All adult patients with primary sclerosing cholangitis should be screened at least annually for cholangiocarcinoma and gallbladder cancer, particularly in the first year after their diagnosis, according to a clinical practice update published in Clinical Gastroenterology and Hepatology.

Individuals with primary sclerosing cholangitis have a 400-fold higher risk of cholangiocarcinoma, compared with the general population, and around one-third of cancers are detected within 1 year of the cholangitis diagnosis, Christopher L. Bowlus, MD, of the University of California, Davis, and coauthors wrote.

The clinical practice update from the American Gastroenterological Association was in response to the observation that, while there is significant evidence for an increasing incidence of cirrhosis, hepatic decompensation, hepatocellular carcinoma, and liver transplant listing among patients with primary sclerosing cholangitis, there is a lack of good evidence to guide cholangiocarcinoma surveillance in these patients.

“The low prevalence and long duration of PSC [primary sclerosing cholangitis] present substantial barriers to better understanding risk stratification, developing biomarkers, and measuring the impact surveillance has on clinical outcomes,” they wrote.

The first recommendation was that surveillance for cholangiocarcinoma and gallbladder cancer should be considered in all adult patients with primary sclerosing cholangitis, regardless of their disease stage. The authors especially emphasized the importance of surveillance in the first year after a diagnosis of primary sclerosing cholangitis, in patients who also have ulcerative colitis, and in those diagnosed at an older age.

They cited one study that found regular surveillance of patients with primary sclerosing cholangitis was associated with significantly higher 5-year survival rates, compared with those no regular screening (68% vs. 20%; P less than .0061).

In terms of surveillance modalities, the update suggested 6- to 12-monthly imaging of the biliary tree with ultrasound computed tomography, computed tomography, or magnetic resonance imaging – with or without serum carbohydrate antigen 19-9. However the authors wrote that MRI was often preferred to CT because of its superior sensitivity.

They advised against endoscopic retrograde cholangiopancreatography with brush cytology for routine surveillance because of procedural risks. On the other hand, they suggested this procedure, with or without fluorescence in situ hybridization analysis and/or cholangioscopy, could be used for investigation.

“In addition to ERCP [endoscopic retrograde cholangiopancreatography] with brushings, endoscopic ultrasound, intraductal ultrasonography, and cholangioscopy may be used to direct biopsy sampling,” they wrote. Symptoms such as increasing cholestatic biochemistry values, jaundice, fever, right upper quadrant pain, or pruritus should trigger evaluation for cholangiocarcinoma.

However the authors advised “great caution” with the use of fine-needle aspiration of perihilar biliary strictures in liver transplant candidates because of the risk of tumor seeding if the lesion turned out to be cholangiocarcinoma.

On the question of cholangiocarcinoma surveillance in pediatric patients and those with small-duct primary sclerosing cholangitis, the authors wrote that cholangiocarcinoma was so rare in these patients that routine cholangiocarcinoma surveillance was not required.

The clinical update also looked at the prevalence and risk factors for gallbladder cancer, which affects around 2% of individuals with primary sclerosing cholangitis. Two studies found gallbladder polyps in 10%-17% of patients, but the authors noted that “the optimal modality for diagnosis of gallbladder polyps in PSC remains unknown”.

“Because of the high risk of malignancy in gallbladder mass lesions and a 5-year survival rate of 5% to 10% for gallbladder cancer, patients should undergo annual US [ultrasound] screening,” they wrote.

They said the question of whether to perform a cholecystectomy in patients with gallbladder polyps should be guided by the size and growth of the polyps because there is an increased risk of gallbladder cancer in polyps larger than 8 mm and by the clinical status of the patient.

Finally, the update examined the issue of hepatocellular carcinoma in patients with primary sclerosing cholangitis, which – while rare – may increase with the presence of cirrhosis.

The authors advised that patients with primary sclerosing cholangitis and cirrhosis should undergo surveillance for hepatocellular carcinoma every 6 months with ultrasound, CT, or MRI.

“We anticipate that with the development of large patient cohorts, advances in uncovering genetic and other risk factors for cholangiocarcinoma, and development of effective treatments for PSC, further refinement of this practice update will be required.”

Two authors declared consultancies, grants and research contracts with the pharmaceutical sector. No other conflicts of interest were declared.

SOURCE: Bowlus C et al. Clin Gastroenterol Hepatol. 2019 Jul 12. doi 10.1016/j.cgh.2019.07.011.

All adult patients with primary sclerosing cholangitis should be screened at least annually for cholangiocarcinoma and gallbladder cancer, particularly in the first year after their diagnosis, according to a clinical practice update published in Clinical Gastroenterology and Hepatology.

Individuals with primary sclerosing cholangitis have a 400-fold higher risk of cholangiocarcinoma, compared with the general population, and around one-third of cancers are detected within 1 year of the cholangitis diagnosis, Christopher L. Bowlus, MD, of the University of California, Davis, and coauthors wrote.

The clinical practice update from the American Gastroenterological Association was in response to the observation that, while there is significant evidence for an increasing incidence of cirrhosis, hepatic decompensation, hepatocellular carcinoma, and liver transplant listing among patients with primary sclerosing cholangitis, there is a lack of good evidence to guide cholangiocarcinoma surveillance in these patients.

“The low prevalence and long duration of PSC [primary sclerosing cholangitis] present substantial barriers to better understanding risk stratification, developing biomarkers, and measuring the impact surveillance has on clinical outcomes,” they wrote.

The first recommendation was that surveillance for cholangiocarcinoma and gallbladder cancer should be considered in all adult patients with primary sclerosing cholangitis, regardless of their disease stage. The authors especially emphasized the importance of surveillance in the first year after a diagnosis of primary sclerosing cholangitis, in patients who also have ulcerative colitis, and in those diagnosed at an older age.

They cited one study that found regular surveillance of patients with primary sclerosing cholangitis was associated with significantly higher 5-year survival rates, compared with those no regular screening (68% vs. 20%; P less than .0061).

In terms of surveillance modalities, the update suggested 6- to 12-monthly imaging of the biliary tree with ultrasound computed tomography, computed tomography, or magnetic resonance imaging – with or without serum carbohydrate antigen 19-9. However the authors wrote that MRI was often preferred to CT because of its superior sensitivity.

They advised against endoscopic retrograde cholangiopancreatography with brush cytology for routine surveillance because of procedural risks. On the other hand, they suggested this procedure, with or without fluorescence in situ hybridization analysis and/or cholangioscopy, could be used for investigation.

