school programs may mitigate toxic stress and prevent the development of an ACE-associated health condition. 

Dr. Vega, Health Sciences Clinical Professor, Family Medicine, University of California, Irvine, disclosed ties with McNeil Pharmaceuticals. Alejandra Hurtado, MD candidate, University of California, Irvine School of Medicine, has disclosed no relevant financial relationships.

A version of this article appeared on Medscape.com.

We recently shared a clinical case drawn from a family medicine practice about the effect of adverse childhood experiences (ACEs) on health. The widespread epidemiology and significant health consequences require a focus on the prevention and management of ACEs. 
 

The Centers for Disease Control and Prevention published an important monograph on ACEs in 2019. Although it is evidence based, most of the interventions recommended to reduce ACEs and their sequelae are larger policy and public health efforts that go well beyond the clinician’s office. Important highlights from these recommended strategies to reduce ACEs include:

• Strengthen economic support for families through policies such as the earned income tax credit and child tax credit.
• Establish routine parental work/shift times to optimize cognitive outcomes in children.
• Promote social norms for healthy families through public health campaigns and legislative efforts to reduce corporal punishment of children. Bystander training that targets boys and men has also proven effective in reducing sexual violence.
• Facilitate early in-home visitation for at-risk families as well as high-quality childcare.
• Employ social-emotional learning approaches for children and adolescents, which can improve aggressive or violent behavior, rates of substance use, and academic success.
• Connect youth to after-school programs featuring caring adults.

But clinicians still play a vital role in the prevention and management of ACEs among their patients. Akin to gathering a patient’s past medical history or family history is initiating universal ACE screening in practice and exploring related topics in conversation.

The ACEs Aware initiative in California provides a comprehensive ACE screening clinical workflow to help implement these conversations in practice, including the assessment of associated health conditions and their appropriate clinical follow-up. While it is encouraged to universally screen patients, the key screenings to prioritize for the pediatric population are “parental depression, severe stress, unhealthy drug use, domestic violence, harsh punishment, [and] food insecurity.” Moreover, a systematic review by Steen and colleagues shared insight into newer interpretations of ACE screening which relate trauma to “[...] community violence, poverty, housing instability, structural racism, environmental blight, and climate change.” 

These exposures are now being investigated for a connection to the toxic stress response. In the long term, this genetic regulatory mechanism can be affected by “high doses of cumulative adversity experienced during critical and sensitive periods of early life development — without the buffering protections of trusted, nurturing caregivers and safe, stable environments.” This micro and macro lens fosters a deeper clinician understanding of a patient’s trauma origin and can better guide appropriate clinical follow-up. 

ACE-associated health conditions can be neurologic, endocrine, metabolic, or immune system–related. Early diagnosis and treatment of these conditions can help prevent long-term health care complications, costly for both patient and the health care system. 

After the initial clinical assessment, physicians can educate patients about the ways that ACE-associated health conditions are a consequence of toxic stress exposure. From there, physicians should rely on a broader integrated health team, within the health system and the community, to offer clinical interventions and services to mitigate patients’ toxic stress. The ACEs Aware Stress Buster wheel highlights seven targets to strategize stress regulation. This wheel can be used to identify existing protective factors for patients and track treatment progress, which may buffer the negative impact of stressors and contribute to health and resilience. 

The burden of universal screenings in primary care is high. Without ACE screening, however, the opportunity to address downstream health effects from toxic stress may be lost. Dubowitz and colleagues suggest ways to successfully incorporate ACE screenings in clinical workflow:

• Utilize technology to implement a streamlined referral processing/tracking system.
• Train clinicians to respond competently to positive ACE screens.
• Gather in-network and community-based resources for patients.

In addition, prioritize screening for families with children younger than 6 years of age to begin interventions as early as possible. Primary care clinicians have the unique opportunity to provide appropriate intervention over continual care. An intervention as simple as encouraging pediatric patient involvement in after-school programs may mitigate toxic stress and prevent the development of an ACE-associated health condition. 

Dr. Vega, Health Sciences Clinical Professor, Family Medicine, University of California, Irvine, disclosed ties with McNeil Pharmaceuticals. Alejandra Hurtado, MD candidate, University of California, Irvine School of Medicine, has disclosed no relevant financial relationships.

A version of this article appeared on Medscape.com.

BOSTON — Patients who have had a stroke are thought to be at a higher risk for another one, but oral anticoagulation with edoxaban led to no discernible reduction in the risk for a second stroke, and the risk for major bleeding was more than quadruple the risk with no anticoagulation, a subanalysis of a major European trial has shown.

“There is no interaction between prior stroke or TIA [transient ischemic attack] and the treatment effect, and this is true for the primary outcome and the safety outcome,” Paulus Kirchoff, MD, director of cardiology at the University Heart and Vascular Center in Hamburg, Germany, said during his presentation of a subanalysis of the NOAH-AFNET 6 trial at the annual meeting of the Heart Rhythm Society (HRS) 2024. However, “there is a signal for more safety events in patients randomized to anticoagulation with a prior stroke.”

The subanalysis involved 253 patients who had had a stroke or TIA and who had device-detected atrial fibrillation (AF) from the overall NOAH-AFNET 6 population of 2536 patients, which enrolled patients 65 years and older with at least one additional CHA2DS-VASc risk factor and patients 75 years and older with device-detected subclinical AF episodes of at least 6 minutes. Patients were randomized to either edoxaban or no anticoagulation, but 53.9% of the no-anticoagulation group was taking aspirin at trial enrollment. Anticoagulation with edoxaban was shown to have no significant impact on stroke rates or other cardiovascular outcomes.
 

Subanalysis Results

In the subanalysis, a composite of stroke, systemic embolism, and cardiovascular death — the primary outcome — was similar in the edoxaban and no-anticoagulation groups (14/122 patients [11.5%] vs 16/131 patients [12.2%]; 5.7% vs 6.3% per patient-year).

The rate of recurrent stroke was also similar in the edoxaban and no-anticoagulation groups (4 of 122 patients [3.3%] vs 6 of 131 patients [4.6%]; 1.6% vs 2.3% per patient-year). And there were eight cardiovascular deaths in each group.

However, edoxaban patients had significantly higher rates of major bleeding.

“This is a subanalysis, so what we see in terms of the number of patients with events is not powered for a definitive answer, but we do see that there were 10 major bleeds in the group of patients with a prior stroke or TIA in NOAH,” Dr. Kirchoff reported. “Eight of those 10 major bleeds occurred in patients randomized to edoxaban.”

Results from the NOAH-AFNET 6 trial have been compared with those from the ARTESiA trial, which compared apixaban anticoagulation with aspirin in patients with subclinical AF and was also presented at HRS 2024. ARTESiA showed that apixaban significantly lowered the risk for stroke and systemic embolism.

“In ARTESiA, everyone was on aspirin when they were randomized to no anticoagulation; in NOAH, only about half were on aspirin,” Dr. Kirchoff said.

Both studies had similar outcomes for cardiovascular death in the anticoagulation and no-anticoagulation groups. “It’s not significant; it may be chance, but it’s definitely not the reduction in death that we have seen in the anticoagulant trials,” Dr. Kirchoff said. “When you look at the meta-analyses of the early anticoagulation trials, there’s a one third reduction in death, and here we’re talking about a smaller reduction.”

This research points to a need for a better way to evaluate stroke risk. “We need new markers,” Dr. Kirchoff said. “Some of them may be in the blood or imaging, genetics maybe, and one thing that really emerges from my perspective is that we now have the first evidence to suggest that patients with a very low atrial fibrillation burden have a low stroke rate.”

More research is needed to better understand AF characteristics and stroke risk, he said.
 

AF Care Enters a ‘Gray Zone’

The NOAH-AFNET 6 results, coupled with those from ARTESiA, are changing the paradigm for anticoagulation in patients with stroke, said Taya Glotzer, MD, an electrophysiologist at the Hackensack University Medical Center in Hackensack, New Jersey, who compiled her own analysis of the studies’ outcomes.

“In ARTESiA, the stroke reduction was only 0.44% a year, with a number needed to treat of 250,” she said. “In the NOAH-AFNET 6 main trial, the stroke reduction was 0.2%, with the number needed to treat of 500, and in the NOAH prior stroke patients, there was a 0.7% reduction, with a number needed to treat of 143.”

None of these trials would meet the standard for a class 1 recommendation for anticoagulation with a reduction of even 1%-2% per year, she noted, but they do show that the stroke rate “is very, very low” in prior patients with stroke.

“Prior to 2024, we knew what was black and white; we knew who to anticoagulate and who not to anticoagulate. And now we are in a gray zone, trying to balance the risk of stroke and bleeding. We have to individualize or hope for substudies, perhaps using the CHA2DS-VASc score or other information about the left atrium, to help us make decisions in these patients. It’s not just going to be black and white,” she said.

Dr. Kirchoff had no relevant financial relationships to disclose. Dr. Glotzer disclosed financial relationships with Medtronic, Abbott, Boston Scientific, and MediaSphere Medical.

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

BOSTON — Patients who have had a stroke are thought to be at a higher risk for another one, but oral anticoagulation with edoxaban led to no discernible reduction in the risk for a second stroke, and the risk for major bleeding was more than quadruple the risk with no anticoagulation, a subanalysis of a major European trial has shown.

“There is no interaction between prior stroke or TIA [transient ischemic attack] and the treatment effect, and this is true for the primary outcome and the safety outcome,” Paulus Kirchoff, MD, director of cardiology at the University Heart and Vascular Center in Hamburg, Germany, said during his presentation of a subanalysis of the NOAH-AFNET 6 trial at the annual meeting of the Heart Rhythm Society (HRS) 2024. However, “there is a signal for more safety events in patients randomized to anticoagulation with a prior stroke.”

The subanalysis involved 253 patients who had had a stroke or TIA and who had device-detected atrial fibrillation (AF) from the overall NOAH-AFNET 6 population of 2536 patients, which enrolled patients 65 years and older with at least one additional CHA2DS-VASc risk factor and patients 75 years and older with device-detected subclinical AF episodes of at least 6 minutes. Patients were randomized to either edoxaban or no anticoagulation, but 53.9% of the no-anticoagulation group was taking aspirin at trial enrollment. Anticoagulation with edoxaban was shown to have no significant impact on stroke rates or other cardiovascular outcomes.
 

Subanalysis Results

In the subanalysis, a composite of stroke, systemic embolism, and cardiovascular death — the primary outcome — was similar in the edoxaban and no-anticoagulation groups (14/122 patients [11.5%] vs 16/131 patients [12.2%]; 5.7% vs 6.3% per patient-year).

The rate of recurrent stroke was also similar in the edoxaban and no-anticoagulation groups (4 of 122 patients [3.3%] vs 6 of 131 patients [4.6%]; 1.6% vs 2.3% per patient-year). And there were eight cardiovascular deaths in each group.

However, edoxaban patients had significantly higher rates of major bleeding.

“This is a subanalysis, so what we see in terms of the number of patients with events is not powered for a definitive answer, but we do see that there were 10 major bleeds in the group of patients with a prior stroke or TIA in NOAH,” Dr. Kirchoff reported. “Eight of those 10 major bleeds occurred in patients randomized to edoxaban.”

Results from the NOAH-AFNET 6 trial have been compared with those from the ARTESiA trial, which compared apixaban anticoagulation with aspirin in patients with subclinical AF and was also presented at HRS 2024. ARTESiA showed that apixaban significantly lowered the risk for stroke and systemic embolism.

“In ARTESiA, everyone was on aspirin when they were randomized to no anticoagulation; in NOAH, only about half were on aspirin,” Dr. Kirchoff said.

Both studies had similar outcomes for cardiovascular death in the anticoagulation and no-anticoagulation groups. “It’s not significant; it may be chance, but it’s definitely not the reduction in death that we have seen in the anticoagulant trials,” Dr. Kirchoff said. “When you look at the meta-analyses of the early anticoagulation trials, there’s a one third reduction in death, and here we’re talking about a smaller reduction.”

This research points to a need for a better way to evaluate stroke risk. “We need new markers,” Dr. Kirchoff said. “Some of them may be in the blood or imaging, genetics maybe, and one thing that really emerges from my perspective is that we now have the first evidence to suggest that patients with a very low atrial fibrillation burden have a low stroke rate.”

More research is needed to better understand AF characteristics and stroke risk, he said.
 

AF Care Enters a ‘Gray Zone’

The NOAH-AFNET 6 results, coupled with those from ARTESiA, are changing the paradigm for anticoagulation in patients with stroke, said Taya Glotzer, MD, an electrophysiologist at the Hackensack University Medical Center in Hackensack, New Jersey, who compiled her own analysis of the studies’ outcomes.

“In ARTESiA, the stroke reduction was only 0.44% a year, with a number needed to treat of 250,” she said. “In the NOAH-AFNET 6 main trial, the stroke reduction was 0.2%, with the number needed to treat of 500, and in the NOAH prior stroke patients, there was a 0.7% reduction, with a number needed to treat of 143.”

None of these trials would meet the standard for a class 1 recommendation for anticoagulation with a reduction of even 1%-2% per year, she noted, but they do show that the stroke rate “is very, very low” in prior patients with stroke.

“Prior to 2024, we knew what was black and white; we knew who to anticoagulate and who not to anticoagulate. And now we are in a gray zone, trying to balance the risk of stroke and bleeding. We have to individualize or hope for substudies, perhaps using the CHA2DS-VASc score or other information about the left atrium, to help us make decisions in these patients. It’s not just going to be black and white,” she said.

Dr. Kirchoff had no relevant financial relationships to disclose. Dr. Glotzer disclosed financial relationships with Medtronic, Abbott, Boston Scientific, and MediaSphere Medical.

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

BOSTON — Patients who have had a stroke are thought to be at a higher risk for another one, but oral anticoagulation with edoxaban led to no discernible reduction in the risk for a second stroke, and the risk for major bleeding was more than quadruple the risk with no anticoagulation, a subanalysis of a major European trial has shown.

“There is no interaction between prior stroke or TIA [transient ischemic attack] and the treatment effect, and this is true for the primary outcome and the safety outcome,” Paulus Kirchoff, MD, director of cardiology at the University Heart and Vascular Center in Hamburg, Germany, said during his presentation of a subanalysis of the NOAH-AFNET 6 trial at the annual meeting of the Heart Rhythm Society (HRS) 2024. However, “there is a signal for more safety events in patients randomized to anticoagulation with a prior stroke.”

The subanalysis involved 253 patients who had had a stroke or TIA and who had device-detected atrial fibrillation (AF) from the overall NOAH-AFNET 6 population of 2536 patients, which enrolled patients 65 years and older with at least one additional CHA2DS-VASc risk factor and patients 75 years and older with device-detected subclinical AF episodes of at least 6 minutes. Patients were randomized to either edoxaban or no anticoagulation, but 53.9% of the no-anticoagulation group was taking aspirin at trial enrollment. Anticoagulation with edoxaban was shown to have no significant impact on stroke rates or other cardiovascular outcomes.
 

Subanalysis Results

In the subanalysis, a composite of stroke, systemic embolism, and cardiovascular death — the primary outcome — was similar in the edoxaban and no-anticoagulation groups (14/122 patients [11.5%] vs 16/131 patients [12.2%]; 5.7% vs 6.3% per patient-year).

The rate of recurrent stroke was also similar in the edoxaban and no-anticoagulation groups (4 of 122 patients [3.3%] vs 6 of 131 patients [4.6%]; 1.6% vs 2.3% per patient-year). And there were eight cardiovascular deaths in each group.

However, edoxaban patients had significantly higher rates of major bleeding.

“This is a subanalysis, so what we see in terms of the number of patients with events is not powered for a definitive answer, but we do see that there were 10 major bleeds in the group of patients with a prior stroke or TIA in NOAH,” Dr. Kirchoff reported. “Eight of those 10 major bleeds occurred in patients randomized to edoxaban.”

Results from the NOAH-AFNET 6 trial have been compared with those from the ARTESiA trial, which compared apixaban anticoagulation with aspirin in patients with subclinical AF and was also presented at HRS 2024. ARTESiA showed that apixaban significantly lowered the risk for stroke and systemic embolism.

“In ARTESiA, everyone was on aspirin when they were randomized to no anticoagulation; in NOAH, only about half were on aspirin,” Dr. Kirchoff said.

Both studies had similar outcomes for cardiovascular death in the anticoagulation and no-anticoagulation groups. “It’s not significant; it may be chance, but it’s definitely not the reduction in death that we have seen in the anticoagulant trials,” Dr. Kirchoff said. “When you look at the meta-analyses of the early anticoagulation trials, there’s a one third reduction in death, and here we’re talking about a smaller reduction.”

This research points to a need for a better way to evaluate stroke risk. “We need new markers,” Dr. Kirchoff said. “Some of them may be in the blood or imaging, genetics maybe, and one thing that really emerges from my perspective is that we now have the first evidence to suggest that patients with a very low atrial fibrillation burden have a low stroke rate.”

More research is needed to better understand AF characteristics and stroke risk, he said.
 