“In addition to ERCP [endoscopic retrograde cholangiopancreatography] with brushings, endoscopic ultrasound, intraductal ultrasonography, and cholangioscopy may be used to direct biopsy sampling,” they wrote. Symptoms such as increasing cholestatic biochemistry values, jaundice, fever, right upper quadrant pain, or pruritus should trigger evaluation for cholangiocarcinoma.

However the authors advised “great caution” with the use of fine-needle aspiration of perihilar biliary strictures in liver transplant candidates because of the risk of tumor seeding if the lesion turned out to be cholangiocarcinoma.

On the question of cholangiocarcinoma surveillance in pediatric patients and those with small-duct primary sclerosing cholangitis, the authors wrote that cholangiocarcinoma was so rare in these patients that routine cholangiocarcinoma surveillance was not required.

The clinical update also looked at the prevalence and risk factors for gallbladder cancer, which affects around 2% of individuals with primary sclerosing cholangitis. Two studies found gallbladder polyps in 10%-17% of patients, but the authors noted that “the optimal modality for diagnosis of gallbladder polyps in PSC remains unknown”.

“Because of the high risk of malignancy in gallbladder mass lesions and a 5-year survival rate of 5% to 10% for gallbladder cancer, patients should undergo annual US [ultrasound] screening,” they wrote.

They said the question of whether to perform a cholecystectomy in patients with gallbladder polyps should be guided by the size and growth of the polyps because there is an increased risk of gallbladder cancer in polyps larger than 8 mm and by the clinical status of the patient.

Finally, the update examined the issue of hepatocellular carcinoma in patients with primary sclerosing cholangitis, which – while rare – may increase with the presence of cirrhosis.

The authors advised that patients with primary sclerosing cholangitis and cirrhosis should undergo surveillance for hepatocellular carcinoma every 6 months with ultrasound, CT, or MRI.

“We anticipate that with the development of large patient cohorts, advances in uncovering genetic and other risk factors for cholangiocarcinoma, and development of effective treatments for PSC, further refinement of this practice update will be required.”

Two authors declared consultancies, grants and research contracts with the pharmaceutical sector. No other conflicts of interest were declared.

SOURCE: Bowlus C et al. Clin Gastroenterol Hepatol. 2019 Jul 12. doi 10.1016/j.cgh.2019.07.011.

All adult patients with primary sclerosing cholangitis should be screened at least annually for cholangiocarcinoma and gallbladder cancer, particularly in the first year after their diagnosis, according to a clinical practice update published in Clinical Gastroenterology and Hepatology.

Individuals with primary sclerosing cholangitis have a 400-fold higher risk of cholangiocarcinoma, compared with the general population, and around one-third of cancers are detected within 1 year of the cholangitis diagnosis, Christopher L. Bowlus, MD, of the University of California, Davis, and coauthors wrote.

The clinical practice update from the American Gastroenterological Association was in response to the observation that, while there is significant evidence for an increasing incidence of cirrhosis, hepatic decompensation, hepatocellular carcinoma, and liver transplant listing among patients with primary sclerosing cholangitis, there is a lack of good evidence to guide cholangiocarcinoma surveillance in these patients.

“The low prevalence and long duration of PSC [primary sclerosing cholangitis] present substantial barriers to better understanding risk stratification, developing biomarkers, and measuring the impact surveillance has on clinical outcomes,” they wrote.

The first recommendation was that surveillance for cholangiocarcinoma and gallbladder cancer should be considered in all adult patients with primary sclerosing cholangitis, regardless of their disease stage. The authors especially emphasized the importance of surveillance in the first year after a diagnosis of primary sclerosing cholangitis, in patients who also have ulcerative colitis, and in those diagnosed at an older age.

They cited one study that found regular surveillance of patients with primary sclerosing cholangitis was associated with significantly higher 5-year survival rates, compared with those no regular screening (68% vs. 20%; P less than .0061).

In terms of surveillance modalities, the update suggested 6- to 12-monthly imaging of the biliary tree with ultrasound computed tomography, computed tomography, or magnetic resonance imaging – with or without serum carbohydrate antigen 19-9. However the authors wrote that MRI was often preferred to CT because of its superior sensitivity.

They advised against endoscopic retrograde cholangiopancreatography with brush cytology for routine surveillance because of procedural risks. On the other hand, they suggested this procedure, with or without fluorescence in situ hybridization analysis and/or cholangioscopy, could be used for investigation.

“In addition to ERCP [endoscopic retrograde cholangiopancreatography] with brushings, endoscopic ultrasound, intraductal ultrasonography, and cholangioscopy may be used to direct biopsy sampling,” they wrote. Symptoms such as increasing cholestatic biochemistry values, jaundice, fever, right upper quadrant pain, or pruritus should trigger evaluation for cholangiocarcinoma.

However the authors advised “great caution” with the use of fine-needle aspiration of perihilar biliary strictures in liver transplant candidates because of the risk of tumor seeding if the lesion turned out to be cholangiocarcinoma.

On the question of cholangiocarcinoma surveillance in pediatric patients and those with small-duct primary sclerosing cholangitis, the authors wrote that cholangiocarcinoma was so rare in these patients that routine cholangiocarcinoma surveillance was not required.

The clinical update also looked at the prevalence and risk factors for gallbladder cancer, which affects around 2% of individuals with primary sclerosing cholangitis. Two studies found gallbladder polyps in 10%-17% of patients, but the authors noted that “the optimal modality for diagnosis of gallbladder polyps in PSC remains unknown”.

“Because of the high risk of malignancy in gallbladder mass lesions and a 5-year survival rate of 5% to 10% for gallbladder cancer, patients should undergo annual US [ultrasound] screening,” they wrote.

They said the question of whether to perform a cholecystectomy in patients with gallbladder polyps should be guided by the size and growth of the polyps because there is an increased risk of gallbladder cancer in polyps larger than 8 mm and by the clinical status of the patient.

Finally, the update examined the issue of hepatocellular carcinoma in patients with primary sclerosing cholangitis, which – while rare – may increase with the presence of cirrhosis.

The authors advised that patients with primary sclerosing cholangitis and cirrhosis should undergo surveillance for hepatocellular carcinoma every 6 months with ultrasound, CT, or MRI.

“We anticipate that with the development of large patient cohorts, advances in uncovering genetic and other risk factors for cholangiocarcinoma, and development of effective treatments for PSC, further refinement of this practice update will be required.”

Two authors declared consultancies, grants and research contracts with the pharmaceutical sector. No other conflicts of interest were declared.