AF Care Enters a ‘Gray Zone’

The NOAH-AFNET 6 results, coupled with those from ARTESiA, are changing the paradigm for anticoagulation in patients with stroke, said Taya Glotzer, MD, an electrophysiologist at the Hackensack University Medical Center in Hackensack, New Jersey, who compiled her own analysis of the studies’ outcomes.

“In ARTESiA, the stroke reduction was only 0.44% a year, with a number needed to treat of 250,” she said. “In the NOAH-AFNET 6 main trial, the stroke reduction was 0.2%, with the number needed to treat of 500, and in the NOAH prior stroke patients, there was a 0.7% reduction, with a number needed to treat of 143.”

None of these trials would meet the standard for a class 1 recommendation for anticoagulation with a reduction of even 1%-2% per year, she noted, but they do show that the stroke rate “is very, very low” in prior patients with stroke.

“Prior to 2024, we knew what was black and white; we knew who to anticoagulate and who not to anticoagulate. And now we are in a gray zone, trying to balance the risk of stroke and bleeding. We have to individualize or hope for substudies, perhaps using the CHA2DS-VASc score or other information about the left atrium, to help us make decisions in these patients. It’s not just going to be black and white,” she said.

Dr. Kirchoff had no relevant financial relationships to disclose. Dr. Glotzer disclosed financial relationships with Medtronic, Abbott, Boston Scientific, and MediaSphere Medical.

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

Most doctors mind their manners. But surgeons are the most likely to be reported for unprofessional behavior, while physicians practicing in pediatric settings are the least likely, according to a recent study of more than 35,000 physicians.

The research, published on June 6 in JAMA Network Open, found that fewer than 10% of physicians were reported by their coworkers for at least one instance of unprofessional behavior, and only 1% showed a pattern of such reports.

Data were gathered from the Center for Patient and Professional Advocacy’s (CPPA’s) Coworker Observation Reporting System (CORS) program, a national collaborative in which 193 participating hospitals and practice sites file safety-event reports involving medical workers’ unprofessional behaviors. An algorithm that weights CORS reports based on recency and severity was used to analyze the data. The study was spearheaded by William O. Cooper, MD, MPH, director of the CPPA at Vanderbilt University Medical Center, Nashville, Tennessee.

The retrospective cohort study included deidentified data on credentialed physicians, not including residents or fellows, who practiced at a CORS site between 2018 and 2022.
 

Why Surgeons?

The authors speculated that the reason surgeons were reported for unprofessional behavior more often than their colleagues in nonsurgical specialties was because surgery is a more stressful environment than other specialties and requires more teamwork, resulting in more interactions during high-stakes events.

Daniel Katz, MD, professor and vice chair of education for the Department of Anesthesiology, Perioperative and Pain Medicine at the Icahn School of Medicine at Mount Sinai, New York City, added that part of the problem is that surgeons are expected to perform at very high levels all the time.

“When things that are outside the control of the surgeon don’t go well,” Dr. Katz said, “that can lead to increased frustration and negative emotions, which will then bring out these kinds of behaviors.”
 

Types of Unprofessional Behaviors

The most common out-of-bounds behaviors reported involved disrespectful communication or lack of professional responsibility. In one example, a physician called a coworker a “bossy cow” when the coworker reminded the physician of the need to do a timeout before beginning a bronchoscopy.

In another case involving professional responsibility, a coworker asked a physician if the team should wait for a disoriented patient’s spouse to arrive. The doctor’s response: “We’ll be here all night if we do that. If you won’t sign as a witness, I’ll get someone else who will.”

The least common reports involved unprofessionalism related to medical care or professional integrity. One cited a physician removing a Foley catheter without wearing gloves and having visible urine on his hands and not washing them before touching other things in the room. In a reported lapse of professional integrity, a physician billed at level five after spending only 4 minutes with a patient.
 

Impact of Unprofessional Behavior

Unprofessional behavior among physicians is more than just unpleasant. It can threaten the functioning of teams and increase patient complications. In addition, individuals who model unprofessional behaviors are associated with increased malpractice claims, the study’s authors wrote.

Dr. Katz agreed that unprofessional behavior is damaging to both patients and the profession as a whole.

However, this doesn’t happen because some doctors are bad, he said. Physicians today are working in a pressure cooker. The current healthcare environment, with its increased administrative burdens, lack of staffing, and other problems, has increased the overall level of stress and led to burnout among healthcare personnel.

“You have to fix the system to create a working environment that doesn’t cause somebody to explode,” Dr. Katz said.

The goal of the CORS program and this study, Dr. Cooper said, is to help physicians better weather these stresses.
 

Study Limitations

The authors noted some weaknesses in the study. Some unprofessional behavior may go unreported because of fear of retaliation or for other reasons victims or witnesses did not feel safe to report their colleagues. Also, reports were not evaluated to ensure the truth of the accusations. The records reviewed did not include the gender of the physician, though the researchers pointed out that previous studies have shown that women are less likely than men to receive CORS reports.
 

A version of this article appeared on Medscape.com.

Most doctors mind their manners. But surgeons are the most likely to be reported for unprofessional behavior, while physicians practicing in pediatric settings are the least likely, according to a recent study of more than 35,000 physicians.

The research, published on June 6 in JAMA Network Open, found that fewer than 10% of physicians were reported by their coworkers for at least one instance of unprofessional behavior, and only 1% showed a pattern of such reports.

Data were gathered from the Center for Patient and Professional Advocacy’s (CPPA’s) Coworker Observation Reporting System (CORS) program, a national collaborative in which 193 participating hospitals and practice sites file safety-event reports involving medical workers’ unprofessional behaviors. An algorithm that weights CORS reports based on recency and severity was used to analyze the data. The study was spearheaded by William O. Cooper, MD, MPH, director of the CPPA at Vanderbilt University Medical Center, Nashville, Tennessee.

The retrospective cohort study included deidentified data on credentialed physicians, not including residents or fellows, who practiced at a CORS site between 2018 and 2022.
 

Why Surgeons?

The authors speculated that the reason surgeons were reported for unprofessional behavior more often than their colleagues in nonsurgical specialties was because surgery is a more stressful environment than other specialties and requires more teamwork, resulting in more interactions during high-stakes events.

Daniel Katz, MD, professor and vice chair of education for the Department of Anesthesiology, Perioperative and Pain Medicine at the Icahn School of Medicine at Mount Sinai, New York City, added that part of the problem is that surgeons are expected to perform at very high levels all the time.

“When things that are outside the control of the surgeon don’t go well,” Dr. Katz said, “that can lead to increased frustration and negative emotions, which will then bring out these kinds of behaviors.”
 

Types of Unprofessional Behaviors

The most common out-of-bounds behaviors reported involved disrespectful communication or lack of professional responsibility. In one example, a physician called a coworker a “bossy cow” when the coworker reminded the physician of the need to do a timeout before beginning a bronchoscopy.

In another case involving professional responsibility, a coworker asked a physician if the team should wait for a disoriented patient’s spouse to arrive. The doctor’s response: “We’ll be here all night if we do that. If you won’t sign as a witness, I’ll get someone else who will.”

The least common reports involved unprofessionalism related to medical care or professional integrity. One cited a physician removing a Foley catheter without wearing gloves and having visible urine on his hands and not washing them before touching other things in the room. In a reported lapse of professional integrity, a physician billed at level five after spending only 4 minutes with a patient.
 

Impact of Unprofessional Behavior

Unprofessional behavior among physicians is more than just unpleasant. It can threaten the functioning of teams and increase patient complications. In addition, individuals who model unprofessional behaviors are associated with increased malpractice claims, the study’s authors wrote.

Dr. Katz agreed that unprofessional behavior is damaging to both patients and the profession as a whole.

However, this doesn’t happen because some doctors are bad, he said. Physicians today are working in a pressure cooker. The current healthcare environment, with its increased administrative burdens, lack of staffing, and other problems, has increased the overall level of stress and led to burnout among healthcare personnel.

“You have to fix the system to create a working environment that doesn’t cause somebody to explode,” Dr. Katz said.

The goal of the CORS program and this study, Dr. Cooper said, is to help physicians better weather these stresses.
 

Study Limitations

The authors noted some weaknesses in the study. Some unprofessional behavior may go unreported because of fear of retaliation or for other reasons victims or witnesses did not feel safe to report their colleagues. Also, reports were not evaluated to ensure the truth of the accusations. The records reviewed did not include the gender of the physician, though the researchers pointed out that previous studies have shown that women are less likely than men to receive CORS reports.
 

A version of this article appeared on Medscape.com.

Most doctors mind their manners. But surgeons are the most likely to be reported for unprofessional behavior, while physicians practicing in pediatric settings are the least likely, according to a recent study of more than 35,000 physicians.

The research, published on June 6 in JAMA Network Open, found that fewer than 10% of physicians were reported by their coworkers for at least one instance of unprofessional behavior, and only 1% showed a pattern of such reports.

Data were gathered from the Center for Patient and Professional Advocacy’s (CPPA’s) Coworker Observation Reporting System (CORS) program, a national collaborative in which 193 participating hospitals and practice sites file safety-event reports involving medical workers’ unprofessional behaviors. An algorithm that weights CORS reports based on recency and severity was used to analyze the data. The study was spearheaded by William O. Cooper, MD, MPH, director of the CPPA at Vanderbilt University Medical Center, Nashville, Tennessee.

The retrospective cohort study included deidentified data on credentialed physicians, not including residents or fellows, who practiced at a CORS site between 2018 and 2022.
 

Why Surgeons?

The authors speculated that the reason surgeons were reported for unprofessional behavior more often than their colleagues in nonsurgical specialties was because surgery is a more stressful environment than other specialties and requires more teamwork, resulting in more interactions during high-stakes events.

Daniel Katz, MD, professor and vice chair of education for the Department of Anesthesiology, Perioperative and Pain Medicine at the Icahn School of Medicine at Mount Sinai, New York City, added that part of the problem is that surgeons are expected to perform at very high levels all the time.

“When things that are outside the control of the surgeon don’t go well,” Dr. Katz said, “that can lead to increased frustration and negative emotions, which will then bring out these kinds of behaviors.”
 

Types of Unprofessional Behaviors

The most common out-of-bounds behaviors reported involved disrespectful communication or lack of professional responsibility. In one example, a physician called a coworker a “bossy cow” when the coworker reminded the physician of the need to do a timeout before beginning a bronchoscopy.

In another case involving professional responsibility, a coworker asked a physician if the team should wait for a disoriented patient’s spouse to arrive. The doctor’s response: “We’ll be here all night if we do that. If you won’t sign as a witness, I’ll get someone else who will.”

The least common reports involved unprofessionalism related to medical care or professional integrity. One cited a physician removing a Foley catheter without wearing gloves and having visible urine on his hands and not washing them before touching other things in the room. In a reported lapse of professional integrity, a physician billed at level five after spending only 4 minutes with a patient.
 

Impact of Unprofessional Behavior

Unprofessional behavior among physicians is more than just unpleasant. It can threaten the functioning of teams and increase patient complications. In addition, individuals who model unprofessional behaviors are associated with increased malpractice claims, the study’s authors wrote.

Dr. Katz agreed that unprofessional behavior is damaging to both patients and the profession as a whole.

However, this doesn’t happen because some doctors are bad, he said. Physicians today are working in a pressure cooker. The current healthcare environment, with its increased administrative burdens, lack of staffing, and other problems, has increased the overall level of stress and led to burnout among healthcare personnel.

“You have to fix the system to create a working environment that doesn’t cause somebody to explode,” Dr. Katz said.

The goal of the CORS program and this study, Dr. Cooper said, is to help physicians better weather these stresses.
 

Study Limitations

The authors noted some weaknesses in the study. Some unprofessional behavior may go unreported because of fear of retaliation or for other reasons victims or witnesses did not feel safe to report their colleagues. Also, reports were not evaluated to ensure the truth of the accusations. The records reviewed did not include the gender of the physician, though the researchers pointed out that previous studies have shown that women are less likely than men to receive CORS reports.
 

A version of this article appeared on Medscape.com.

Almost all primary care providers (PCPs) have taken on diagnosing and managing ADHD. With about 12% of school aged children affected, typical PCPs can expect about 240 children with ADHD under their care. Adopting this primary care function has been helped by having clear diagnostic criteria for the three DMS 5 “presentations” of ADHD, open source tools (e.g. Vanderbilts), expectation of collaboration by educators, American Academy of Pediatrics (AAP) guidelines for diagnosis and management, Society for Developmental–Behavioral Pediatrics guidelines for “complex ADHD,” and access to effective medication treatments PCPs can provide (although less so for behavioral ones), cultural acceptance of individuals with ADHD, and especially reliable payment by insurers.

Screening

But what about PCP management of autism spectrum disorder (ASD), now affecting 2.8%, for an expected 60 children under care for each of us? PCP detection and care for children with ASD is more complex than ADHD, but even more essential, so we need to learn the skills. It is more essential because very early detection and entry into evidence-based intervention has long-term benefits for the child and family that are not as crucial for ADHD. While ADHD symptoms may not impact functioning until age 7 or even 12 years of age, signs of ASD usually emerge earlier (by 18 months) but gradually and about 30% after apparently normal development even to age 2 years.

Screening is crucial, but unfortunately not perfect. Recent AAP surveys show that most PCPs screen for autism at the recommended 18 and 24 months. But what happens after that? How many offices are tracking referrals for positive screens for needed evaluations and early intervention? Our data shows that tracking is rarely done and children do not start to get the benefit of early intervention until 4.5 years of age, on average.
 

Diagnostic Testing

And screening is the easiest part of addressing ASD. Wait times for diagnostic testing can be agonizing months to years. Multiple programs are training PCPs to perform hands-on 10- to 30-minute secondary screening with considerable success. You can become proficient on tools such as STAT (Screening Tool for Autism in Two-Year-Olds), RITA-T (Rapid Interactive Screening Test for Autism in Toddlers), BISCUIT (Baby and Infant Screen for Children with Autism Traits), SORF (Systematic Observation of Red Flags), ADEC (Autism Detection in Early Childhood) or CARS (Childhood Autism Rating Scale) with a few hours of training. Even secondary assessments done virtually by PCPs such as TELE-ASD-PEDS quite accurately predict a verifiable ASD diagnosis for those referred by concerns. Some problems of the reported accuracy of these secondary screening processes have to do with validation in samples of children for whom parents or clinicians already had concern and generally not including many younger children in whom it is so important to detect. Level of confidence of developmental and behavioral pediatricians of the presence of ASD is highly related to ultimate diagnosis. But success with PCPs’ mastering secondary screening has not yet been reported to convince insurers to approve payment for intervention services such as Applied Behavior Analysis (ABA).

Comorbidity

Co-existing conditions affect the majority of patients with ASD (70%), compared with ADHD, but with a broader range and more debilitating and difficult to manage conditions. More medical co-existing issues such as intellectual disability (25%-75%), seizures (12%-26%), motor incoordination (51%), GI conditions (9%-91%), sleep difficulty (50%-80%), sleep apnea, congenital heart disease, avoidant-restrictive food intake disorder, autoimmune disorders, and genetic syndromes (e.g. Fragile X, tuberous sclerosis, Down, Angelman’s, untreated PKU, neurofibromatosis, Klinefelter syndrome) reflect the range of underpinnings of ASD. The need to detect and manage these co-existing issues, besides assessing hearing and vision, makes our skilled involvement and vigilance in ASD care essential. Referring for help from OTs, PTs, speech pathologists, neurologists, psychologists, and special educators as issues in their domains are prioritized is also our responsibility. We must also help families balance utilizing these resources so as to avoid overwhelm.

Anxiety (50%), ADHD (37%-85%), depression (54%), bipolar (7.3%), suicidal ideation (40% starting < 8 years), and emotion dysregulation, familiar to us from our management of ADHD, may develop but are often less well defined and more intractable in ASD, making use of screening tools essential. Using a system like CHADIS that has online pre-visit and monitoring screens delivered based on algorithms for the numerous co-existing conditions, automated handouts, and functions to make and track referral success can facilitate care for this complex chronic condition. Identifying mental health providers with ASD expertise is more difficult, so more management is on us. While medications for these conditions can be beneficial, we need to learn to use lower doses, slower dose increases, and employ problem-solving of side effects with more parent collaboration than for ADHD as children with ASD often cannot self-report effectively. We need to ask about the common ad hoc use of complementary medications and substances (32%-87%) that may be complicating. Of course, these conditions and the caveats of management require more of our time with the patient and family as well as communication with the many other professionals involved. It is important to set our own and our families’ expectations (and schedules) for much more frequent contact and also to bill appropriately with chronic care (99487,89,90) and collaborative care CPT codes (99492,3,4 or G2214).
 

Behavioral Manifestations

During our care, the often extreme behavioral manifestations of ASD may be the most pressing issues. We need new understanding and skills to sort out and counsel on inflexible, explosive, and sensory triggered behaviors. Just as for ADHD, using the approach of Functional Behavioral Assessment and plans for home as well as school behavior can be key. More difficult in ASD is looking for physical causes, since the child may not provide clear cues because of communication and sensory differences. Conditions common in children with ASD such as constipation, dental caries, otitis, dietary intolerances, allergies, migraine, sleep deficits, menstrual cramps, or fears and changes from puberty manifesting behaviorally are often tricky to sort out.