SOURCE: Bowlus C et al. Clin Gastroenterol Hepatol. 2019 Jul 12. doi 10.1016/j.cgh.2019.07.011.

Adult athletes who underwent knee surgery and had higher levels of preoperative pain catastrophizing were significantly less likely to return to preinjury activity, based on data from 101 individuals.

decade3d/Thinkstock

Pain is highly subjective, and pain perception can play a role in postsurgical outcomes, but the relationships among preoperative pain perception and short-term outcomes including returning to sports have not been well-studied, wrote Joshua S. Everhart, MD, of The Ohio State University Wexner Medical Center, Columbus, and colleagues.

In a study published in the Journal of Science and Medicine in Sport, the researchers assessed 101 adult athletes who underwent knee surgery at a single center. The average age of the patients was 33 years, and 49 were women.

Pain perception and coping were assessed via the McGill Pain questionnaire (SF-MPQ), Pain Catastrophizing Scale (PCS), Pain Coping Measure (PCM), and the brief COPE subscales of acceptance, denial, positive reframing, and use of instrumental support.

Patients who were severe pain catastrophizers (defined as scores greater than 36 on the Pain Catastrophizing Scale) had increased odds of not returning to a similar level of sport (OR 11.3).

Higher scores on the brief COPE subscale of “use of instrumental support” (instruments designed to help patients cope with pain) had a protective effect on returning to preinjury activity (OR 0.72 per point increase). However, higher COPE-denial scores were significantly associated with lower odds of improvement in kinesiophobia (OR 0.43).

Patients with greater levels of problem-focused coping had significantly greater improvement in International Knee Documentation Committee (IKDC) scores, as did patients who were older and more active.

“Specific coping strategies appear to moderate the effect of pain perceptions on postoperative outcomes, with some coping strategies being protective and others being harmful,” the researchers said.

The findings were limited by several factors including the use of multiple comparisons, the inability to assess the impact of pain perception after knee rehabilitation independent of surgery, and the small number of some uncommon procedures, the researchers noted.

However, the results suggest that “recognition of pain perception and coping styles early on in treatment may help sports medicine providers identify patients at risk for an unsatisfactory subjective outcome,” they concluded.

The researchers had no financial conflicts to disclose.

SOURCE: Everhart JS et al. J Sci Med Sport. 2019. doi: 10.1016/j.jsams.2019.09.011.

Adult athletes who underwent knee surgery and had higher levels of preoperative pain catastrophizing were significantly less likely to return to preinjury activity, based on data from 101 individuals.

decade3d/Thinkstock

Pain is highly subjective, and pain perception can play a role in postsurgical outcomes, but the relationships among preoperative pain perception and short-term outcomes including returning to sports have not been well-studied, wrote Joshua S. Everhart, MD, of The Ohio State University Wexner Medical Center, Columbus, and colleagues.

In a study published in the Journal of Science and Medicine in Sport, the researchers assessed 101 adult athletes who underwent knee surgery at a single center. The average age of the patients was 33 years, and 49 were women.

Pain perception and coping were assessed via the McGill Pain questionnaire (SF-MPQ), Pain Catastrophizing Scale (PCS), Pain Coping Measure (PCM), and the brief COPE subscales of acceptance, denial, positive reframing, and use of instrumental support.

Patients who were severe pain catastrophizers (defined as scores greater than 36 on the Pain Catastrophizing Scale) had increased odds of not returning to a similar level of sport (OR 11.3).

Higher scores on the brief COPE subscale of “use of instrumental support” (instruments designed to help patients cope with pain) had a protective effect on returning to preinjury activity (OR 0.72 per point increase). However, higher COPE-denial scores were significantly associated with lower odds of improvement in kinesiophobia (OR 0.43).

Patients with greater levels of problem-focused coping had significantly greater improvement in International Knee Documentation Committee (IKDC) scores, as did patients who were older and more active.

“Specific coping strategies appear to moderate the effect of pain perceptions on postoperative outcomes, with some coping strategies being protective and others being harmful,” the researchers said.

The findings were limited by several factors including the use of multiple comparisons, the inability to assess the impact of pain perception after knee rehabilitation independent of surgery, and the small number of some uncommon procedures, the researchers noted.

However, the results suggest that “recognition of pain perception and coping styles early on in treatment may help sports medicine providers identify patients at risk for an unsatisfactory subjective outcome,” they concluded.

The researchers had no financial conflicts to disclose.

SOURCE: Everhart JS et al. J Sci Med Sport. 2019. doi: 10.1016/j.jsams.2019.09.011.

Adult athletes who underwent knee surgery and had higher levels of preoperative pain catastrophizing were significantly less likely to return to preinjury activity, based on data from 101 individuals.

decade3d/Thinkstock

Pain is highly subjective, and pain perception can play a role in postsurgical outcomes, but the relationships among preoperative pain perception and short-term outcomes including returning to sports have not been well-studied, wrote Joshua S. Everhart, MD, of The Ohio State University Wexner Medical Center, Columbus, and colleagues.

In a study published in the Journal of Science and Medicine in Sport, the researchers assessed 101 adult athletes who underwent knee surgery at a single center. The average age of the patients was 33 years, and 49 were women.

Pain perception and coping were assessed via the McGill Pain questionnaire (SF-MPQ), Pain Catastrophizing Scale (PCS), Pain Coping Measure (PCM), and the brief COPE subscales of acceptance, denial, positive reframing, and use of instrumental support.

Patients who were severe pain catastrophizers (defined as scores greater than 36 on the Pain Catastrophizing Scale) had increased odds of not returning to a similar level of sport (OR 11.3).

Higher scores on the brief COPE subscale of “use of instrumental support” (instruments designed to help patients cope with pain) had a protective effect on returning to preinjury activity (OR 0.72 per point increase). However, higher COPE-denial scores were significantly associated with lower odds of improvement in kinesiophobia (OR 0.43).

Patients with greater levels of problem-focused coping had significantly greater improvement in International Knee Documentation Committee (IKDC) scores, as did patients who were older and more active.

“Specific coping strategies appear to moderate the effect of pain perceptions on postoperative outcomes, with some coping strategies being protective and others being harmful,” the researchers said.

The findings were limited by several factors including the use of multiple comparisons, the inability to assess the impact of pain perception after knee rehabilitation independent of surgery, and the small number of some uncommon procedures, the researchers noted.

However, the results suggest that “recognition of pain perception and coping styles early on in treatment may help sports medicine providers identify patients at risk for an unsatisfactory subjective outcome,” they concluded.