While the diagnosis of ASD, as for ADHD, does not require any laboratory testing, looking for possible causes is important information for the family and someday may also lead to genetic or other therapies. We need to know that recommendations include screening for Ferritin, Pb, chromosomal microarray and FMR I testing as well as checking that PKU was normal; MECP 2 is indicated in females and symptomatic males; and PTENS testing for children with head circumference greater than 2.5-3 SD. Metabolic and mitochondrial assays are indicated only when symptoms suggest. We need to develop confidence to reserve MRIs or EEGs for cases with abnormal neuro. exams, regression, or history of seizures. It is demanding to keep up with AAP recommendations in this very active area of research.
 

Interventions

The interventions for ADHD are generally school accommodations and therapies for comorbidities. In contrast, since core social communication skills are the main deficit in ASD, all children screened positive for ASD should be referred for early intervention while awaiting, as well as after, diagnosis. While all states have no or low-cost early intervention, quality and quantity (of hours offered) varies. We should also recommend and try to determine if evidence-based intervention is being provided, such as pivotal response training, UCLA discrete trial therapy, Carbone’s verbal behavior, applied behavior analysis (ABA), Early Start Denver Model, and sometimes music and social skills trainings (effect size 0.42-0.76). Such professional interventions have best evidence with more than 25 hours/week but 15 hours has benefit for higher functioning children. CBT can help anxiety even in younger children. One way for families to provide more hours and more generalizable intervention is coaching by the PLAY Project or DIRFloortime, parent mediated interventions with evidence, some with training both in person or online. Alternative communication training and other condition specific assistance are often needed (e.g. Picture Exchange Communication System for nonverbal children).

While we should already be familiar with writing 504 plan and IEP requests to schools, which also apply to children with ASD, in addition we need to be ready to advise about other legal rights including autism waivers, wraparound services, guardianship, and trust accounts. We can share quality educational materials available online (e.g. from Autism Speaks, SPARK, and Autism Navigator). Social media groups may be supportive, but also may contain disinformation we need to dispel.

Unfortunately, templates, questionnaires, and lack of interdisciplinary referral and communication functions of EHRs don’t support the complexities of care for ASD. While the AAP has guidelines for diagnosis and management and an online toolkit, consider adding a system with an autism-specific module like CHADIS and joining the Autism Care Network or ECHO Autism sessions to get both information and support to take on the evolving critical role of autism care.
 

Dr. Howard is assistant professor of pediatrics at Johns Hopkins University, Baltimore, and creator of CHADIS. She had no other relevant disclosures. Dr. Howard’s contribution to this publication was as a paid expert to MDedge News. E-mail her at pdnews@mdedge.com.

Almost all primary care providers (PCPs) have taken on diagnosing and managing ADHD. With about 12% of school aged children affected, typical PCPs can expect about 240 children with ADHD under their care. Adopting this primary care function has been helped by having clear diagnostic criteria for the three DMS 5 “presentations” of ADHD, open source tools (e.g. Vanderbilts), expectation of collaboration by educators, American Academy of Pediatrics (AAP) guidelines for diagnosis and management, Society for Developmental–Behavioral Pediatrics guidelines for “complex ADHD,” and access to effective medication treatments PCPs can provide (although less so for behavioral ones), cultural acceptance of individuals with ADHD, and especially reliable payment by insurers.

Screening

But what about PCP management of autism spectrum disorder (ASD), now affecting 2.8%, for an expected 60 children under care for each of us? PCP detection and care for children with ASD is more complex than ADHD, but even more essential, so we need to learn the skills. It is more essential because very early detection and entry into evidence-based intervention has long-term benefits for the child and family that are not as crucial for ADHD. While ADHD symptoms may not impact functioning until age 7 or even 12 years of age, signs of ASD usually emerge earlier (by 18 months) but gradually and about 30% after apparently normal development even to age 2 years.

Screening is crucial, but unfortunately not perfect. Recent AAP surveys show that most PCPs screen for autism at the recommended 18 and 24 months. But what happens after that? How many offices are tracking referrals for positive screens for needed evaluations and early intervention? Our data shows that tracking is rarely done and children do not start to get the benefit of early intervention until 4.5 years of age, on average.
 

Diagnostic Testing

And screening is the easiest part of addressing ASD. Wait times for diagnostic testing can be agonizing months to years. Multiple programs are training PCPs to perform hands-on 10- to 30-minute secondary screening with considerable success. You can become proficient on tools such as STAT (Screening Tool for Autism in Two-Year-Olds), RITA-T (Rapid Interactive Screening Test for Autism in Toddlers), BISCUIT (Baby and Infant Screen for Children with Autism Traits), SORF (Systematic Observation of Red Flags), ADEC (Autism Detection in Early Childhood) or CARS (Childhood Autism Rating Scale) with a few hours of training. Even secondary assessments done virtually by PCPs such as TELE-ASD-PEDS quite accurately predict a verifiable ASD diagnosis for those referred by concerns. Some problems of the reported accuracy of these secondary screening processes have to do with validation in samples of children for whom parents or clinicians already had concern and generally not including many younger children in whom it is so important to detect. Level of confidence of developmental and behavioral pediatricians of the presence of ASD is highly related to ultimate diagnosis. But success with PCPs’ mastering secondary screening has not yet been reported to convince insurers to approve payment for intervention services such as Applied Behavior Analysis (ABA).

Comorbidity

Co-existing conditions affect the majority of patients with ASD (70%), compared with ADHD, but with a broader range and more debilitating and difficult to manage conditions. More medical co-existing issues such as intellectual disability (25%-75%), seizures (12%-26%), motor incoordination (51%), GI conditions (9%-91%), sleep difficulty (50%-80%), sleep apnea, congenital heart disease, avoidant-restrictive food intake disorder, autoimmune disorders, and genetic syndromes (e.g. Fragile X, tuberous sclerosis, Down, Angelman’s, untreated PKU, neurofibromatosis, Klinefelter syndrome) reflect the range of underpinnings of ASD. The need to detect and manage these co-existing issues, besides assessing hearing and vision, makes our skilled involvement and vigilance in ASD care essential. Referring for help from OTs, PTs, speech pathologists, neurologists, psychologists, and special educators as issues in their domains are prioritized is also our responsibility. We must also help families balance utilizing these resources so as to avoid overwhelm.

Anxiety (50%), ADHD (37%-85%), depression (54%), bipolar (7.3%), suicidal ideation (40% starting < 8 years), and emotion dysregulation, familiar to us from our management of ADHD, may develop but are often less well defined and more intractable in ASD, making use of screening tools essential. Using a system like CHADIS that has online pre-visit and monitoring screens delivered based on algorithms for the numerous co-existing conditions, automated handouts, and functions to make and track referral success can facilitate care for this complex chronic condition. Identifying mental health providers with ASD expertise is more difficult, so more management is on us. While medications for these conditions can be beneficial, we need to learn to use lower doses, slower dose increases, and employ problem-solving of side effects with more parent collaboration than for ADHD as children with ASD often cannot self-report effectively. We need to ask about the common ad hoc use of complementary medications and substances (32%-87%) that may be complicating. Of course, these conditions and the caveats of management require more of our time with the patient and family as well as communication with the many other professionals involved. It is important to set our own and our families’ expectations (and schedules) for much more frequent contact and also to bill appropriately with chronic care (99487,89,90) and collaborative care CPT codes (99492,3,4 or G2214).
 

Behavioral Manifestations

During our care, the often extreme behavioral manifestations of ASD may be the most pressing issues. We need new understanding and skills to sort out and counsel on inflexible, explosive, and sensory triggered behaviors. Just as for ADHD, using the approach of Functional Behavioral Assessment and plans for home as well as school behavior can be key. More difficult in ASD is looking for physical causes, since the child may not provide clear cues because of communication and sensory differences. Conditions common in children with ASD such as constipation, dental caries, otitis, dietary intolerances, allergies, migraine, sleep deficits, menstrual cramps, or fears and changes from puberty manifesting behaviorally are often tricky to sort out.

While the diagnosis of ASD, as for ADHD, does not require any laboratory testing, looking for possible causes is important information for the family and someday may also lead to genetic or other therapies. We need to know that recommendations include screening for Ferritin, Pb, chromosomal microarray and FMR I testing as well as checking that PKU was normal; MECP 2 is indicated in females and symptomatic males; and PTENS testing for children with head circumference greater than 2.5-3 SD. Metabolic and mitochondrial assays are indicated only when symptoms suggest. We need to develop confidence to reserve MRIs or EEGs for cases with abnormal neuro. exams, regression, or history of seizures. It is demanding to keep up with AAP recommendations in this very active area of research.
 

Interventions

The interventions for ADHD are generally school accommodations and therapies for comorbidities. In contrast, since core social communication skills are the main deficit in ASD, all children screened positive for ASD should be referred for early intervention while awaiting, as well as after, diagnosis. While all states have no or low-cost early intervention, quality and quantity (of hours offered) varies. We should also recommend and try to determine if evidence-based intervention is being provided, such as pivotal response training, UCLA discrete trial therapy, Carbone’s verbal behavior, applied behavior analysis (ABA), Early Start Denver Model, and sometimes music and social skills trainings (effect size 0.42-0.76). Such professional interventions have best evidence with more than 25 hours/week but 15 hours has benefit for higher functioning children. CBT can help anxiety even in younger children. One way for families to provide more hours and more generalizable intervention is coaching by the PLAY Project or DIRFloortime, parent mediated interventions with evidence, some with training both in person or online. Alternative communication training and other condition specific assistance are often needed (e.g. Picture Exchange Communication System for nonverbal children).

While we should already be familiar with writing 504 plan and IEP requests to schools, which also apply to children with ASD, in addition we need to be ready to advise about other legal rights including autism waivers, wraparound services, guardianship, and trust accounts. We can share quality educational materials available online (e.g. from Autism Speaks, SPARK, and Autism Navigator). Social media groups may be supportive, but also may contain disinformation we need to dispel.

Unfortunately, templates, questionnaires, and lack of interdisciplinary referral and communication functions of EHRs don’t support the complexities of care for ASD. While the AAP has guidelines for diagnosis and management and an online toolkit, consider adding a system with an autism-specific module like CHADIS and joining the Autism Care Network or ECHO Autism sessions to get both information and support to take on the evolving critical role of autism care.
 

Dr. Howard is assistant professor of pediatrics at Johns Hopkins University, Baltimore, and creator of CHADIS. She had no other relevant disclosures. Dr. Howard’s contribution to this publication was as a paid expert to MDedge News. E-mail her at pdnews@mdedge.com.

Almost all primary care providers (PCPs) have taken on diagnosing and managing ADHD. With about 12% of school aged children affected, typical PCPs can expect about 240 children with ADHD under their care. Adopting this primary care function has been helped by having clear diagnostic criteria for the three DMS 5 “presentations” of ADHD, open source tools (e.g. Vanderbilts), expectation of collaboration by educators, American Academy of Pediatrics (AAP) guidelines for diagnosis and management, Society for Developmental–Behavioral Pediatrics guidelines for “complex ADHD,” and access to effective medication treatments PCPs can provide (although less so for behavioral ones), cultural acceptance of individuals with ADHD, and especially reliable payment by insurers.

Screening

But what about PCP management of autism spectrum disorder (ASD), now affecting 2.8%, for an expected 60 children under care for each of us? PCP detection and care for children with ASD is more complex than ADHD, but even more essential, so we need to learn the skills. It is more essential because very early detection and entry into evidence-based intervention has long-term benefits for the child and family that are not as crucial for ADHD. While ADHD symptoms may not impact functioning until age 7 or even 12 years of age, signs of ASD usually emerge earlier (by 18 months) but gradually and about 30% after apparently normal development even to age 2 years.

Screening is crucial, but unfortunately not perfect. Recent AAP surveys show that most PCPs screen for autism at the recommended 18 and 24 months. But what happens after that? How many offices are tracking referrals for positive screens for needed evaluations and early intervention? Our data shows that tracking is rarely done and children do not start to get the benefit of early intervention until 4.5 years of age, on average.
 

Diagnostic Testing

And screening is the easiest part of addressing ASD. Wait times for diagnostic testing can be agonizing months to years. Multiple programs are training PCPs to perform hands-on 10- to 30-minute secondary screening with considerable success. You can become proficient on tools such as STAT (Screening Tool for Autism in Two-Year-Olds), RITA-T (Rapid Interactive Screening Test for Autism in Toddlers), BISCUIT (Baby and Infant Screen for Children with Autism Traits), SORF (Systematic Observation of Red Flags), ADEC (Autism Detection in Early Childhood) or CARS (Childhood Autism Rating Scale) with a few hours of training. Even secondary assessments done virtually by PCPs such as TELE-ASD-PEDS quite accurately predict a verifiable ASD diagnosis for those referred by concerns. Some problems of the reported accuracy of these secondary screening processes have to do with validation in samples of children for whom parents or clinicians already had concern and generally not including many younger children in whom it is so important to detect. Level of confidence of developmental and behavioral pediatricians of the presence of ASD is highly related to ultimate diagnosis. But success with PCPs’ mastering secondary screening has not yet been reported to convince insurers to approve payment for intervention services such as Applied Behavior Analysis (ABA).

Comorbidity

Co-existing conditions affect the majority of patients with ASD (70%), compared with ADHD, but with a broader range and more debilitating and difficult to manage conditions. More medical co-existing issues such as intellectual disability (25%-75%), seizures (12%-26%), motor incoordination (51%), GI conditions (9%-91%), sleep difficulty (50%-80%), sleep apnea, congenital heart disease, avoidant-restrictive food intake disorder, autoimmune disorders, and genetic syndromes (e.g. Fragile X, tuberous sclerosis, Down, Angelman’s, untreated PKU, neurofibromatosis, Klinefelter syndrome) reflect the range of underpinnings of ASD. The need to detect and manage these co-existing issues, besides assessing hearing and vision, makes our skilled involvement and vigilance in ASD care essential. Referring for help from OTs, PTs, speech pathologists, neurologists, psychologists, and special educators as issues in their domains are prioritized is also our responsibility. We must also help families balance utilizing these resources so as to avoid overwhelm.

Anxiety (50%), ADHD (37%-85%), depression (54%), bipolar (7.3%), suicidal ideation (40% starting < 8 years), and emotion dysregulation, familiar to us from our management of ADHD, may develop but are often less well defined and more intractable in ASD, making use of screening tools essential. Using a system like CHADIS that has online pre-visit and monitoring screens delivered based on algorithms for the numerous co-existing conditions, automated handouts, and functions to make and track referral success can facilitate care for this complex chronic condition. Identifying mental health providers with ASD expertise is more difficult, so more management is on us. While medications for these conditions can be beneficial, we need to learn to use lower doses, slower dose increases, and employ problem-solving of side effects with more parent collaboration than for ADHD as children with ASD often cannot self-report effectively. We need to ask about the common ad hoc use of complementary medications and substances (32%-87%) that may be complicating. Of course, these conditions and the caveats of management require more of our time with the patient and family as well as communication with the many other professionals involved. It is important to set our own and our families’ expectations (and schedules) for much more frequent contact and also to bill appropriately with chronic care (99487,89,90) and collaborative care CPT codes (99492,3,4 or G2214).
 

Behavioral Manifestations

During our care, the often extreme behavioral manifestations of ASD may be the most pressing issues. We need new understanding and skills to sort out and counsel on inflexible, explosive, and sensory triggered behaviors. Just as for ADHD, using the approach of Functional Behavioral Assessment and plans for home as well as school behavior can be key. More difficult in ASD is looking for physical causes, since the child may not provide clear cues because of communication and sensory differences. Conditions common in children with ASD such as constipation, dental caries, otitis, dietary intolerances, allergies, migraine, sleep deficits, menstrual cramps, or fears and changes from puberty manifesting behaviorally are often tricky to sort out.

While the diagnosis of ASD, as for ADHD, does not require any laboratory testing, looking for possible causes is important information for the family and someday may also lead to genetic or other therapies. We need to know that recommendations include screening for Ferritin, Pb, chromosomal microarray and FMR I testing as well as checking that PKU was normal; MECP 2 is indicated in females and symptomatic males; and PTENS testing for children with head circumference greater than 2.5-3 SD. Metabolic and mitochondrial assays are indicated only when symptoms suggest. We need to develop confidence to reserve MRIs or EEGs for cases with abnormal neuro. exams, regression, or history of seizures. It is demanding to keep up with AAP recommendations in this very active area of research.
 

Interventions

The interventions for ADHD are generally school accommodations and therapies for comorbidities. In contrast, since core social communication skills are the main deficit in ASD, all children screened positive for ASD should be referred for early intervention while awaiting, as well as after, diagnosis. While all states have no or low-cost early intervention, quality and quantity (of hours offered) varies. We should also recommend and try to determine if evidence-based intervention is being provided, such as pivotal response training, UCLA discrete trial therapy, Carbone’s verbal behavior, applied behavior analysis (ABA), Early Start Denver Model, and sometimes music and social skills trainings (effect size 0.42-0.76). Such professional interventions have best evidence with more than 25 hours/week but 15 hours has benefit for higher functioning children. CBT can help anxiety even in younger children. One way for families to provide more hours and more generalizable intervention is coaching by the PLAY Project or DIRFloortime, parent mediated interventions with evidence, some with training both in person or online. Alternative communication training and other condition specific assistance are often needed (e.g. Picture Exchange Communication System for nonverbal children).

While we should already be familiar with writing 504 plan and IEP requests to schools, which also apply to children with ASD, in addition we need to be ready to advise about other legal rights including autism waivers, wraparound services, guardianship, and trust accounts. We can share quality educational materials available online (e.g. from Autism Speaks, SPARK, and Autism Navigator). Social media groups may be supportive, but also may contain disinformation we need to dispel.

Unfortunately, templates, questionnaires, and lack of interdisciplinary referral and communication functions of EHRs don’t support the complexities of care for ASD. While the AAP has guidelines for diagnosis and management and an online toolkit, consider adding a system with an autism-specific module like CHADIS and joining the Autism Care Network or ECHO Autism sessions to get both information and support to take on the evolving critical role of autism care.
 

Dr. Howard is assistant professor of pediatrics at Johns Hopkins University, Baltimore, and creator of CHADIS. She had no other relevant disclosures. Dr. Howard’s contribution to this publication was as a paid expert to MDedge News. E-mail her at pdnews@mdedge.com.

A new broadly inclusive definition of long COVID from the National Academies of Sciences, Engineering, and Medicine (NASEM) has been developed with the aim of improving consistency, documentation, and treatment for both adults and children.

According to the 2024 NASEM definition of long COVID issued on June 11, 2024, “Long COVID is an infection-associated chronic condition that occurs after SARS-CoV-2 infection and is present for at least 3 months as a continuous, relapsing and remitting, or progressive disease state that affects one or more organ systems.” 

People with long COVID may present with one or more of a long list of symptoms, such as shortness of breath, rapid heartbeat, extreme fatigue, post-exertional malaise, or sleep disturbance and with single or multiple diagnosable conditions, including interstitial lung disease, arrhythmias, postural orthostatic tachycardia syndrome (POTS), myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS), diabetes, or autoimmune disorders. The condition can exacerbate preexisting health conditions or present as new ones. 

The definition does not require laboratory confirmation or other proof of initial infection. Long COVID can follow SARS-CoV-2 infection of any severity, including asymptomatic infections, whether or not they were initially recognized. 

Several working definitions and terms for long COVID had previously been proposed, including those from the World Health Organization (WHO) and the US Centers for Disease Control and Prevention, but no common definition or terminology had been established.

The new definition was developed at the request of the Administration for Strategic Preparedness and Response and the Office of the Assistant Secretary for Health (OASH). It was written by a multi-stakeholder panel convened by NASEM, which recommended that the new definition be universally adopted by the federal government, clinical societies and associations, public health practitioners, clinicians, payers, the drug industry, and others using the term long COVID. 

Recent surveys suggest that approximately 7% of Americans have experienced or are experiencing long COVID. “It’s millions of people,” panel chair Harvey V. Fineberg, MD, president of the Gordon and Betty Moore Foundation, told this news organization. 

The new definition “does not erase the problem of clinical judgment ... But we think this definition has the real advantage of elevating to the clinician’s mind the real likelihood in the current environment of prevalence of this virus that a presenting patient’s strange symptoms are both real and maybe related as an expression of long COVID,” Dr. Fineberg noted. 

One way this new definition differs from previous ones such as WHO’s, he said, is “they talk about a diagnosis of exclusion. One of the important points in our definition is that other diagnosable conditions like ME/CFS or POTS can be part of the picture of long COVID. They are not alternative. They are, in fact, an expression of long COVID.”

Indeed, the NASEM report also introduces the term infection-associated chronic condition (IACC). This was important, Dr. Fineberg said, “because it’s the larger family of conditions of which long COVID is a part. It emphasizes a relatedness of long COVID to other conditions that can follow from a variety of infections. We also adopted the term ‘disease state’ to convey the seriousness and reality of this condition in the lives of patients.” 
 

Comments on New Definition

In a statement provided to this news organization, Lucinda Bateman, MD, and Brayden Yellman, MD, co-medical directors of the Bateman-Horne Center in Salt Lake City, said that “describing long COVID as an IACC ... not only meets the NASEM goal of allowing clinicians, researchers, and public health officials to meaningfully identify and serve all persons who suffer illness or disability in the wake of a SARS-CoV-2 infection, but also draws direct comparison to other known IACC’s (such as ME/CFS, post-treatment Lyme, POTS) that have been plaguing many for decades.”

Dr. Fineberg noted another important aspect of the NASEM report: “Our definition includes an explicit statement on equity, explaining that long COVID can affect anyone, young and old, different races, different ages, different sexes, different genders, different orientations, different socioeconomic conditions ... This does not mean that every single person is at equal risk. There are risk factors, but the important point is the universal nature of this as a condition.”

Two clinical directors of long COVID programs who were contacted by this news organization praised the new definition. Zijian Chen, MD, director of Mount Sinai’s Center for Post-COVID Care, New York, said that it’s “very similar to the definition that we have used for our clinical practice since 2020. It is very important that the broad definition helps to be inclusive of all patients that may be affected. The inclusion of children as a consideration is important as well, since there is routinely less focus on children because they tend to have less disease frequency ... The creation of a unified definition helps both with clinical practice and research.”

Nisha Viswanathan, MD, director of the long COVID program at the University of California, Los Angeles, said: “I think they left it intentionally broad for the medical practitioner to not necessarily use the definition to rule out individuals, but to perhaps use more of a clinical gestalt to help rule in this diagnosis ... I think this definition is providing clarity to health care providers on what exactly would be falling under the long-COVID diagnosis header.” 

Dr. Viswanathan also said that she anticipates this definition to help patients make their case in filing disability claims. “Because long COVID has not previously had a good fleshed-out definition, it was very easy for disability providers to reject claims for patients who continue to have symptoms ... I actually think this might help our patients ultimately in their attempt to be able to have the ability to care for themselves when they’re disabled enough to not be able to work.”

Written into the report is the expectation that the definition “will evolve as new evidence emerges and the understanding of long COVID matures.” The writing committee calls for reexamination in “no more than 3 years.” Factors that would prompt a reevaluation could include improved testing methods, discovery of medical factors and/or biomarkers that distinguish long COVID from other conditions, and new treatments. 

Meanwhile, Dr. Fineberg told this news organization, “If this definition adds to the readiness, awareness, openness, and response to the patient with long COVID, it will have done its job.” 

Dr. Fineberg, Dr. Bateman, Dr. Yellman, Dr. Viswanathan, and Dr. Chen have no relevant disclosures.

A version of this article appeared on Medscape.com.

A new broadly inclusive definition of long COVID from the National Academies of Sciences, Engineering, and Medicine (NASEM) has been developed with the aim of improving consistency, documentation, and treatment for both adults and children.

According to the 2024 NASEM definition of long COVID issued on June 11, 2024, “Long COVID is an infection-associated chronic condition that occurs after SARS-CoV-2 infection and is present for at least 3 months as a continuous, relapsing and remitting, or progressive disease state that affects one or more organ systems.” 

People with long COVID may present with one or more of a long list of symptoms, such as shortness of breath, rapid heartbeat, extreme fatigue, post-exertional malaise, or sleep disturbance and with single or multiple diagnosable conditions, including interstitial lung disease, arrhythmias, postural orthostatic tachycardia syndrome (POTS), myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS), diabetes, or autoimmune disorders. The condition can exacerbate preexisting health conditions or present as new ones. 

The definition does not require laboratory confirmation or other proof of initial infection. Long COVID can follow SARS-CoV-2 infection of any severity, including asymptomatic infections, whether or not they were initially recognized. 

Several working definitions and terms for long COVID had previously been proposed, including those from the World Health Organization (WHO) and the US Centers for Disease Control and Prevention, but no common definition or terminology had been established.

The new definition was developed at the request of the Administration for Strategic Preparedness and Response and the Office of the Assistant Secretary for Health (OASH). It was written by a multi-stakeholder panel convened by NASEM, which recommended that the new definition be universally adopted by the federal government, clinical societies and associations, public health practitioners, clinicians, payers, the drug industry, and others using the term long COVID. 

Recent surveys suggest that approximately 7% of Americans have experienced or are experiencing long COVID. “It’s millions of people,” panel chair Harvey V. Fineberg, MD, president of the Gordon and Betty Moore Foundation, told this news organization. 

The new definition “does not erase the problem of clinical judgment ... But we think this definition has the real advantage of elevating to the clinician’s mind the real likelihood in the current environment of prevalence of this virus that a presenting patient’s strange symptoms are both real and maybe related as an expression of long COVID,” Dr. Fineberg noted. 

One way this new definition differs from previous ones such as WHO’s, he said, is “they talk about a diagnosis of exclusion. One of the important points in our definition is that other diagnosable conditions like ME/CFS or POTS can be part of the picture of long COVID. They are not alternative. They are, in fact, an expression of long COVID.”

Indeed, the NASEM report also introduces the term infection-associated chronic condition (IACC). This was important, Dr. Fineberg said, “because it’s the larger family of conditions of which long COVID is a part. It emphasizes a relatedness of long COVID to other conditions that can follow from a variety of infections. We also adopted the term ‘disease state’ to convey the seriousness and reality of this condition in the lives of patients.” 
 

Comments on New Definition

In a statement provided to this news organization, Lucinda Bateman, MD, and Brayden Yellman, MD, co-medical directors of the Bateman-Horne Center in Salt Lake City, said that “describing long COVID as an IACC ... not only meets the NASEM goal of allowing clinicians, researchers, and public health officials to meaningfully identify and serve all persons who suffer illness or disability in the wake of a SARS-CoV-2 infection, but also draws direct comparison to other known IACC’s (such as ME/CFS, post-treatment Lyme, POTS) that have been plaguing many for decades.”

Dr. Fineberg noted another important aspect of the NASEM report: “Our definition includes an explicit statement on equity, explaining that long COVID can affect anyone, young and old, different races, different ages, different sexes, different genders, different orientations, different socioeconomic conditions ... This does not mean that every single person is at equal risk. There are risk factors, but the important point is the universal nature of this as a condition.”

Two clinical directors of long COVID programs who were contacted by this news organization praised the new definition. Zijian Chen, MD, director of Mount Sinai’s Center for Post-COVID Care, New York, said that it’s “very similar to the definition that we have used for our clinical practice since 2020. It is very important that the broad definition helps to be inclusive of all patients that may be affected. The inclusion of children as a consideration is important as well, since there is routinely less focus on children because they tend to have less disease frequency ... The creation of a unified definition helps both with clinical practice and research.”

Nisha Viswanathan, MD, director of the long COVID program at the University of California, Los Angeles, said: “I think they left it intentionally broad for the medical practitioner to not necessarily use the definition to rule out individuals, but to perhaps use more of a clinical gestalt to help rule in this diagnosis ... I think this definition is providing clarity to health care providers on what exactly would be falling under the long-COVID diagnosis header.” 

Dr. Viswanathan also said that she anticipates this definition to help patients make their case in filing disability claims. “Because long COVID has not previously had a good fleshed-out definition, it was very easy for disability providers to reject claims for patients who continue to have symptoms ... I actually think this might help our patients ultimately in their attempt to be able to have the ability to care for themselves when they’re disabled enough to not be able to work.”

Written into the report is the expectation that the definition “will evolve as new evidence emerges and the understanding of long COVID matures.” The writing committee calls for reexamination in “no more than 3 years.” Factors that would prompt a reevaluation could include improved testing methods, discovery of medical factors and/or biomarkers that distinguish long COVID from other conditions, and new treatments. 

Meanwhile, Dr. Fineberg told this news organization, “If this definition adds to the readiness, awareness, openness, and response to the patient with long COVID, it will have done its job.” 

Dr. Fineberg, Dr. Bateman, Dr. Yellman, Dr. Viswanathan, and Dr. Chen have no relevant disclosures.

A version of this article appeared on Medscape.com.

A new broadly inclusive definition of long COVID from the National Academies of Sciences, Engineering, and Medicine (NASEM) has been developed with the aim of improving consistency, documentation, and treatment for both adults and children.

According to the 2024 NASEM definition of long COVID issued on June 11, 2024, “Long COVID is an infection-associated chronic condition that occurs after SARS-CoV-2 infection and is present for at least 3 months as a continuous, relapsing and remitting, or progressive disease state that affects one or more organ systems.” 

People with long COVID may present with one or more of a long list of symptoms, such as shortness of breath, rapid heartbeat, extreme fatigue, post-exertional malaise, or sleep disturbance and with single or multiple diagnosable conditions, including interstitial lung disease, arrhythmias, postural orthostatic tachycardia syndrome (POTS), myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS), diabetes, or autoimmune disorders. The condition can exacerbate preexisting health conditions or present as new ones. 

The definition does not require laboratory confirmation or other proof of initial infection. Long COVID can follow SARS-CoV-2 infection of any severity, including asymptomatic infections, whether or not they were initially recognized. 

Several working definitions and terms for long COVID had previously been proposed, including those from the World Health Organization (WHO) and the US Centers for Disease Control and Prevention, but no common definition or terminology had been established.

The new definition was developed at the request of the Administration for Strategic Preparedness and Response and the Office of the Assistant Secretary for Health (OASH). It was written by a multi-stakeholder panel convened by NASEM, which recommended that the new definition be universally adopted by the federal government, clinical societies and associations, public health practitioners, clinicians, payers, the drug industry, and others using the term long COVID. 

Recent surveys suggest that approximately 7% of Americans have experienced or are experiencing long COVID. “It’s millions of people,” panel chair Harvey V. Fineberg, MD, president of the Gordon and Betty Moore Foundation, told this news organization. 

The new definition “does not erase the problem of clinical judgment ... But we think this definition has the real advantage of elevating to the clinician’s mind the real likelihood in the current environment of prevalence of this virus that a presenting patient’s strange symptoms are both real and maybe related as an expression of long COVID,” Dr. Fineberg noted. 

One way this new definition differs from previous ones such as WHO’s, he said, is “they talk about a diagnosis of exclusion. One of the important points in our definition is that other diagnosable conditions like ME/CFS or POTS can be part of the picture of long COVID. They are not alternative. They are, in fact, an expression of long COVID.”

Indeed, the NASEM report also introduces the term infection-associated chronic condition (IACC). This was important, Dr. Fineberg said, “because it’s the larger family of conditions of which long COVID is a part. It emphasizes a relatedness of long COVID to other conditions that can follow from a variety of infections. We also adopted the term ‘disease state’ to convey the seriousness and reality of this condition in the lives of patients.” 
 

Comments on New Definition

In a statement provided to this news organization, Lucinda Bateman, MD, and Brayden Yellman, MD, co-medical directors of the Bateman-Horne Center in Salt Lake City, said that “describing long COVID as an IACC ... not only meets the NASEM goal of allowing clinicians, researchers, and public health officials to meaningfully identify and serve all persons who suffer illness or disability in the wake of a SARS-CoV-2 infection, but also draws direct comparison to other known IACC’s (such as ME/CFS, post-treatment Lyme, POTS) that have been plaguing many for decades.”

Dr. Fineberg noted another important aspect of the NASEM report: “Our definition includes an explicit statement on equity, explaining that long COVID can affect anyone, young and old, different races, different ages, different sexes, different genders, different orientations, different socioeconomic conditions ... This does not mean that every single person is at equal risk. There are risk factors, but the important point is the universal nature of this as a condition.”

Two clinical directors of long COVID programs who were contacted by this news organization praised the new definition. Zijian Chen, MD, director of Mount Sinai’s Center for Post-COVID Care, New York, said that it’s “very similar to the definition that we have used for our clinical practice since 2020. It is very important that the broad definition helps to be inclusive of all patients that may be affected. The inclusion of children as a consideration is important as well, since there is routinely less focus on children because they tend to have less disease frequency ... The creation of a unified definition helps both with clinical practice and research.”

Nisha Viswanathan, MD, director of the long COVID program at the University of California, Los Angeles, said: “I think they left it intentionally broad for the medical practitioner to not necessarily use the definition to rule out individuals, but to perhaps use more of a clinical gestalt to help rule in this diagnosis ... I think this definition is providing clarity to health care providers on what exactly would be falling under the long-COVID diagnosis header.” 

Dr. Viswanathan also said that she anticipates this definition to help patients make their case in filing disability claims. “Because long COVID has not previously had a good fleshed-out definition, it was very easy for disability providers to reject claims for patients who continue to have symptoms ... I actually think this might help our patients ultimately in their attempt to be able to have the ability to care for themselves when they’re disabled enough to not be able to work.”

Written into the report is the expectation that the definition “will evolve as new evidence emerges and the understanding of long COVID matures.” The writing committee calls for reexamination in “no more than 3 years.” Factors that would prompt a reevaluation could include improved testing methods, discovery of medical factors and/or biomarkers that distinguish long COVID from other conditions, and new treatments. 

Meanwhile, Dr. Fineberg told this news organization, “If this definition adds to the readiness, awareness, openness, and response to the patient with long COVID, it will have done its job.” 

Dr. Fineberg, Dr. Bateman, Dr. Yellman, Dr. Viswanathan, and Dr. Chen have no relevant disclosures.