The researchers had no financial conflicts to disclose.

SOURCE: Everhart JS et al. J Sci Med Sport. 2019. doi: 10.1016/j.jsams.2019.09.011.

MADRID –  In the context of three previous trials, a new phase 3 trial demonstrates that the efficacy of as-needed inhaled corticosteroids (ICS) plus a long-acting beta agonist (LABA) is at least comparable to  maintenance ICS for preventing severe exacerbations in the routine care of patients with mild to moderate asthma, according to a presentation at the 2019 ERS International Congress.

Ted Bosworth/MDedge News

This “real-world” study, called PRACTICAL, produced results similar to those of the previous three studies. It showed similar or modestly improved efficacy for the as-needed approach in patients enrolled with mild to moderate asthma, according to Joanna Hardy, MD, a research fellow at the Medical Research Institute of New Zealand, Wellington.

Currently, the Global Initiative for Asthma (GINA) guidelines identify either of the two strategies tested in this trial as acceptable for patients eligible for step 2 asthma control. This study, as in the three trials published previously, provided reassurance that an as-needed approach is adequate for patients insufficiently adherent to daily maintenance therapy.

In PRACTICAL, the results of which were published just prior to the 2019 ERS Congress (Lancet 2019;394:919-28), 890 patients were randomized to use of a single inhaler containing 200 mcg budesonide plus 6 mcg formoterol as needed for symptoms or to a maintenance regimen with the same dose of budesonide taken twice daily. The protocol allowed 250 mcg terbutaline as needed for symptom control in the maintenance arm. The patients were followed for 52 weeks.

For the primary endpoint of the per-patient number of severe exacerbations, defined as need for 3 consecutive days of oral corticosteroids or an emergency department visit to receive oral corticosteroids, the as-needed approach reduced the relative risk by 31% (hazard ratio, 0.69; P = .049). The per-patient rates of exacerbations for the as-needed and maintenance arms were 0.0119 and 0.172, respectively.

The time to first exacerbation, a secondary endpoint, approached significance in favor of as-needed treatment (HR 0.6; P = .05). There was no difference in asthma control as measured with the Asthma Control Questionnaire or in lung function as measured with forced expiratory volume in 1 second (FEV₁₎ at any visit or at the end of the study.

Two SYGMA trials (SYGMA 1 and SYGMA 2), both published in the New England Journal of Medicine in 2018, addressed the same question. Most like PRACTICAL, SYGMA 2 randomized 4,215 patients with mild asthma and found as-needed budesonide/formoterol noninferior to budesonide maintenance for preventing severe exacerbations.

In SYGMA 1, which included an as-needed terbutaline arm, 3,849 patients were randomized. Although as-needed budesonide-formoterol was inferior to budesonide maintenance in that study (but superior to as-needed to terbutaline), the adherence to budesonide maintenance was 78.9%, which Dr. Hardy said does not reflect real-world patient behavior.

“The problem is that we have a lot of data to show that adherence to maintenance asthma therapy in mild asthma is poor,” Dr. Hardy said. In PRACTICAL, all patients were provided with an asthma action plan but no strategies were offered to improve compliance over those employed in usual practice.

In the open-label Novel START trial, published in 2019 in the New England Journal of Medicine, the question posed was different. In that study, which randomized 675 patients, as-needed budesonide/formoterol was superior to as-needed albuterol for prevention of asthma exacerbations at 52 weeks, the time point employed in all four studies. The results, while confirming the importance of the ICS component, have been generally interpreted as supporting the as-needed therapy in mild asthma.

At the ERS 2019 Congress, one of the moderators of the session in which Dr. Hardy spoke, Guy Brusselle, MD, Ghent (Belgium) University, agreed that the available evidence supports as-needed therapy as a viable strategy in mild asthma, but expressed concern about applying this conclusion to patients who have asthma requiring therapy beyond GINA step 2.

“These data might put patients who need GINA step 3 or 4 therapy at risk of not receiving the maintenance therapy they need for disease control,” Dr. Brusselle said.

In light of the challenge of separating those with moderate from mild asthma, Dr. Brusselle suggested another arm to add to real-world clinical trials attempting to identify the most effective approach.

“The optimal arm might be maintenance budesonide with as-needed ICS/LABA,” Dr. Brusselle said. He explained that even if compliance is low, at least some patients will be receiving a maintenance therapy, and this approach might ultimately offer more benefit than one in which patients do not even consider maintenance.

Dr. Hardy reports no potential conflicts of interest.

MADRID –  In the context of three previous trials, a new phase 3 trial demonstrates that the efficacy of as-needed inhaled corticosteroids (ICS) plus a long-acting beta agonist (LABA) is at least comparable to  maintenance ICS for preventing severe exacerbations in the routine care of patients with mild to moderate asthma, according to a presentation at the 2019 ERS International Congress.

Ted Bosworth/MDedge News

This “real-world” study, called PRACTICAL, produced results similar to those of the previous three studies. It showed similar or modestly improved efficacy for the as-needed approach in patients enrolled with mild to moderate asthma, according to Joanna Hardy, MD, a research fellow at the Medical Research Institute of New Zealand, Wellington.

Currently, the Global Initiative for Asthma (GINA) guidelines identify either of the two strategies tested in this trial as acceptable for patients eligible for step 2 asthma control. This study, as in the three trials published previously, provided reassurance that an as-needed approach is adequate for patients insufficiently adherent to daily maintenance therapy.

In PRACTICAL, the results of which were published just prior to the 2019 ERS Congress (Lancet 2019;394:919-28), 890 patients were randomized to use of a single inhaler containing 200 mcg budesonide plus 6 mcg formoterol as needed for symptoms or to a maintenance regimen with the same dose of budesonide taken twice daily. The protocol allowed 250 mcg terbutaline as needed for symptom control in the maintenance arm. The patients were followed for 52 weeks.

For the primary endpoint of the per-patient number of severe exacerbations, defined as need for 3 consecutive days of oral corticosteroids or an emergency department visit to receive oral corticosteroids, the as-needed approach reduced the relative risk by 31% (hazard ratio, 0.69; P = .049). The per-patient rates of exacerbations for the as-needed and maintenance arms were 0.0119 and 0.172, respectively.

The time to first exacerbation, a secondary endpoint, approached significance in favor of as-needed treatment (HR 0.6; P = .05). There was no difference in asthma control as measured with the Asthma Control Questionnaire or in lung function as measured with forced expiratory volume in 1 second (FEV₁₎ at any visit or at the end of the study.

Two SYGMA trials (SYGMA 1 and SYGMA 2), both published in the New England Journal of Medicine in 2018, addressed the same question. Most like PRACTICAL, SYGMA 2 randomized 4,215 patients with mild asthma and found as-needed budesonide/formoterol noninferior to budesonide maintenance for preventing severe exacerbations.

In SYGMA 1, which included an as-needed terbutaline arm, 3,849 patients were randomized. Although as-needed budesonide-formoterol was inferior to budesonide maintenance in that study (but superior to as-needed to terbutaline), the adherence to budesonide maintenance was 78.9%, which Dr. Hardy said does not reflect real-world patient behavior.

“The problem is that we have a lot of data to show that adherence to maintenance asthma therapy in mild asthma is poor,” Dr. Hardy said. In PRACTICAL, all patients were provided with an asthma action plan but no strategies were offered to improve compliance over those employed in usual practice.

In the open-label Novel START trial, published in 2019 in the New England Journal of Medicine, the question posed was different. In that study, which randomized 675 patients, as-needed budesonide/formoterol was superior to as-needed albuterol for prevention of asthma exacerbations at 52 weeks, the time point employed in all four studies. The results, while confirming the importance of the ICS component, have been generally interpreted as supporting the as-needed therapy in mild asthma.

At the ERS 2019 Congress, one of the moderators of the session in which Dr. Hardy spoke, Guy Brusselle, MD, Ghent (Belgium) University, agreed that the available evidence supports as-needed therapy as a viable strategy in mild asthma, but expressed concern about applying this conclusion to patients who have asthma requiring therapy beyond GINA step 2.

“These data might put patients who need GINA step 3 or 4 therapy at risk of not receiving the maintenance therapy they need for disease control,” Dr. Brusselle said.

In light of the challenge of separating those with moderate from mild asthma, Dr. Brusselle suggested another arm to add to real-world clinical trials attempting to identify the most effective approach.

“The optimal arm might be maintenance budesonide with as-needed ICS/LABA,” Dr. Brusselle said. He explained that even if compliance is low, at least some patients will be receiving a maintenance therapy, and this approach might ultimately offer more benefit than one in which patients do not even consider maintenance.

Dr. Hardy reports no potential conflicts of interest.

MADRID –  In the context of three previous trials, a new phase 3 trial demonstrates that the efficacy of as-needed inhaled corticosteroids (ICS) plus a long-acting beta agonist (LABA) is at least comparable to  maintenance ICS for preventing severe exacerbations in the routine care of patients with mild to moderate asthma, according to a presentation at the 2019 ERS International Congress.

Ted Bosworth/MDedge News

This “real-world” study, called PRACTICAL, produced results similar to those of the previous three studies. It showed similar or modestly improved efficacy for the as-needed approach in patients enrolled with mild to moderate asthma, according to Joanna Hardy, MD, a research fellow at the Medical Research Institute of New Zealand, Wellington.

Currently, the Global Initiative for Asthma (GINA) guidelines identify either of the two strategies tested in this trial as acceptable for patients eligible for step 2 asthma control. This study, as in the three trials published previously, provided reassurance that an as-needed approach is adequate for patients insufficiently adherent to daily maintenance therapy.

In PRACTICAL, the results of which were published just prior to the 2019 ERS Congress (Lancet 2019;394:919-28), 890 patients were randomized to use of a single inhaler containing 200 mcg budesonide plus 6 mcg formoterol as needed for symptoms or to a maintenance regimen with the same dose of budesonide taken twice daily. The protocol allowed 250 mcg terbutaline as needed for symptom control in the maintenance arm. The patients were followed for 52 weeks.

For the primary endpoint of the per-patient number of severe exacerbations, defined as need for 3 consecutive days of oral corticosteroids or an emergency department visit to receive oral corticosteroids, the as-needed approach reduced the relative risk by 31% (hazard ratio, 0.69; P = .049). The per-patient rates of exacerbations for the as-needed and maintenance arms were 0.0119 and 0.172, respectively.

The time to first exacerbation, a secondary endpoint, approached significance in favor of as-needed treatment (HR 0.6; P = .05). There was no difference in asthma control as measured with the Asthma Control Questionnaire or in lung function as measured with forced expiratory volume in 1 second (FEV₁₎ at any visit or at the end of the study.

Two SYGMA trials (SYGMA 1 and SYGMA 2), both published in the New England Journal of Medicine in 2018, addressed the same question. Most like PRACTICAL, SYGMA 2 randomized 4,215 patients with mild asthma and found as-needed budesonide/formoterol noninferior to budesonide maintenance for preventing severe exacerbations.

In SYGMA 1, which included an as-needed terbutaline arm, 3,849 patients were randomized. Although as-needed budesonide-formoterol was inferior to budesonide maintenance in that study (but superior to as-needed to terbutaline), the adherence to budesonide maintenance was 78.9%, which Dr. Hardy said does not reflect real-world patient behavior.

“The problem is that we have a lot of data to show that adherence to maintenance asthma therapy in mild asthma is poor,” Dr. Hardy said. In PRACTICAL, all patients were provided with an asthma action plan but no strategies were offered to improve compliance over those employed in usual practice.

In the open-label Novel START trial, published in 2019 in the New England Journal of Medicine, the question posed was different. In that study, which randomized 675 patients, as-needed budesonide/formoterol was superior to as-needed albuterol for prevention of asthma exacerbations at 52 weeks, the time point employed in all four studies. The results, while confirming the importance of the ICS component, have been generally interpreted as supporting the as-needed therapy in mild asthma.

At the ERS 2019 Congress, one of the moderators of the session in which Dr. Hardy spoke, Guy Brusselle, MD, Ghent (Belgium) University, agreed that the available evidence supports as-needed therapy as a viable strategy in mild asthma, but expressed concern about applying this conclusion to patients who have asthma requiring therapy beyond GINA step 2.

“These data might put patients who need GINA step 3 or 4 therapy at risk of not receiving the maintenance therapy they need for disease control,” Dr. Brusselle said.

In light of the challenge of separating those with moderate from mild asthma, Dr. Brusselle suggested another arm to add to real-world clinical trials attempting to identify the most effective approach.

“The optimal arm might be maintenance budesonide with as-needed ICS/LABA,” Dr. Brusselle said. He explained that even if compliance is low, at least some patients will be receiving a maintenance therapy, and this approach might ultimately offer more benefit than one in which patients do not even consider maintenance.

Dr. Hardy reports no potential conflicts of interest.