A version of this article appeared on Medscape.com.

Stuart Malcolm, MD, a primary care physician who practices in Oregon and northern California, started seeing patients with long COVID early in the pandemic. Back then, he was frustrated by the obstacles and lack of standard diagnostic tests and treatments. Four years later, well, he still is.

“Something I learned the last few years is the logistics to get people care is really, really hard,” he said. “There’s a lot of frustration. It’s mostly frustration.”

For long COVID doctors and patients, there has been little to no progress addressing the challenges, leaving many discouraged. Researchers and clinicians now have a greater understanding of what health agencies formally call post-COVID condition, but the wide spectrum of symptoms, slow progress in launching pharmacologic clinical trials, and the research toward understanding the underlying causes mean standardized diagnostic tests and definitive treatments remain elusive.

“The frustration is that we aren’t able to help everyone with our current knowledge base. And I think the frustration lies not just with us physicians but also with patients because they’re at the point where if they tried everything, literally everything and haven’t gotten better,” said Zijian Chen, MD, director of the Mount Sinai Center for Post-COVID Care in New York City.
 

Wanted: More Funding, More Doctors, More Clinics

Between 10% and 20% of the estimated hundreds of millions of people infected worldwide with SARS-CoV-2 in the first 2 years went on to develop long-term symptoms. While many recover over time, doctors who have treated long COVID since 2020 said they see some patients still wrestling with the condition after 4 years.

The latest Centers for Disease Control and Prevention Household Pulse Survey, taken between March 5 and April 1, 2024, estimated that nearly 7% of the adult population — more than 18 million people — currently have long COVID. Data from other countries also suggest that millions have been living with long COVID for years now, and hundreds of thousands have seen their day-to-day activities significantly affected.

There is an urgent need for more funding, long COVID clinicians, multidisciplinary clinics, and education for non–long COVID physicians and specialists, doctors said. Instead, funding remains limited, clinics are closing, wait times are “horrendously long,” patients are left in limbo, and physicians are burning out.

“What’s changed in some ways is that there’s even less access to COVID rehab, which sounds crazy because there was very little to begin with,” said Alexandra Rendely, MD, a physical medicine and rehabilitation physician with the interdisciplinary Toronto Rehab, a part of the University Health Network of teaching hospitals in Toronto, Ontario, Canada.

“Patients are still being diagnosed every day, yet the resources available are becoming less and less.”

COVID-19 money earmarked during the pandemic was mostly limited to temporary emergency measures. As those funds dwindled, governments and institutions have decreased financial support. The Long COVID Moonshot campaign, organized by patients with long COVID, is pushing Congress to support $1 billion in annual research funding to close the financial chasm.
 

The Clinical Trial Conundrum

While long COVID clinics have come a long way in helping patients, gaps remain. Doctors may be unwilling to prescribe off-label treatments without proper clinical trials due to the potential risks and liabilities involved or due to the controversial or unconventional nature of the therapies, said Dr. Malcolm, who left his primary care practice more than 2 years ago to focus on long COVID.

In the absence of standard treatments, Dr. Malcolm and other doctors said they must take a trial-and-error approach in treating patients with long COVID that centers on addressing symptoms and not the underlying condition.

“There are actually a lot of treatments and a lot of them are not curative, but they can help people,” he said.

Dr. Malcolm, who is a medical director at Real Time Health Monitoring, a private clinic in the San Francisco Bay Area that specializes in long COVID and myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS), said it was important for him to be with a clinical team that understood and was supportive of his treatment decisions and was able to offer clinical support for those treatments if needed.

For physicians looking for clinical data before prescribing certain medications, the wait may be long. More than $1.5 billion in US federal funding has been earmarked to study long COVID, but the National Institutes of Health (NIH) has faced criticism from patients and scientists alike for its slow progress and emphasis on observational studies instead of research that could unravel the biological roots of long COVID. Among the clinical trials announced by the NIH’s RECOVER initiative, only a handful involve studying pharmaceutical treatments.

A 2023 editorial published in The Lancet called out the “dismal state of clinical research relative to the substantial burden of [long COVID]” and said, “we are clearly lacking tested pharmacological interventions that treat the underlying pathophysiology.” At the time of publication, it noted that of the 386 long COVID trials listed on ClinicalTrials.gov, only 12 were actually testing pharmacologic interventions.

There are also diagnostic and insurance barriers. The specialized tests that can detect long COVID anomalies are neither commonly known by primary care practitioners nor easily requested at the local lab, can be expensive, and are typically not covered by insurance, Dr. Malcolm explained.

Patients with long COVID also have the added barrier of being unable to advocate as easily because of their energy limitations, doctors said. Patients may appear outwardly fine, but fatigue and brain fog are among the many problems that cannot be measured in appearances. The condition has upended lives, some losing jobs, even homes, and the mental toll is why there is a “not insignificant” suicide rate.
 

One Patient’s 4-Year Journey

Charlie McCone, 34, used to be a tennis player and an active musician. But he’s spent the past 4 years mostly housebound, grappling with the aftermath of a SARS-CoV-2 infection he contracted in March 2020. He went from biking daily to work 10 miles and back to having at most 2 hours of energy per day.

In the first year alone, Mr. McCone saw more than two dozen doctors and specialists. The conditions now associated with long COVID, like ME/CFS, mast cell activation syndrome (a condition in which a patient experiences episodes of allergic symptoms such as hives, swelling, low blood pressure, and difficulty breathing), or dysautonomia (conditions that affect the autonomic nervous system, which controls automatic processes in the body) were not on physicians’ radars.

Then in 2021, he became bedbound for more than half a year after a Delta variant reinfection. He developed neurologic symptoms, including incapacitating fatigue, post-exertional malaise (where symptoms worsened after minimal physical or mental activity), left-sided weakness, and cognitive impairment. He stopped working altogether. But the worst was the shortness of breath he felt 24/7, even at rest. A battery of lab tests revealed nothing abnormal. He tried numerous drugs and the classic respiratory treatments.

Mr. McCone eventually connected with Dr. Malcolm over X and developed what he describes as an effective patient-doctor collaboration. When studies came out suggesting microclots were a common issue with patients with long COVID and positive outcomes were reported from anticoagulant therapy, they knew it could be one of the answers.

“After 3 weeks on [the antiplatelet drug], I was like, oh my god, my lungs are finally opening up,” said Mr. McCone. He has taken the medication for more than a year and a half, and some days he doesn’t even think about his respiratory symptoms.

“That trial-and-error process is just really long and hard and costly,” said Dr. Malcolm.

Today, fatigue and cognitive stamina are Mr. McCone’s main challenges, and he is far from recovered.

“[I had a] very fulfilling, happy life and now, it’s hard to think about. I’ve come a long way with my mental health and all this, but I’ve lost 4 years,” Mr. McCone said. “The prospect of me being here when I’m 40 seems very real ... so it’s pretty devastating.”
 

Lessons Learned, Hope Amid Ongoing Research

Despite the daunting obstacle, doctors said the science has come a long way for a new disease. We now know long COVID is likely caused by a combination of triggers, including viral reservoir in the tissue, inflammation, autoimmunity, and microclots; severity of infection is not necessarily an accurate risk factor predictor — long COVID can strike even those who had a mild infection; upward of 200 symptoms have been identified; and we know more about potential biomarkers that could lead to better diagnostic tools.

Unlike many other diseases and conditions with standard treatment protocols, long COVID treatments are typically aimed at addressing individual symptoms.

“It is very detailed and individualized to the patient’s specific symptoms and to the patient’s specific needs,” Dr. Rendely said. Symptoms can also fluctuate, relapse, or wax and wane, for example, so what ails a patient at their first doctor’s appointment could be completely different at the next appointment 2 months later.

Doctors are still hopeful the RECOVER research, which includes trials that look at autonomic and cognitive dysfunctions, will pave the way for more effective long COVID therapies. In Canada, Dr. Rendely is also eying the RECLAIM trial that is currently testing the effectiveness of pentoxifylline, which helps blood flow, and ibudilast, an anti-inflammatory drug.

Doctors are also hopeful when they see patients who have made “tremendous gains” or even full recoveries through their clinics. “It’s a new diagnosis, so I always tell my patients to think of this as a journey because I’m learning along with you,” said Jai Marathe, MD, an infectious disease physician at Boston Medical Center and an assistant professor of infectious diseases at Boston University Chobanian & Avedisian School of Medicine.

“Now we have 4 years of experience, but at the same time, no two long COVID patients are alike.”

Long COVID has also changed the way physicians view healthcare and how they practice medicine.

“I am a completely different person than I used to be because of this illness, and I don’t even have it. That is how profoundly it has affected how I view the universe,” said Dr. Malcolm. “I’ve been doing this for 4 years, and I’m very hopeful. But I don’t think about this in terms of months anymore. I think about this in terms of years.”

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

Stuart Malcolm, MD, a primary care physician who practices in Oregon and northern California, started seeing patients with long COVID early in the pandemic. Back then, he was frustrated by the obstacles and lack of standard diagnostic tests and treatments. Four years later, well, he still is.

“Something I learned the last few years is the logistics to get people care is really, really hard,” he said. “There’s a lot of frustration. It’s mostly frustration.”

For long COVID doctors and patients, there has been little to no progress addressing the challenges, leaving many discouraged. Researchers and clinicians now have a greater understanding of what health agencies formally call post-COVID condition, but the wide spectrum of symptoms, slow progress in launching pharmacologic clinical trials, and the research toward understanding the underlying causes mean standardized diagnostic tests and definitive treatments remain elusive.

“The frustration is that we aren’t able to help everyone with our current knowledge base. And I think the frustration lies not just with us physicians but also with patients because they’re at the point where if they tried everything, literally everything and haven’t gotten better,” said Zijian Chen, MD, director of the Mount Sinai Center for Post-COVID Care in New York City.
 

Wanted: More Funding, More Doctors, More Clinics

Between 10% and 20% of the estimated hundreds of millions of people infected worldwide with SARS-CoV-2 in the first 2 years went on to develop long-term symptoms. While many recover over time, doctors who have treated long COVID since 2020 said they see some patients still wrestling with the condition after 4 years.

The latest Centers for Disease Control and Prevention Household Pulse Survey, taken between March 5 and April 1, 2024, estimated that nearly 7% of the adult population — more than 18 million people — currently have long COVID. Data from other countries also suggest that millions have been living with long COVID for years now, and hundreds of thousands have seen their day-to-day activities significantly affected.

There is an urgent need for more funding, long COVID clinicians, multidisciplinary clinics, and education for non–long COVID physicians and specialists, doctors said. Instead, funding remains limited, clinics are closing, wait times are “horrendously long,” patients are left in limbo, and physicians are burning out.

“What’s changed in some ways is that there’s even less access to COVID rehab, which sounds crazy because there was very little to begin with,” said Alexandra Rendely, MD, a physical medicine and rehabilitation physician with the interdisciplinary Toronto Rehab, a part of the University Health Network of teaching hospitals in Toronto, Ontario, Canada.

“Patients are still being diagnosed every day, yet the resources available are becoming less and less.”

COVID-19 money earmarked during the pandemic was mostly limited to temporary emergency measures. As those funds dwindled, governments and institutions have decreased financial support. The Long COVID Moonshot campaign, organized by patients with long COVID, is pushing Congress to support $1 billion in annual research funding to close the financial chasm.
 

The Clinical Trial Conundrum

While long COVID clinics have come a long way in helping patients, gaps remain. Doctors may be unwilling to prescribe off-label treatments without proper clinical trials due to the potential risks and liabilities involved or due to the controversial or unconventional nature of the therapies, said Dr. Malcolm, who left his primary care practice more than 2 years ago to focus on long COVID.

In the absence of standard treatments, Dr. Malcolm and other doctors said they must take a trial-and-error approach in treating patients with long COVID that centers on addressing symptoms and not the underlying condition.

“There are actually a lot of treatments and a lot of them are not curative, but they can help people,” he said.

Dr. Malcolm, who is a medical director at Real Time Health Monitoring, a private clinic in the San Francisco Bay Area that specializes in long COVID and myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS), said it was important for him to be with a clinical team that understood and was supportive of his treatment decisions and was able to offer clinical support for those treatments if needed.

For physicians looking for clinical data before prescribing certain medications, the wait may be long. More than $1.5 billion in US federal funding has been earmarked to study long COVID, but the National Institutes of Health (NIH) has faced criticism from patients and scientists alike for its slow progress and emphasis on observational studies instead of research that could unravel the biological roots of long COVID. Among the clinical trials announced by the NIH’s RECOVER initiative, only a handful involve studying pharmaceutical treatments.

A 2023 editorial published in The Lancet called out the “dismal state of clinical research relative to the substantial burden of [long COVID]” and said, “we are clearly lacking tested pharmacological interventions that treat the underlying pathophysiology.” At the time of publication, it noted that of the 386 long COVID trials listed on ClinicalTrials.gov, only 12 were actually testing pharmacologic interventions.

There are also diagnostic and insurance barriers. The specialized tests that can detect long COVID anomalies are neither commonly known by primary care practitioners nor easily requested at the local lab, can be expensive, and are typically not covered by insurance, Dr. Malcolm explained.

Patients with long COVID also have the added barrier of being unable to advocate as easily because of their energy limitations, doctors said. Patients may appear outwardly fine, but fatigue and brain fog are among the many problems that cannot be measured in appearances. The condition has upended lives, some losing jobs, even homes, and the mental toll is why there is a “not insignificant” suicide rate.
 

One Patient’s 4-Year Journey

Charlie McCone, 34, used to be a tennis player and an active musician. But he’s spent the past 4 years mostly housebound, grappling with the aftermath of a SARS-CoV-2 infection he contracted in March 2020. He went from biking daily to work 10 miles and back to having at most 2 hours of energy per day.

In the first year alone, Mr. McCone saw more than two dozen doctors and specialists. The conditions now associated with long COVID, like ME/CFS, mast cell activation syndrome (a condition in which a patient experiences episodes of allergic symptoms such as hives, swelling, low blood pressure, and difficulty breathing), or dysautonomia (conditions that affect the autonomic nervous system, which controls automatic processes in the body) were not on physicians’ radars.

Then in 2021, he became bedbound for more than half a year after a Delta variant reinfection. He developed neurologic symptoms, including incapacitating fatigue, post-exertional malaise (where symptoms worsened after minimal physical or mental activity), left-sided weakness, and cognitive impairment. He stopped working altogether. But the worst was the shortness of breath he felt 24/7, even at rest. A battery of lab tests revealed nothing abnormal. He tried numerous drugs and the classic respiratory treatments.

Mr. McCone eventually connected with Dr. Malcolm over X and developed what he describes as an effective patient-doctor collaboration. When studies came out suggesting microclots were a common issue with patients with long COVID and positive outcomes were reported from anticoagulant therapy, they knew it could be one of the answers.

“After 3 weeks on [the antiplatelet drug], I was like, oh my god, my lungs are finally opening up,” said Mr. McCone. He has taken the medication for more than a year and a half, and some days he doesn’t even think about his respiratory symptoms.

“That trial-and-error process is just really long and hard and costly,” said Dr. Malcolm.

Today, fatigue and cognitive stamina are Mr. McCone’s main challenges, and he is far from recovered.

“[I had a] very fulfilling, happy life and now, it’s hard to think about. I’ve come a long way with my mental health and all this, but I’ve lost 4 years,” Mr. McCone said. “The prospect of me being here when I’m 40 seems very real ... so it’s pretty devastating.”
 

Lessons Learned, Hope Amid Ongoing Research

Despite the daunting obstacle, doctors said the science has come a long way for a new disease. We now know long COVID is likely caused by a combination of triggers, including viral reservoir in the tissue, inflammation, autoimmunity, and microclots; severity of infection is not necessarily an accurate risk factor predictor — long COVID can strike even those who had a mild infection; upward of 200 symptoms have been identified; and we know more about potential biomarkers that could lead to better diagnostic tools.

Unlike many other diseases and conditions with standard treatment protocols, long COVID treatments are typically aimed at addressing individual symptoms.

“It is very detailed and individualized to the patient’s specific symptoms and to the patient’s specific needs,” Dr. Rendely said. Symptoms can also fluctuate, relapse, or wax and wane, for example, so what ails a patient at their first doctor’s appointment could be completely different at the next appointment 2 months later.

Doctors are still hopeful the RECOVER research, which includes trials that look at autonomic and cognitive dysfunctions, will pave the way for more effective long COVID therapies. In Canada, Dr. Rendely is also eying the RECLAIM trial that is currently testing the effectiveness of pentoxifylline, which helps blood flow, and ibudilast, an anti-inflammatory drug.

Doctors are also hopeful when they see patients who have made “tremendous gains” or even full recoveries through their clinics. “It’s a new diagnosis, so I always tell my patients to think of this as a journey because I’m learning along with you,” said Jai Marathe, MD, an infectious disease physician at Boston Medical Center and an assistant professor of infectious diseases at Boston University Chobanian & Avedisian School of Medicine.

“Now we have 4 years of experience, but at the same time, no two long COVID patients are alike.”

Long COVID has also changed the way physicians view healthcare and how they practice medicine.