Despite theoretical preferences for either the anterior or the posterior approach to peroral endoscopic myotomy (POEM) in patients with achalasia, a new study has found no significant difference between the two in regard to clinical success or safety.

“Both approaches are equivalently safe when performed by experienced operators,” wrote Mouen A. Khashab, MD, of Johns Hopkins Medicine in Baltimore and coauthors, adding that the most notable difference was “closure was rated as easier during the posterior approach,” and fewer clips were needed. The study was published in Gastrointestinal Endoscopy.

To analyze and compare the efficacy of the two POEM approaches, the researchers conducted a multicenter controlled clinical trial of 150 patients with achalasia. They were randomized into two groups: those receiving POEM with the anterior approach (n = 73) or the posterior approach (n = 77). Of those patients, 148 received POEM and 138 completed 1-year follow-up. At 3, 6, and 12 months’ follow-up by phone call, patients were evaluated via outcomes that included Eckardt and dysphagia scores, quality of life scales, and gastroesophageal reflux disease questionnaire score.

Technical success was achieved in all 77 patients in the posterior group compared with 71 patients (97.3%) in the anterior group (P = .23). Both groups had a median length of hospital stay post procedure of 2 days. Adverse events occurred in seven patients (9%) in the posterior group and in eight patients (11%) in the anterior group (P = .703).

Though no significant differences were found between the two groups in time to perform mucosal incision, submucosal tunneling, myotomy, or closure, the median difficulty of closure in the posterior group was lower than in the anterior group (P = .002). In addition, fewer clips were needed during closure in the posterior approach.

After per-protocol analysis, clinical success at 1 year was achieved in 89% of patients in the posterior group (95% confidence interval, 81%-96%) and 90% of patients in the anterior group (95% CI, 82%-97%). At 1-year follow-up, both groups had an Eckardt score of 0 (P = .994) and their median gastroesophageal reflux disease score was 6 (P = .73). All patients who completed quality of life questionnaires reported improvements, with a median change in pain of 23 in the anterior group and 34 in the posterior group (P = .49). The posterior group also reported a greater median change in social functioning (50 vs. 38; P = .02).

The authors noted their study’s potential limitations, including relying on the Eckardt scoring system – one that was recently questioned in terms of validity – to determine clinical success. However, they also offered an argument in favor of clinical scoring because of “the importance of symptom improvement from the patient perspective.” Also, because of the lack of prestudy data comparing the anterior and posterior approaches, they chose 15% as the noninferiority margin for clinical efficacy, which could be regarded as a limitation as well.

Four of the authors reported potential conflicts of interest, including serving as consultants for various medical companies, serving on medical advisory boards, and receiving research support and personal fees. The other authors reported no conflicts of interest.

SOURCE: Khashab MA et al. Gastrointest Endosc. 2019 Aug 10. doi: 10.1016/j.gie.2019.07.034.

Despite theoretical preferences for either the anterior or the posterior approach to peroral endoscopic myotomy (POEM) in patients with achalasia, a new study has found no significant difference between the two in regard to clinical success or safety.

“Both approaches are equivalently safe when performed by experienced operators,” wrote Mouen A. Khashab, MD, of Johns Hopkins Medicine in Baltimore and coauthors, adding that the most notable difference was “closure was rated as easier during the posterior approach,” and fewer clips were needed. The study was published in Gastrointestinal Endoscopy.

To analyze and compare the efficacy of the two POEM approaches, the researchers conducted a multicenter controlled clinical trial of 150 patients with achalasia. They were randomized into two groups: those receiving POEM with the anterior approach (n = 73) or the posterior approach (n = 77). Of those patients, 148 received POEM and 138 completed 1-year follow-up. At 3, 6, and 12 months’ follow-up by phone call, patients were evaluated via outcomes that included Eckardt and dysphagia scores, quality of life scales, and gastroesophageal reflux disease questionnaire score.

Technical success was achieved in all 77 patients in the posterior group compared with 71 patients (97.3%) in the anterior group (P = .23). Both groups had a median length of hospital stay post procedure of 2 days. Adverse events occurred in seven patients (9%) in the posterior group and in eight patients (11%) in the anterior group (P = .703).

Though no significant differences were found between the two groups in time to perform mucosal incision, submucosal tunneling, myotomy, or closure, the median difficulty of closure in the posterior group was lower than in the anterior group (P = .002). In addition, fewer clips were needed during closure in the posterior approach.

After per-protocol analysis, clinical success at 1 year was achieved in 89% of patients in the posterior group (95% confidence interval, 81%-96%) and 90% of patients in the anterior group (95% CI, 82%-97%). At 1-year follow-up, both groups had an Eckardt score of 0 (P = .994) and their median gastroesophageal reflux disease score was 6 (P = .73). All patients who completed quality of life questionnaires reported improvements, with a median change in pain of 23 in the anterior group and 34 in the posterior group (P = .49). The posterior group also reported a greater median change in social functioning (50 vs. 38; P = .02).

The authors noted their study’s potential limitations, including relying on the Eckardt scoring system – one that was recently questioned in terms of validity – to determine clinical success. However, they also offered an argument in favor of clinical scoring because of “the importance of symptom improvement from the patient perspective.” Also, because of the lack of prestudy data comparing the anterior and posterior approaches, they chose 15% as the noninferiority margin for clinical efficacy, which could be regarded as a limitation as well.

Four of the authors reported potential conflicts of interest, including serving as consultants for various medical companies, serving on medical advisory boards, and receiving research support and personal fees. The other authors reported no conflicts of interest.

SOURCE: Khashab MA et al. Gastrointest Endosc. 2019 Aug 10. doi: 10.1016/j.gie.2019.07.034.

Despite theoretical preferences for either the anterior or the posterior approach to peroral endoscopic myotomy (POEM) in patients with achalasia, a new study has found no significant difference between the two in regard to clinical success or safety.

“Both approaches are equivalently safe when performed by experienced operators,” wrote Mouen A. Khashab, MD, of Johns Hopkins Medicine in Baltimore and coauthors, adding that the most notable difference was “closure was rated as easier during the posterior approach,” and fewer clips were needed. The study was published in Gastrointestinal Endoscopy.

To analyze and compare the efficacy of the two POEM approaches, the researchers conducted a multicenter controlled clinical trial of 150 patients with achalasia. They were randomized into two groups: those receiving POEM with the anterior approach (n = 73) or the posterior approach (n = 77). Of those patients, 148 received POEM and 138 completed 1-year follow-up. At 3, 6, and 12 months’ follow-up by phone call, patients were evaluated via outcomes that included Eckardt and dysphagia scores, quality of life scales, and gastroesophageal reflux disease questionnaire score.

Technical success was achieved in all 77 patients in the posterior group compared with 71 patients (97.3%) in the anterior group (P = .23). Both groups had a median length of hospital stay post procedure of 2 days. Adverse events occurred in seven patients (9%) in the posterior group and in eight patients (11%) in the anterior group (P = .703).

Though no significant differences were found between the two groups in time to perform mucosal incision, submucosal tunneling, myotomy, or closure, the median difficulty of closure in the posterior group was lower than in the anterior group (P = .002). In addition, fewer clips were needed during closure in the posterior approach.

After per-protocol analysis, clinical success at 1 year was achieved in 89% of patients in the posterior group (95% confidence interval, 81%-96%) and 90% of patients in the anterior group (95% CI, 82%-97%). At 1-year follow-up, both groups had an Eckardt score of 0 (P = .994) and their median gastroesophageal reflux disease score was 6 (P = .73). All patients who completed quality of life questionnaires reported improvements, with a median change in pain of 23 in the anterior group and 34 in the posterior group (P = .49). The posterior group also reported a greater median change in social functioning (50 vs. 38; P = .02).

The authors noted their study’s potential limitations, including relying on the Eckardt scoring system – one that was recently questioned in terms of validity – to determine clinical success. However, they also offered an argument in favor of clinical scoring because of “the importance of symptom improvement from the patient perspective.” Also, because of the lack of prestudy data comparing the anterior and posterior approaches, they chose 15% as the noninferiority margin for clinical efficacy, which could be regarded as a limitation as well.

Four of the authors reported potential conflicts of interest, including serving as consultants for various medical companies, serving on medical advisory boards, and receiving research support and personal fees. The other authors reported no conflicts of interest.

SOURCE: Khashab MA et al. Gastrointest Endosc. 2019 Aug 10. doi: 10.1016/j.gie.2019.07.034.

Despite theoretical preferences for either the anterior or the posterior approach to peroral endoscopic myotomy (POEM) in patients with achalasia, a new study has found no significant difference between the two in regard to clinical success or safety.

“Both approaches are equivalently safe when performed by experienced operators,” wrote Mouen A. Khashab, MD, of Johns Hopkins Medicine in Baltimore and coauthors, adding that the most notable difference was “closure was rated as easier during the posterior approach,” and fewer clips were needed. The study was published in Gastrointestinal Endoscopy.

To analyze and compare the efficacy of the two POEM approaches, the researchers conducted a multicenter controlled clinical trial of 150 patients with achalasia. They were randomized into two groups: those receiving POEM with the anterior approach (n = 73) or the posterior approach (n = 77). Of those patients, 148 received POEM and 138 completed 1-year follow-up. At 3, 6, and 12 months’ follow-up by phone call, patients were evaluated via outcomes that included Eckardt and dysphagia scores, quality of life scales, and gastroesophageal reflux disease questionnaire score.

Technical success was achieved in all 77 patients in the posterior group compared with 71 patients (97.3%) in the anterior group (P = .23). Both groups had a median length of hospital stay post procedure of 2 days. Adverse events occurred in seven patients (9%) in the posterior group and in eight patients (11%) in the anterior group (P = .703).

Though no significant differences were found between the two groups in time to perform mucosal incision, submucosal tunneling, myotomy, or closure, the median difficulty of closure in the posterior group was lower than in the anterior group (P = .002). In addition, fewer clips were needed during closure in the posterior approach.

After per-protocol analysis, clinical success at 1 year was achieved in 89% of patients in the posterior group (95% confidence interval, 81%-96%) and 90% of patients in the anterior group (95% CI, 82%-97%). At 1-year follow-up, both groups had an Eckardt score of 0 (P = .994) and their median gastroesophageal reflux disease score was 6 (P = .73). All patients who completed quality of life questionnaires reported improvements, with a median change in pain of 23 in the anterior group and 34 in the posterior group (P = .49). The posterior group also reported a greater median change in social functioning (50 vs. 38; P = .02).

The authors noted their study’s potential limitations, including relying on the Eckardt scoring system – one that was recently questioned in terms of validity – to determine clinical success. However, they also offered an argument in favor of clinical scoring because of “the importance of symptom improvement from the patient perspective.” Also, because of the lack of prestudy data comparing the anterior and posterior approaches, they chose 15% as the noninferiority margin for clinical efficacy, which could be regarded as a limitation as well.

Four of the authors reported potential conflicts of interest, including serving as consultants for various medical companies, serving on medical advisory boards, and receiving research support and personal fees. The other authors reported no conflicts of interest.

SOURCE: Khashab MA et al. Gastrointest Endosc. 2019 Aug 10. doi: 10.1016/j.gie.2019.07.034.

The AGA Center for GI Innovation and Technology supports innovation and the development of new technology in gastroenterology, hepatology, nutrition and obesity by guiding medical device and therapeutics innovators through the technology development and adoption process. To learn more visit www.gastro.org/CIGT. 

Despite theoretical preferences for either the anterior or the posterior approach to peroral endoscopic myotomy (POEM) in patients with achalasia, a new study has found no significant difference between the two in regard to clinical success or safety.

“Both approaches are equivalently safe when performed by experienced operators,” wrote Mouen A. Khashab, MD, of Johns Hopkins Medicine in Baltimore and coauthors, adding that the most notable difference was “closure was rated as easier during the posterior approach,” and fewer clips were needed. The study was published in Gastrointestinal Endoscopy.

To analyze and compare the efficacy of the two POEM approaches, the researchers conducted a multicenter controlled clinical trial of 150 patients with achalasia. They were randomized into two groups: those receiving POEM with the anterior approach (n = 73) or the posterior approach (n = 77). Of those patients, 148 received POEM and 138 completed 1-year follow-up. At 3, 6, and 12 months’ follow-up by phone call, patients were evaluated via outcomes that included Eckardt and dysphagia scores, quality of life scales, and gastroesophageal reflux disease questionnaire score.

Technical success was achieved in all 77 patients in the posterior group compared with 71 patients (97.3%) in the anterior group (P = .23). Both groups had a median length of hospital stay post procedure of 2 days. Adverse events occurred in seven patients (9%) in the posterior group and in eight patients (11%) in the anterior group (P = .703).