“I am a completely different person than I used to be because of this illness, and I don’t even have it. That is how profoundly it has affected how I view the universe,” said Dr. Malcolm. “I’ve been doing this for 4 years, and I’m very hopeful. But I don’t think about this in terms of months anymore. I think about this in terms of years.”

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

Stuart Malcolm, MD, a primary care physician who practices in Oregon and northern California, started seeing patients with long COVID early in the pandemic. Back then, he was frustrated by the obstacles and lack of standard diagnostic tests and treatments. Four years later, well, he still is.

“Something I learned the last few years is the logistics to get people care is really, really hard,” he said. “There’s a lot of frustration. It’s mostly frustration.”

For long COVID doctors and patients, there has been little to no progress addressing the challenges, leaving many discouraged. Researchers and clinicians now have a greater understanding of what health agencies formally call post-COVID condition, but the wide spectrum of symptoms, slow progress in launching pharmacologic clinical trials, and the research toward understanding the underlying causes mean standardized diagnostic tests and definitive treatments remain elusive.

“The frustration is that we aren’t able to help everyone with our current knowledge base. And I think the frustration lies not just with us physicians but also with patients because they’re at the point where if they tried everything, literally everything and haven’t gotten better,” said Zijian Chen, MD, director of the Mount Sinai Center for Post-COVID Care in New York City.
 

Wanted: More Funding, More Doctors, More Clinics

Between 10% and 20% of the estimated hundreds of millions of people infected worldwide with SARS-CoV-2 in the first 2 years went on to develop long-term symptoms. While many recover over time, doctors who have treated long COVID since 2020 said they see some patients still wrestling with the condition after 4 years.

The latest Centers for Disease Control and Prevention Household Pulse Survey, taken between March 5 and April 1, 2024, estimated that nearly 7% of the adult population — more than 18 million people — currently have long COVID. Data from other countries also suggest that millions have been living with long COVID for years now, and hundreds of thousands have seen their day-to-day activities significantly affected.

There is an urgent need for more funding, long COVID clinicians, multidisciplinary clinics, and education for non–long COVID physicians and specialists, doctors said. Instead, funding remains limited, clinics are closing, wait times are “horrendously long,” patients are left in limbo, and physicians are burning out.

“What’s changed in some ways is that there’s even less access to COVID rehab, which sounds crazy because there was very little to begin with,” said Alexandra Rendely, MD, a physical medicine and rehabilitation physician with the interdisciplinary Toronto Rehab, a part of the University Health Network of teaching hospitals in Toronto, Ontario, Canada.

“Patients are still being diagnosed every day, yet the resources available are becoming less and less.”

COVID-19 money earmarked during the pandemic was mostly limited to temporary emergency measures. As those funds dwindled, governments and institutions have decreased financial support. The Long COVID Moonshot campaign, organized by patients with long COVID, is pushing Congress to support $1 billion in annual research funding to close the financial chasm.
 

The Clinical Trial Conundrum

While long COVID clinics have come a long way in helping patients, gaps remain. Doctors may be unwilling to prescribe off-label treatments without proper clinical trials due to the potential risks and liabilities involved or due to the controversial or unconventional nature of the therapies, said Dr. Malcolm, who left his primary care practice more than 2 years ago to focus on long COVID.

In the absence of standard treatments, Dr. Malcolm and other doctors said they must take a trial-and-error approach in treating patients with long COVID that centers on addressing symptoms and not the underlying condition.

“There are actually a lot of treatments and a lot of them are not curative, but they can help people,” he said.

Dr. Malcolm, who is a medical director at Real Time Health Monitoring, a private clinic in the San Francisco Bay Area that specializes in long COVID and myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS), said it was important for him to be with a clinical team that understood and was supportive of his treatment decisions and was able to offer clinical support for those treatments if needed.

For physicians looking for clinical data before prescribing certain medications, the wait may be long. More than $1.5 billion in US federal funding has been earmarked to study long COVID, but the National Institutes of Health (NIH) has faced criticism from patients and scientists alike for its slow progress and emphasis on observational studies instead of research that could unravel the biological roots of long COVID. Among the clinical trials announced by the NIH’s RECOVER initiative, only a handful involve studying pharmaceutical treatments.

A 2023 editorial published in The Lancet called out the “dismal state of clinical research relative to the substantial burden of [long COVID]” and said, “we are clearly lacking tested pharmacological interventions that treat the underlying pathophysiology.” At the time of publication, it noted that of the 386 long COVID trials listed on ClinicalTrials.gov, only 12 were actually testing pharmacologic interventions.

There are also diagnostic and insurance barriers. The specialized tests that can detect long COVID anomalies are neither commonly known by primary care practitioners nor easily requested at the local lab, can be expensive, and are typically not covered by insurance, Dr. Malcolm explained.

Patients with long COVID also have the added barrier of being unable to advocate as easily because of their energy limitations, doctors said. Patients may appear outwardly fine, but fatigue and brain fog are among the many problems that cannot be measured in appearances. The condition has upended lives, some losing jobs, even homes, and the mental toll is why there is a “not insignificant” suicide rate.
 

One Patient’s 4-Year Journey

Charlie McCone, 34, used to be a tennis player and an active musician. But he’s spent the past 4 years mostly housebound, grappling with the aftermath of a SARS-CoV-2 infection he contracted in March 2020. He went from biking daily to work 10 miles and back to having at most 2 hours of energy per day.

In the first year alone, Mr. McCone saw more than two dozen doctors and specialists. The conditions now associated with long COVID, like ME/CFS, mast cell activation syndrome (a condition in which a patient experiences episodes of allergic symptoms such as hives, swelling, low blood pressure, and difficulty breathing), or dysautonomia (conditions that affect the autonomic nervous system, which controls automatic processes in the body) were not on physicians’ radars.

Then in 2021, he became bedbound for more than half a year after a Delta variant reinfection. He developed neurologic symptoms, including incapacitating fatigue, post-exertional malaise (where symptoms worsened after minimal physical or mental activity), left-sided weakness, and cognitive impairment. He stopped working altogether. But the worst was the shortness of breath he felt 24/7, even at rest. A battery of lab tests revealed nothing abnormal. He tried numerous drugs and the classic respiratory treatments.

Mr. McCone eventually connected with Dr. Malcolm over X and developed what he describes as an effective patient-doctor collaboration. When studies came out suggesting microclots were a common issue with patients with long COVID and positive outcomes were reported from anticoagulant therapy, they knew it could be one of the answers.

“After 3 weeks on [the antiplatelet drug], I was like, oh my god, my lungs are finally opening up,” said Mr. McCone. He has taken the medication for more than a year and a half, and some days he doesn’t even think about his respiratory symptoms.

“That trial-and-error process is just really long and hard and costly,” said Dr. Malcolm.

Today, fatigue and cognitive stamina are Mr. McCone’s main challenges, and he is far from recovered.

“[I had a] very fulfilling, happy life and now, it’s hard to think about. I’ve come a long way with my mental health and all this, but I’ve lost 4 years,” Mr. McCone said. “The prospect of me being here when I’m 40 seems very real ... so it’s pretty devastating.”
 

Lessons Learned, Hope Amid Ongoing Research

Despite the daunting obstacle, doctors said the science has come a long way for a new disease. We now know long COVID is likely caused by a combination of triggers, including viral reservoir in the tissue, inflammation, autoimmunity, and microclots; severity of infection is not necessarily an accurate risk factor predictor — long COVID can strike even those who had a mild infection; upward of 200 symptoms have been identified; and we know more about potential biomarkers that could lead to better diagnostic tools.

Unlike many other diseases and conditions with standard treatment protocols, long COVID treatments are typically aimed at addressing individual symptoms.

“It is very detailed and individualized to the patient’s specific symptoms and to the patient’s specific needs,” Dr. Rendely said. Symptoms can also fluctuate, relapse, or wax and wane, for example, so what ails a patient at their first doctor’s appointment could be completely different at the next appointment 2 months later.

Doctors are still hopeful the RECOVER research, which includes trials that look at autonomic and cognitive dysfunctions, will pave the way for more effective long COVID therapies. In Canada, Dr. Rendely is also eying the RECLAIM trial that is currently testing the effectiveness of pentoxifylline, which helps blood flow, and ibudilast, an anti-inflammatory drug.

Doctors are also hopeful when they see patients who have made “tremendous gains” or even full recoveries through their clinics. “It’s a new diagnosis, so I always tell my patients to think of this as a journey because I’m learning along with you,” said Jai Marathe, MD, an infectious disease physician at Boston Medical Center and an assistant professor of infectious diseases at Boston University Chobanian & Avedisian School of Medicine.

“Now we have 4 years of experience, but at the same time, no two long COVID patients are alike.”

Long COVID has also changed the way physicians view healthcare and how they practice medicine.

“I am a completely different person than I used to be because of this illness, and I don’t even have it. That is how profoundly it has affected how I view the universe,” said Dr. Malcolm. “I’ve been doing this for 4 years, and I’m very hopeful. But I don’t think about this in terms of months anymore. I think about this in terms of years.”

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

BARCELONA, SPAIN — Streptococcus pneumoniae, the bacteria that causes pneumococcal disease, is sixfold more likely to colonize adults older than 60 years who have regular contact with children than those who do not, data from a community-based study showed.

However, there is “no clear evidence of adult-to-adult transmission,” and the researchers, led by Anne L. Wyllie, PhD, from the Yale School of Public Health, New Haven, Connecticut, noted that the study results suggest “the main benefit of adult pneumococcal conjugate vaccine (PCV) immunization is to directly protect adults who are exposed to children, who still carry and transmit some vaccine-type pneumococci despite successful pediatric national immunization programs.”

The data show that relatively high pneumococcus carriage rates are seen in people who have regular contact with children, who have had contact in the previous 2 weeks, and who have had contact for extended periods, Dr. Wyllie explained.

Preschoolers in particular were found to be most likely to transmit pneumococcus to older adults. “It is the 24- to 59-month-olds who are most associated with pneumococcal carriage, more than 1- to 2-year-olds,” she reported. However, transmission rates from children younger than 1 year are higher than those from children aged 1-2 years, she added.

The findings were presented at the European Society of Clinical Microbiology and Infectious Diseases (ESCMID) 2024 global conference, formerly known as the ECCMID conference.
 

Originally Designed to Investigate Adult-to-Adult Transmission

The researchers wanted to understand the sources and dynamics of transmission, as well as the risk factors for pneumococcal disease in older adults, to help predict the effect of PCVs in people older than 60 years.

Although “we designed the study to specifically look at transmission between adults, in the end, we were presented with a very unique scenario” — restricted social mixing as a result of the COVID pandemic — during which “no community activities were happening,” Dr. Wyllie said. Because of this, the team was able to determine “the source of acquisition or transmission to the older adults was, very likely, coming from contact with children.”

Pneumococci are commonly found in respiratory tracts of healthy people. The US Centers for Disease Control and Prevention estimated that 20%-60% of school-aged children may be colonized compared with only 5%-10% of adults without children.

The longitudinal study was conducted among household pairs, such as married couples who were both aged at least 60 years and who did not have people younger than 60 years living in the household, in New Haven over two winter seasons: 2020-2021 and 2021-2022.

Self-collected saliva samples were assessed, and surveys on social behaviors and health were completed every 2 weeks for a 10-week period (with six study visits). The saliva sampling method was used because the researchers considered it to be more effective than samples from nasopharyngeal swabs. Quantitative polymerase chain reaction assays were used to test the saliva samples for the presence of pneumococcal DNA (pneumococcus genes piaB and lytA) and the diversity of pneumococcal strains (36 serotypes were targeted).
 

Strongly Suggestive of Transmission From Children to Older Adults

Of the 121 adults living in 61 households who were enrolled in the study, 62 adults participated in both seasons. Mean age was 70.9 years (range, 60-86 years), 51% of participants were women, and 85% were White.

Overall, 52 of 1088 (4.8%) samples tested positive for pneumococcus, and 27 of 121 (22.3%) adults were colonized on at least one sampling visit. Some were colonized at multiple timepoints, and two were colonized throughout the 10-week sampling period. Of the two participants who were colonized at five of six timepoints, one reported daily contact with children younger than 5 years and children aged 5-9 years in the two study seasons. This person was also positive at three of six sampling points during the first study season.

There were five instances in which both members of the household were carriers in the same season, although not necessarily at the same timepoint. Numbers were too small to determine whether transmission had occurred between the household pairs.

Contact with a 24- to 59-month-old child (older than 2 years but younger than 5 years) had the strongest association with elevated odds of carrying pneumococcus, the authors reported in their preprint, although the frequency and intensity of contact also mattered.

At any sampled time (point prevalence), pneumococcal carriage was substantially — just over sixfold — higher among older adults who had contact with children daily or every few days (10%) than among those who had no contact with children (1.6%).

In particular, contact between adults and children younger than 5 years and children aged 5-9 years was found to lead to elevated point prevalences of 13.8% and 14.1%, respectively. Pneumococcal carriage in children older than 10 years was lower, with a point prevalence of 8.3%.

The younger the child, the greater the point prevalence; point prevalences were 13.8% for samples from children aged 1 year and younger, 10.5% for samples from children aged 1-2 years, and 17.8% for children aged 2-5 years.

Carriage prevalence was higher in older adults who reported daily contact with children (15.7%) or contact every few days (14.0%) than in those who reported contact with children only once or twice a month (4.5%) or never (1.8%), they wrote.

“Older people who have a lot of contact with kids and are more susceptible to respiratory viruses can get a secondary infection from pneumococcus, especially during the cold and flu seasons. Vaccination can help to protect them or lessen severity of the illness,” Wyllie pointed out.

However, adult PCV immunization may not have a major impact on onward transmission to other adults, the authors wrote in their preprint.

This study supports prior work demonstrating that pneumococcal colonization is greater in households with children than in those without, said Stephen Pelton, MD, a pediatric infectious disease specialist from Boston University schools of medicine and public health. “The unique aspect is that Dr. Wyllie’s group has looked at individuals over age 60 and used the most sensitive methods currently available to detect pneumococcal carriage.”

“At the most recent ISPPD [International Society of Pneumonia and Pneumococcal Diseases conference], the role of adult-to-adult transmission in the community was discussed. This study confirms the critical role children play in community transmission of the pneumococcus,” Dr. Pelton noted.

Dr. Wyllie received consulting and/or advisory board fees from Pfizer, Merck, Diasorin, PPS Health, Primary Health, Co-Diagnostics, and Global Diagnostic Systems for work unrelated to this project and is the principal investigator on research grants from Pfizer, Merck, NIH RADx-UP, and SalivaDirect, Inc. to Yale University and from NIH RADx, Balvi.io, and Shield T3 to SalivaDirect, Inc. Dr. Pelton received honoraria from Merck, Pfizer, Sanofi, and GSK for participation in Pneumococcal Advisory Boards and DSMB (Sanofi). Boston Medical Center received grant funding for investigator-initiated research from Merck and Pfizer.
 

A version of this article appeared on Medscape.com.

BARCELONA, SPAIN — Streptococcus pneumoniae, the bacteria that causes pneumococcal disease, is sixfold more likely to colonize adults older than 60 years who have regular contact with children than those who do not, data from a community-based study showed.

However, there is “no clear evidence of adult-to-adult transmission,” and the researchers, led by Anne L. Wyllie, PhD, from the Yale School of Public Health, New Haven, Connecticut, noted that the study results suggest “the main benefit of adult pneumococcal conjugate vaccine (PCV) immunization is to directly protect adults who are exposed to children, who still carry and transmit some vaccine-type pneumococci despite successful pediatric national immunization programs.”

The data show that relatively high pneumococcus carriage rates are seen in people who have regular contact with children, who have had contact in the previous 2 weeks, and who have had contact for extended periods, Dr. Wyllie explained.

Preschoolers in particular were found to be most likely to transmit pneumococcus to older adults. “It is the 24- to 59-month-olds who are most associated with pneumococcal carriage, more than 1- to 2-year-olds,” she reported. However, transmission rates from children younger than 1 year are higher than those from children aged 1-2 years, she added.

The findings were presented at the European Society of Clinical Microbiology and Infectious Diseases (ESCMID) 2024 global conference, formerly known as the ECCMID conference.
 

Originally Designed to Investigate Adult-to-Adult Transmission

The researchers wanted to understand the sources and dynamics of transmission, as well as the risk factors for pneumococcal disease in older adults, to help predict the effect of PCVs in people older than 60 years.

Although “we designed the study to specifically look at transmission between adults, in the end, we were presented with a very unique scenario” — restricted social mixing as a result of the COVID pandemic — during which “no community activities were happening,” Dr. Wyllie said. Because of this, the team was able to determine “the source of acquisition or transmission to the older adults was, very likely, coming from contact with children.”

Pneumococci are commonly found in respiratory tracts of healthy people. The US Centers for Disease Control and Prevention estimated that 20%-60% of school-aged children may be colonized compared with only 5%-10% of adults without children.

The longitudinal study was conducted among household pairs, such as married couples who were both aged at least 60 years and who did not have people younger than 60 years living in the household, in New Haven over two winter seasons: 2020-2021 and 2021-2022.

Self-collected saliva samples were assessed, and surveys on social behaviors and health were completed every 2 weeks for a 10-week period (with six study visits). The saliva sampling method was used because the researchers considered it to be more effective than samples from nasopharyngeal swabs. Quantitative polymerase chain reaction assays were used to test the saliva samples for the presence of pneumococcal DNA (pneumococcus genes piaB and lytA) and the diversity of pneumococcal strains (36 serotypes were targeted).
 

Strongly Suggestive of Transmission From Children to Older Adults

Of the 121 adults living in 61 households who were enrolled in the study, 62 adults participated in both seasons. Mean age was 70.9 years (range, 60-86 years), 51% of participants were women, and 85% were White.

Overall, 52 of 1088 (4.8%) samples tested positive for pneumococcus, and 27 of 121 (22.3%) adults were colonized on at least one sampling visit. Some were colonized at multiple timepoints, and two were colonized throughout the 10-week sampling period. Of the two participants who were colonized at five of six timepoints, one reported daily contact with children younger than 5 years and children aged 5-9 years in the two study seasons. This person was also positive at three of six sampling points during the first study season.

There were five instances in which both members of the household were carriers in the same season, although not necessarily at the same timepoint. Numbers were too small to determine whether transmission had occurred between the household pairs.

Contact with a 24- to 59-month-old child (older than 2 years but younger than 5 years) had the strongest association with elevated odds of carrying pneumococcus, the authors reported in their preprint, although the frequency and intensity of contact also mattered.

At any sampled time (point prevalence), pneumococcal carriage was substantially — just over sixfold — higher among older adults who had contact with children daily or every few days (10%) than among those who had no contact with children (1.6%).

In particular, contact between adults and children younger than 5 years and children aged 5-9 years was found to lead to elevated point prevalences of 13.8% and 14.1%, respectively. Pneumococcal carriage in children older than 10 years was lower, with a point prevalence of 8.3%.

The younger the child, the greater the point prevalence; point prevalences were 13.8% for samples from children aged 1 year and younger, 10.5% for samples from children aged 1-2 years, and 17.8% for children aged 2-5 years.

Carriage prevalence was higher in older adults who reported daily contact with children (15.7%) or contact every few days (14.0%) than in those who reported contact with children only once or twice a month (4.5%) or never (1.8%), they wrote.

“Older people who have a lot of contact with kids and are more susceptible to respiratory viruses can get a secondary infection from pneumococcus, especially during the cold and flu seasons. Vaccination can help to protect them or lessen severity of the illness,” Wyllie pointed out.

However, adult PCV immunization may not have a major impact on onward transmission to other adults, the authors wrote in their preprint.

This study supports prior work demonstrating that pneumococcal colonization is greater in households with children than in those without, said Stephen Pelton, MD, a pediatric infectious disease specialist from Boston University schools of medicine and public health. “The unique aspect is that Dr. Wyllie’s group has looked at individuals over age 60 and used the most sensitive methods currently available to detect pneumococcal carriage.”

“At the most recent ISPPD [International Society of Pneumonia and Pneumococcal Diseases conference], the role of adult-to-adult transmission in the community was discussed. This study confirms the critical role children play in community transmission of the pneumococcus,” Dr. Pelton noted.

Dr. Wyllie received consulting and/or advisory board fees from Pfizer, Merck, Diasorin, PPS Health, Primary Health, Co-Diagnostics, and Global Diagnostic Systems for work unrelated to this project and is the principal investigator on research grants from Pfizer, Merck, NIH RADx-UP, and SalivaDirect, Inc. to Yale University and from NIH RADx, Balvi.io, and Shield T3 to SalivaDirect, Inc. Dr. Pelton received honoraria from Merck, Pfizer, Sanofi, and GSK for participation in Pneumococcal Advisory Boards and DSMB (Sanofi). Boston Medical Center received grant funding for investigator-initiated research from Merck and Pfizer.
 

A version of this article appeared on Medscape.com.

BARCELONA, SPAIN — Streptococcus pneumoniae, the bacteria that causes pneumococcal disease, is sixfold more likely to colonize adults older than 60 years who have regular contact with children than those who do not, data from a community-based study showed.

However, there is “no clear evidence of adult-to-adult transmission,” and the researchers, led by Anne L. Wyllie, PhD, from the Yale School of Public Health, New Haven, Connecticut, noted that the study results suggest “the main benefit of adult pneumococcal conjugate vaccine (PCV) immunization is to directly protect adults who are exposed to children, who still carry and transmit some vaccine-type pneumococci despite successful pediatric national immunization programs.”

The data show that relatively high pneumococcus carriage rates are seen in people who have regular contact with children, who have had contact in the previous 2 weeks, and who have had contact for extended periods, Dr. Wyllie explained.

Preschoolers in particular were found to be most likely to transmit pneumococcus to older adults. “It is the 24- to 59-month-olds who are most associated with pneumococcal carriage, more than 1- to 2-year-olds,” she reported. However, transmission rates from children younger than 1 year are higher than those from children aged 1-2 years, she added.

The findings were presented at the European Society of Clinical Microbiology and Infectious Diseases (ESCMID) 2024 global conference, formerly known as the ECCMID conference.
 

Originally Designed to Investigate Adult-to-Adult Transmission

The researchers wanted to understand the sources and dynamics of transmission, as well as the risk factors for pneumococcal disease in older adults, to help predict the effect of PCVs in people older than 60 years.

Although “we designed the study to specifically look at transmission between adults, in the end, we were presented with a very unique scenario” — restricted social mixing as a result of the COVID pandemic — during which “no community activities were happening,” Dr. Wyllie said. Because of this, the team was able to determine “the source of acquisition or transmission to the older adults was, very likely, coming from contact with children.”

Pneumococci are commonly found in respiratory tracts of healthy people. The US Centers for Disease Control and Prevention estimated that 20%-60% of school-aged children may be colonized compared with only 5%-10% of adults without children.

The longitudinal study was conducted among household pairs, such as married couples who were both aged at least 60 years and who did not have people younger than 60 years living in the household, in New Haven over two winter seasons: 2020-2021 and 2021-2022.

Self-collected saliva samples were assessed, and surveys on social behaviors and health were completed every 2 weeks for a 10-week period (with six study visits). The saliva sampling method was used because the researchers considered it to be more effective than samples from nasopharyngeal swabs. Quantitative polymerase chain reaction assays were used to test the saliva samples for the presence of pneumococcal DNA (pneumococcus genes piaB and lytA) and the diversity of pneumococcal strains (36 serotypes were targeted).
 

Strongly Suggestive of Transmission From Children to Older Adults

Of the 121 adults living in 61 households who were enrolled in the study, 62 adults participated in both seasons. Mean age was 70.9 years (range, 60-86 years), 51% of participants were women, and 85% were White.

Overall, 52 of 1088 (4.8%) samples tested positive for pneumococcus, and 27 of 121 (22.3%) adults were colonized on at least one sampling visit. Some were colonized at multiple timepoints, and two were colonized throughout the 10-week sampling period. Of the two participants who were colonized at five of six timepoints, one reported daily contact with children younger than 5 years and children aged 5-9 years in the two study seasons. This person was also positive at three of six sampling points during the first study season.

There were five instances in which both members of the household were carriers in the same season, although not necessarily at the same timepoint. Numbers were too small to determine whether transmission had occurred between the household pairs.

Contact with a 24- to 59-month-old child (older than 2 years but younger than 5 years) had the strongest association with elevated odds of carrying pneumococcus, the authors reported in their preprint, although the frequency and intensity of contact also mattered.

At any sampled time (point prevalence), pneumococcal carriage was substantially — just over sixfold — higher among older adults who had contact with children daily or every few days (10%) than among those who had no contact with children (1.6%).

In particular, contact between adults and children younger than 5 years and children aged 5-9 years was found to lead to elevated point prevalences of 13.8% and 14.1%, respectively. Pneumococcal carriage in children older than 10 years was lower, with a point prevalence of 8.3%.

The younger the child, the greater the point prevalence; point prevalences were 13.8% for samples from children aged 1 year and younger, 10.5% for samples from children aged 1-2 years, and 17.8% for children aged 2-5 years.

Carriage prevalence was higher in older adults who reported daily contact with children (15.7%) or contact every few days (14.0%) than in those who reported contact with children only once or twice a month (4.5%) or never (1.8%), they wrote.

“Older people who have a lot of contact with kids and are more susceptible to respiratory viruses can get a secondary infection from pneumococcus, especially during the cold and flu seasons. Vaccination can help to protect them or lessen severity of the illness,” Wyllie pointed out.

However, adult PCV immunization may not have a major impact on onward transmission to other adults, the authors wrote in their preprint.

This study supports prior work demonstrating that pneumococcal colonization is greater in households with children than in those without, said Stephen Pelton, MD, a pediatric infectious disease specialist from Boston University schools of medicine and public health. “The unique aspect is that Dr. Wyllie’s group has looked at individuals over age 60 and used the most sensitive methods currently available to detect pneumococcal carriage.”

“At the most recent ISPPD [International Society of Pneumonia and Pneumococcal Diseases conference], the role of adult-to-adult transmission in the community was discussed. This study confirms the critical role children play in community transmission of the pneumococcus,” Dr. Pelton noted.

Dr. Wyllie received consulting and/or advisory board fees from Pfizer, Merck, Diasorin, PPS Health, Primary Health, Co-Diagnostics, and Global Diagnostic Systems for work unrelated to this project and is the principal investigator on research grants from Pfizer, Merck, NIH RADx-UP, and SalivaDirect, Inc. to Yale University and from NIH RADx, Balvi.io, and Shield T3 to SalivaDirect, Inc. Dr. Pelton received honoraria from Merck, Pfizer, Sanofi, and GSK for participation in Pneumococcal Advisory Boards and DSMB (Sanofi). Boston Medical Center received grant funding for investigator-initiated research from Merck and Pfizer.
 

A version of this article appeared on Medscape.com.

When doctors and patients consider the appendix, it’s often with urgency. In cases of appendicitis, the clock could be ticking down to a life-threatening burst. Thus, despite recent research suggesting antibiotics could be an alternative therapy, appendectomy remains standard for uncomplicated appendicitis.

But what if removing the appendix could raise the risk for gastrointestinal (GI) diseases like irritable bowel syndrome and colorectal cancer? That’s what some emerging science suggests. And though the research is early and mixed, it’s enough to give some health professionals pause.

“If there’s no reason to remove the appendix, then it’s better to have one,” said Heather Smith, PhD, a comparative anatomist at Midwestern University, Glendale, Arizona. Preemptive removal is not supported by the evidence, she said.

To be fair, we’ve come a long way since 1928, when American physician Miles Breuer, MD, suggested that people with infected appendixes should be left to perish, so as to remove their inferior DNA from the gene pool (he called such people “uncivilized” and “candidates for extinction”). Charles Darwin, while less radical, believed the appendix was at best useless — a mere vestige of our ancestors switching diets from leaves to fruits.

What we know now is that the appendix isn’t just a troublesome piece of worthless flesh. Instead, it may act as a safe house for friendly gut bacteria and a training camp for the immune system. It also appears to play a role in several medical conditions, from ulcerative colitis and colorectal cancer to Parkinson’s disease and lupus. The roughly 300,000 Americans who undergo appendectomy each year should be made aware of this, some experts say. But the frustrating truth is, scientists are still trying to figure out in which cases having an appendix is protective and in which we may be better off without it.
 

A ‘Worm’ as Intestinal Protection

The appendix is a blind pouch (meaning its ending is closed off) that extends from the large intestine. Not all mammals have one; it’s been found in several species of primates and rodents, as well as in rabbits, wombats, and Florida manatees, among others (dogs and cats don’t have it). While a human appendix “looks like a little worm,” Dr. Smith said, these anatomical structures come in various sizes and shapes. Some are thick, as in a beaver, while others are long and spiraling, like a rabbit’s.

Comparative anatomy studies reveal that the appendix has evolved independently at least 29 times throughout mammalian evolution. This suggests that “it has some kind of an adaptive function,” Dr. Smith said. When French scientists analyzed data from 258 species of mammals, they discovered that those that possess an appendix live longer than those without one. A possible explanation, the researchers wrote, may lie with the appendix’s role in preventing diarrhea.

Their 2023 study supported this hypothesis. Based on veterinary records of 45 different species of primates housed in a French zoo, the scientists established that primates with appendixes are far less likely to suffer severe diarrhea than those that don’t possess this organ. The appendix, it appears, might be our tiny weapon against bowel troubles.

For immunologist William Parker, PhD, a visiting scholar at the University of North Carolina at Chapel Hill, these data are “about as good as we could hope for” in support of the idea that the appendix might protect mammals from GI problems. An experiment on humans would be unethical, Dr. Parker said. But observational studies offer clues.

One study showed that compared with people with an intact appendix, young adults with a history of appendectomy have more than double the risk of developing a serious infection with non-typhoidal Salmonella of the kind that would require hospitalization.
 

A ‘Safe House’ for Bacteria

Such studies add weight to a theory that Dr. Parker and his colleagues developed back in 2007: That the appendix acts as a “safe house” for beneficial gut bacteria.

Think of the colon as a wide pipe, Dr. Parker said, that may become contaminated with a pathogen such as Salmonella. Diarrhea follows, and the pipe gets repeatedly flushed, wiping everything clean, including your friendly gut microbiome. Luckily, “you’ve got this little offshoot of that pipe,” where the flow can’t really get in “because it’s so constricted,” Dr. Parker said. The friendly gut microbes can survive inside the appendix and repopulate the colon once diarrhea is over. Dr. Parker and his colleagues found that the human appendix contains a thick layer of beneficial bacteria. “They were right where we predicted they would be,” he said.

This safe house hypothesis could explain why the gut microbiome may be different in people who no longer have an appendix. In one small study, people who’d had an appendectomy had a less diverse microbiome, with a lower abundance of beneficial strains such as Butyricicoccus and Barnesiella, than did those with intact appendixes.

The appendix likely has a second function, too, Dr. Smith said: It may serve as a training camp for the immune system. “When there is an invading pathogen in the gut, it helps the GI system to mount the immune response,” she said. The human appendix is rich in special cells known as M cells. These act as scouts, detecting and capturing invasive bacteria and viruses and presenting them to the body’s defense team, such as the T lymphocytes.

If the appendix shelters beneficial bacteria and boosts immune response, that may explain its links to various diseases. According to an epidemiological study from Taiwan,patients who underwent an appendectomy have a 46% higher risk of developing irritable bowel syndrome (IBS) — a disease associated with a low abundance of Butyricicoccus bacteria. This is why, the study authors wrote, doctors should pay careful attention to people who’ve had their appendixes removed, monitoring them for potential symptoms of IBS.

The same database helped uncover other connections between appendectomy and disease. For one, there was type 2 diabetes: Within 3 years of the surgery, patients under 30 had double the risk of developing this disorder. Then there was lupus: While those who underwent appendectomy generally had higher risk for this autoimmune disease, women were particularly affected.
 

The Contentious Connections

The most heated scientific discussion surrounds the links between the appendix and conditions such as Parkinson’s disease, ulcerative colitis, and colorectal cancer. A small 2019 study showed, for example, that appendectomy may improve symptoms of certain forms of ulcerative colitis that don’t respond to standard medical treatments. A third of patients improved after their appendix was removed, and 17% fully recovered.

Why? According to Dr. Parker, appendectomy may work for ulcerative colitis because it’s “a way of suppressing the immune system, especially in the lower intestinal areas.” A 2023 meta-analysis found that people who’d had their appendix removed before being diagnosed with ulcerative colitis were less likely to need their colon removed later on.

Such a procedure may have a serious side effect, however: Colorectal cancer. French scientists discovered that removing the appendix may reduce the numbers of certain immune cells called CD3+ and CD8+ T cells, causing a weakened immune surveillance. As a result, tumor cells might escape detection.

Yet the links between appendix removal and cancer are far from clear. A recent meta-analysis found that while people with appendectomies generally had a higher risk for colorectal cancer, for Europeans, these effects were insignificant. In fact, removal of the appendix actually protected European women from this particular form of cancer. For Parker, such mixed results may stem from the fact that treatments and populations vary widely. The issue “may depend on complex social and medical factors,” Dr. Parker said.

Things also appear complicated with Parkinson’s disease — another condition linked to the appendix. A large epidemiological study showed that appendectomy is associated with a lower risk for Parkinson’s disease and a delayed age of Parkinson’s onset. It also found that a normal appendix contains α-synuclein, a protein that may accumulate in the brain and contribute to the development of Parkinson’s. “Although α-synuclein is toxic when in the brain, it appears to be quite normal when present in the appendix,” said Luis Vitetta, PhD, MD, a clinical epidemiologist at the University of Sydney, Camperdown, Australia. Yet, not all studies find that removing the appendix lowers the risk for Parkinson’s. In fact, some show the opposite results.
 

How Should Doctors View the Appendix?

Even with these mysteries and contradictions, Dr. Vitetta said, a healthy appendix in a healthy body appears to be protective. This is why, he said, when someone is diagnosed with appendicitis, careful assessment is essential before surgery is performed.

“Perhaps an antibiotic can actually help fix it,” he said. A 2020 study published in The New England Journal of Medicine showed that antibiotics may indeed be a good alternative to surgery for the treatment of appendicitis. “We don’t want necessarily to remove an appendix that could be beneficial,” Dr. Smith said.

The many links between the appendix and various diseases mean that doctors should be more vigilant when treating patients who’ve had this organ removed, Dr. Parker said. “When a patient loses an appendix, depending on their environment, there may be effects on infection and cancer. So they might need more regular checkups,” he said. This could include monitoring for IBS and colorectal cancer.

What’s more, Dr. Parker believes that research on the appendix puts even more emphasis on the need to protect the gut microbiome — such as taking probiotics with antibiotics. And while we are still a long way from understanding how exactly this worm-like structure affects various diseases, one thing appears quite certain: The appendix is not useless. “If Darwin had the information that we have, he would not have drawn these conclusions,” Dr. Parker said.
 

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

When doctors and patients consider the appendix, it’s often with urgency. In cases of appendicitis, the clock could be ticking down to a life-threatening burst. Thus, despite recent research suggesting antibiotics could be an alternative therapy, appendectomy remains standard for uncomplicated appendicitis.

But what if removing the appendix could raise the risk for gastrointestinal (GI) diseases like irritable bowel syndrome and colorectal cancer? That’s what some emerging science suggests. And though the research is early and mixed, it’s enough to give some health professionals pause.

“If there’s no reason to remove the appendix, then it’s better to have one,” said Heather Smith, PhD, a comparative anatomist at Midwestern University, Glendale, Arizona. Preemptive removal is not supported by the evidence, she said.

To be fair, we’ve come a long way since 1928, when American physician Miles Breuer, MD, suggested that people with infected appendixes should be left to perish, so as to remove their inferior DNA from the gene pool (he called such people “uncivilized” and “candidates for extinction”). Charles Darwin, while less radical, believed the appendix was at best useless — a mere vestige of our ancestors switching diets from leaves to fruits.

What we know now is that the appendix isn’t just a troublesome piece of worthless flesh. Instead, it may act as a safe house for friendly gut bacteria and a training camp for the immune system. It also appears to play a role in several medical conditions, from ulcerative colitis and colorectal cancer to Parkinson’s disease and lupus. The roughly 300,000 Americans who undergo appendectomy each year should be made aware of this, some experts say. But the frustrating truth is, scientists are still trying to figure out in which cases having an appendix is protective and in which we may be better off without it.
 

A ‘Worm’ as Intestinal Protection

The appendix is a blind pouch (meaning its ending is closed off) that extends from the large intestine. Not all mammals have one; it’s been found in several species of primates and rodents, as well as in rabbits, wombats, and Florida manatees, among others (dogs and cats don’t have it). While a human appendix “looks like a little worm,” Dr. Smith said, these anatomical structures come in various sizes and shapes. Some are thick, as in a beaver, while others are long and spiraling, like a rabbit’s.

Comparative anatomy studies reveal that the appendix has evolved independently at least 29 times throughout mammalian evolution. This suggests that “it has some kind of an adaptive function,” Dr. Smith said. When French scientists analyzed data from 258 species of mammals, they discovered that those that possess an appendix live longer than those without one. A possible explanation, the researchers wrote, may lie with the appendix’s role in preventing diarrhea.

Their 2023 study supported this hypothesis. Based on veterinary records of 45 different species of primates housed in a French zoo, the scientists established that primates with appendixes are far less likely to suffer severe diarrhea than those that don’t possess this organ. The appendix, it appears, might be our tiny weapon against bowel troubles.

For immunologist William Parker, PhD, a visiting scholar at the University of North Carolina at Chapel Hill, these data are “about as good as we could hope for” in support of the idea that the appendix might protect mammals from GI problems. An experiment on humans would be unethical, Dr. Parker said. But observational studies offer clues.

One study showed that compared with people with an intact appendix, young adults with a history of appendectomy have more than double the risk of developing a serious infection with non-typhoidal Salmonella of the kind that would require hospitalization.
 

A ‘Safe House’ for Bacteria

Such studies add weight to a theory that Dr. Parker and his colleagues developed back in 2007: That the appendix acts as a “safe house” for beneficial gut bacteria.

Think of the colon as a wide pipe, Dr. Parker said, that may become contaminated with a pathogen such as Salmonella. Diarrhea follows, and the pipe gets repeatedly flushed, wiping everything clean, including your friendly gut microbiome. Luckily, “you’ve got this little offshoot of that pipe,” where the flow can’t really get in “because it’s so constricted,” Dr. Parker said. The friendly gut microbes can survive inside the appendix and repopulate the colon once diarrhea is over. Dr. Parker and his colleagues found that the human appendix contains a thick layer of beneficial bacteria. “They were right where we predicted they would be,” he said.

This safe house hypothesis could explain why the gut microbiome may be different in people who no longer have an appendix. In one small study, people who’d had an appendectomy had a less diverse microbiome, with a lower abundance of beneficial strains such as Butyricicoccus and Barnesiella, than did those with intact appendixes.

The appendix likely has a second function, too, Dr. Smith said: It may serve as a training camp for the immune system. “When there is an invading pathogen in the gut, it helps the GI system to mount the immune response,” she said. The human appendix is rich in special cells known as M cells. These act as scouts, detecting and capturing invasive bacteria and viruses and presenting them to the body’s defense team, such as the T lymphocytes.

If the appendix shelters beneficial bacteria and boosts immune response, that may explain its links to various diseases. According to an epidemiological study from Taiwan,patients who underwent an appendectomy have a 46% higher risk of developing irritable bowel syndrome (IBS) — a disease associated with a low abundance of Butyricicoccus bacteria. This is why, the study authors wrote, doctors should pay careful attention to people who’ve had their appendixes removed, monitoring them for potential symptoms of IBS.

The same database helped uncover other connections between appendectomy and disease. For one, there was type 2 diabetes: Within 3 years of the surgery, patients under 30 had double the risk of developing this disorder. Then there was lupus: While those who underwent appendectomy generally had higher risk for this autoimmune disease, women were particularly affected.
 

The Contentious Connections

The most heated scientific discussion surrounds the links between the appendix and conditions such as Parkinson’s disease, ulcerative colitis, and colorectal cancer. A small 2019 study showed, for example, that appendectomy may improve symptoms of certain forms of ulcerative colitis that don’t respond to standard medical treatments. A third of patients improved after their appendix was removed, and 17% fully recovered.

Why? According to Dr. Parker, appendectomy may work for ulcerative colitis because it’s “a way of suppressing the immune system, especially in the lower intestinal areas.” A 2023 meta-analysis found that people who’d had their appendix removed before being diagnosed with ulcerative colitis were less likely to need their colon removed later on.

Such a procedure may have a serious side effect, however: Colorectal cancer. French scientists discovered that removing the appendix may reduce the numbers of certain immune cells called CD3+ and CD8+ T cells, causing a weakened immune surveillance. As a result, tumor cells might escape detection.

Yet the links between appendix removal and cancer are far from clear. A recent meta-analysis found that while people with appendectomies generally had a higher risk for colorectal cancer, for Europeans, these effects were insignificant. In fact, removal of the appendix actually protected European women from this particular form of cancer. For Parker, such mixed results may stem from the fact that treatments and populations vary widely. The issue “may depend on complex social and medical factors,” Dr. Parker said.

Things also appear complicated with Parkinson’s disease — another condition linked to the appendix. A large epidemiological study showed that appendectomy is associated with a lower risk for Parkinson’s disease and a delayed age of Parkinson’s onset. It also found that a normal appendix contains α-synuclein, a protein that may accumulate in the brain and contribute to the development of Parkinson’s. “Although α-synuclein is toxic when in the brain, it appears to be quite normal when present in the appendix,” said Luis Vitetta, PhD, MD, a clinical epidemiologist at the University of Sydney, Camperdown, Australia. Yet, not all studies find that removing the appendix lowers the risk for Parkinson’s. In fact, some show the opposite results.
 

How Should Doctors View the Appendix?

Even with these mysteries and contradictions, Dr. Vitetta said, a healthy appendix in a healthy body appears to be protective. This is why, he said, when someone is diagnosed with appendicitis, careful assessment is essential before surgery is performed.

“Perhaps an antibiotic can actually help fix it,” he said. A 2020 study published in The New England Journal of Medicine showed that antibiotics may indeed be a good alternative to surgery for the treatment of appendicitis. “We don’t want necessarily to remove an appendix that could be beneficial,” Dr. Smith said.

The many links between the appendix and various diseases mean that doctors should be more vigilant when treating patients who’ve had this organ removed, Dr. Parker said. “When a patient loses an appendix, depending on their environment, there may be effects on infection and cancer. So they might need more regular checkups,” he said. This could include monitoring for IBS and colorectal cancer.

What’s more, Dr. Parker believes that research on the appendix puts even more emphasis on the need to protect the gut microbiome — such as taking probiotics with antibiotics. And while we are still a long way from understanding how exactly this worm-like structure affects various diseases, one thing appears quite certain: The appendix is not useless. “If Darwin had the information that we have, he would not have drawn these conclusions,” Dr. Parker said.
 

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

When doctors and patients consider the appendix, it’s often with urgency. In cases of appendicitis, the clock could be ticking down to a life-threatening burst. Thus, despite recent research suggesting antibiotics could be an alternative therapy, appendectomy remains standard for uncomplicated appendicitis.

But what if removing the appendix could raise the risk for gastrointestinal (GI) diseases like irritable bowel syndrome and colorectal cancer? That’s what some emerging science suggests. And though the research is early and mixed, it’s enough to give some health professionals pause.

“If there’s no reason to remove the appendix, then it’s better to have one,” said Heather Smith, PhD, a comparative anatomist at Midwestern University, Glendale, Arizona. Preemptive removal is not supported by the evidence, she said.

To be fair, we’ve come a long way since 1928, when American physician Miles Breuer, MD, suggested that people with infected appendixes should be left to perish, so as to remove their inferior DNA from the gene pool (he called such people “uncivilized” and “candidates for extinction”). Charles Darwin, while less radical, believed the appendix was at best useless — a mere vestige of our ancestors switching diets from leaves to fruits.

What we know now is that the appendix isn’t just a troublesome piece of worthless flesh. Instead, it may act as a safe house for friendly gut bacteria and a training camp for the immune system. It also appears to play a role in several medical conditions, from ulcerative colitis and colorectal cancer to Parkinson’s disease and lupus. The roughly 300,000 Americans who undergo appendectomy each year should be made aware of this, some experts say. But the frustrating truth is, scientists are still trying to figure out in which cases having an appendix is protective and in which we may be better off without it.
 

A ‘Worm’ as Intestinal Protection

The appendix is a blind pouch (meaning its ending is closed off) that extends from the large intestine. Not all mammals have one; it’s been found in several species of primates and rodents, as well as in rabbits, wombats, and Florida manatees, among others (dogs and cats don’t have it). While a human appendix “looks like a little worm,” Dr. Smith said, these anatomical structures come in various sizes and shapes. Some are thick, as in a beaver, while others are long and spiraling, like a rabbit’s.

Comparative anatomy studies reveal that the appendix has evolved independently at least 29 times throughout mammalian evolution. This suggests that “it has some kind of an adaptive function,” Dr. Smith said. When French scientists analyzed data from 258 species of mammals, they discovered that those that possess an appendix live longer than those without one. A possible explanation, the researchers wrote, may lie with the appendix’s role in preventing diarrhea.

Their 2023 study supported this hypothesis. Based on veterinary records of 45 different species of primates housed in a French zoo, the scientists established that primates with appendixes are far less likely to suffer severe diarrhea than those that don’t possess this organ. The appendix, it appears, might be our tiny weapon against bowel troubles.

For immunologist William Parker, PhD, a visiting scholar at the University of North Carolina at Chapel Hill, these data are “about as good as we could hope for” in support of the idea that the appendix might protect mammals from GI problems. An experiment on humans would be unethical, Dr. Parker said. But observational studies offer clues.

One study showed that compared with people with an intact appendix, young adults with a history of appendectomy have more than double the risk of developing a serious infection with non-typhoidal Salmonella of the kind that would require hospitalization.
 

A ‘Safe House’ for Bacteria

Such studies add weight to a theory that Dr. Parker and his colleagues developed back in 2007: That the appendix acts as a “safe house” for beneficial gut bacteria.

Think of the colon as a wide pipe, Dr. Parker said, that may become contaminated with a pathogen such as Salmonella. Diarrhea follows, and the pipe gets repeatedly flushed, wiping everything clean, including your friendly gut microbiome. Luckily, “you’ve got this little offshoot of that pipe,” where the flow can’t really get in “because it’s so constricted,” Dr. Parker said. The friendly gut microbes can survive inside the appendix and repopulate the colon once diarrhea is over. Dr. Parker and his colleagues found that the human appendix contains a thick layer of beneficial bacteria. “They were right where we predicted they would be,” he said.

This safe house hypothesis could explain why the gut microbiome may be different in people who no longer have an appendix. In one small study, people who’d had an appendectomy had a less diverse microbiome, with a lower abundance of beneficial strains such as Butyricicoccus and Barnesiella, than did those with intact appendixes.

The appendix likely has a second function, too, Dr. Smith said: It may serve as a training camp for the immune system. “When there is an invading pathogen in the gut, it helps the GI system to mount the immune response,” she said. The human appendix is rich in special cells known as M cells. These act as scouts, detecting and capturing invasive bacteria and viruses and presenting them to the body’s defense team, such as the T lymphocytes.

If the appendix shelters beneficial bacteria and boosts immune response, that may explain its links to various diseases. According to an epidemiological study from Taiwan,patients who underwent an appendectomy have a 46% higher risk of developing irritable bowel syndrome (IBS) — a disease associated with a low abundance of Butyricicoccus bacteria. This is why, the study authors wrote, doctors should pay careful attention to people who’ve had their appendixes removed, monitoring them for potential symptoms of IBS.

The same database helped uncover other connections between appendectomy and disease. For one, there was type 2 diabetes: Within 3 years of the surgery, patients under 30 had double the risk of developing this disorder. Then there was lupus: While those who underwent appendectomy generally had higher risk for this autoimmune disease, women were particularly affected.
 

The Contentious Connections

The most heated scientific discussion surrounds the links between the appendix and conditions such as Parkinson’s disease, ulcerative colitis, and colorectal cancer. A small 2019 study showed, for example, that appendectomy may improve symptoms of certain forms of ulcerative colitis that don’t respond to standard medical treatments. A third of patients improved after their appendix was removed, and 17% fully recovered.

Why? According to Dr. Parker, appendectomy may work for ulcerative colitis because it’s “a way of suppressing the immune system, especially in the lower intestinal areas.” A 2023 meta-analysis found that people who’d had their appendix removed before being diagnosed with ulcerative colitis were less likely to need their colon removed later on.

Such a procedure may have a serious side effect, however: Colorectal cancer. French scientists discovered that removing the appendix may reduce the numbers of certain immune cells called CD3+ and CD8+ T cells, causing a weakened immune surveillance. As a result, tumor cells might escape detection.

Yet the links between appendix removal and cancer are far from clear. A recent meta-analysis found that while people with appendectomies generally had a higher risk for colorectal cancer, for Europeans, these effects were insignificant. In fact, removal of the appendix actually protected European women from this particular form of cancer. For Parker, such mixed results may stem from the fact that treatments and populations vary widely. The issue “may depend on complex social and medical factors,” Dr. Parker said.

Things also appear complicated with Parkinson’s disease — another condition linked to the appendix. A large epidemiological study showed that appendectomy is associated with a lower risk for Parkinson’s disease and a delayed age of Parkinson’s onset. It also found that a normal appendix contains α-synuclein, a protein that may accumulate in the brain and contribute to the development of Parkinson’s. “Although α-synuclein is toxic when in the brain, it appears to be quite normal when present in the appendix,” said Luis Vitetta, PhD, MD, a clinical epidemiologist at the University of Sydney, Camperdown, Australia. Yet, not all studies find that removing the appendix lowers the risk for Parkinson’s. In fact, some show the opposite results.
 

How Should Doctors View the Appendix?

Even with these mysteries and contradictions, Dr. Vitetta said, a healthy appendix in a healthy body appears to be protective. This is why, he said, when someone is diagnosed with appendicitis, careful assessment is essential before surgery is performed.

“Perhaps an antibiotic can actually help fix it,” he said. A 2020 study published in The New England Journal of Medicine showed that antibiotics may indeed be a good alternative to surgery for the treatment of appendicitis. “We don’t want necessarily to remove an appendix that could be beneficial,” Dr. Smith said.

The many links between the appendix and various diseases mean that doctors should be more vigilant when treating patients who’ve had this organ removed, Dr. Parker said. “When a patient loses an appendix, depending on their environment, there may be effects on infection and cancer. So they might need more regular checkups,” he said. This could include monitoring for IBS and colorectal cancer.

What’s more, Dr. Parker believes that research on the appendix puts even more emphasis on the need to protect the gut microbiome — such as taking probiotics with antibiotics. And while we are still a long way from understanding how exactly this worm-like structure affects various diseases, one thing appears quite certain: The appendix is not useless. “If Darwin had the information that we have, he would not have drawn these conclusions,” Dr. Parker said.
 

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