Though no significant differences were found between the two groups in time to perform mucosal incision, submucosal tunneling, myotomy, or closure, the median difficulty of closure in the posterior group was lower than in the anterior group (P = .002). In addition, fewer clips were needed during closure in the posterior approach.

After per-protocol analysis, clinical success at 1 year was achieved in 89% of patients in the posterior group (95% confidence interval, 81%-96%) and 90% of patients in the anterior group (95% CI, 82%-97%). At 1-year follow-up, both groups had an Eckardt score of 0 (P = .994) and their median gastroesophageal reflux disease score was 6 (P = .73). All patients who completed quality of life questionnaires reported improvements, with a median change in pain of 23 in the anterior group and 34 in the posterior group (P = .49). The posterior group also reported a greater median change in social functioning (50 vs. 38; P = .02).

The authors noted their study’s potential limitations, including relying on the Eckardt scoring system – one that was recently questioned in terms of validity – to determine clinical success. However, they also offered an argument in favor of clinical scoring because of “the importance of symptom improvement from the patient perspective.” Also, because of the lack of prestudy data comparing the anterior and posterior approaches, they chose 15% as the noninferiority margin for clinical efficacy, which could be regarded as a limitation as well.

Four of the authors reported potential conflicts of interest, including serving as consultants for various medical companies, serving on medical advisory boards, and receiving research support and personal fees. The other authors reported no conflicts of interest.

SOURCE: Khashab MA et al. Gastrointest Endosc. 2019 Aug 10. doi: 10.1016/j.gie.2019.07.034.

The AGA Center for GI Innovation and Technology supports innovation and the development of new technology in gastroenterology, hepatology, nutrition and obesity by guiding medical device and therapeutics innovators through the technology development and adoption process. To learn more visit www.gastro.org/CIGT. 

Despite theoretical preferences for either the anterior or the posterior approach to peroral endoscopic myotomy (POEM) in patients with achalasia, a new study has found no significant difference between the two in regard to clinical success or safety.

“Both approaches are equivalently safe when performed by experienced operators,” wrote Mouen A. Khashab, MD, of Johns Hopkins Medicine in Baltimore and coauthors, adding that the most notable difference was “closure was rated as easier during the posterior approach,” and fewer clips were needed. The study was published in Gastrointestinal Endoscopy.

To analyze and compare the efficacy of the two POEM approaches, the researchers conducted a multicenter controlled clinical trial of 150 patients with achalasia. They were randomized into two groups: those receiving POEM with the anterior approach (n = 73) or the posterior approach (n = 77). Of those patients, 148 received POEM and 138 completed 1-year follow-up. At 3, 6, and 12 months’ follow-up by phone call, patients were evaluated via outcomes that included Eckardt and dysphagia scores, quality of life scales, and gastroesophageal reflux disease questionnaire score.

Technical success was achieved in all 77 patients in the posterior group compared with 71 patients (97.3%) in the anterior group (P = .23). Both groups had a median length of hospital stay post procedure of 2 days. Adverse events occurred in seven patients (9%) in the posterior group and in eight patients (11%) in the anterior group (P = .703).

Though no significant differences were found between the two groups in time to perform mucosal incision, submucosal tunneling, myotomy, or closure, the median difficulty of closure in the posterior group was lower than in the anterior group (P = .002). In addition, fewer clips were needed during closure in the posterior approach.

After per-protocol analysis, clinical success at 1 year was achieved in 89% of patients in the posterior group (95% confidence interval, 81%-96%) and 90% of patients in the anterior group (95% CI, 82%-97%). At 1-year follow-up, both groups had an Eckardt score of 0 (P = .994) and their median gastroesophageal reflux disease score was 6 (P = .73). All patients who completed quality of life questionnaires reported improvements, with a median change in pain of 23 in the anterior group and 34 in the posterior group (P = .49). The posterior group also reported a greater median change in social functioning (50 vs. 38; P = .02).

The authors noted their study’s potential limitations, including relying on the Eckardt scoring system – one that was recently questioned in terms of validity – to determine clinical success. However, they also offered an argument in favor of clinical scoring because of “the importance of symptom improvement from the patient perspective.” Also, because of the lack of prestudy data comparing the anterior and posterior approaches, they chose 15% as the noninferiority margin for clinical efficacy, which could be regarded as a limitation as well.

Four of the authors reported potential conflicts of interest, including serving as consultants for various medical companies, serving on medical advisory boards, and receiving research support and personal fees. The other authors reported no conflicts of interest.

SOURCE: Khashab MA et al. Gastrointest Endosc. 2019 Aug 10. doi: 10.1016/j.gie.2019.07.034.

The AGA Center for GI Innovation and Technology supports innovation and the development of new technology in gastroenterology, hepatology, nutrition and obesity by guiding medical device and therapeutics innovators through the technology development and adoption process. To learn more visit www.gastro.org/CIGT. 

One of the nation’s most preventable diseases is killing newborns in ever-increasing numbers.

Christoph Burgstedt/iStock/Getty Images Plus

Nationwide, 1,306 infants acquired syphilis from their mother in 2018, a 40% rise over 2017, according to federal data released Oct. 8. Seventy-eight of those babies were stillborn, and 16 died after birth.

In California, cases of congenital syphilis – the term used when a mother passes the infection to her baby during pregnancy – continued a stark 7-year climb, to 332 cases, an 18.1% increase from 2017, according to the federal data. Only Texas, Nevada, Louisiana, and Arizona had congenital syphilis rates higher than California’s. Those five states combined made up nearly two-thirds of total cases, although all but 17 states saw increases in their congenital syphilis rates.

The state-by-state numbers were released as part of a broader report from the Centers for Disease Control and Prevention tracking trends in sexually transmitted diseases. Cases of syphilis, gonorrhea, and chlamydia combined reached an all-time high in 2018. Cases of the most infectious stage of syphilis rose 14% to more than 35,000 cases; gonorrhea increased 5% to more than 580,000 cases; and chlamydia increased 3% to more than 1.7 million cases.

For veteran public health workers, the upward trend in congenital syphilis numbers is particularly disturbing because the condition is so easy to prevent. Blood tests can identify infection in pregnant women. The treatment is relatively simple and effective. When caught during pregnancy, transmission from mother to baby generally can be stopped.

“When we see a case of congenital syphilis, it is a hallmark of a health system and a health care failure,” said Virginia Bowen, PhD, an epidemiologist with the CDC and an author of the report.

It takes just a few shots of antibiotics to prevent a baby from getting syphilis from its mother. Left untreated, Treponema pallidum, the corkscrew-shaped organism that causes syphilis, can wiggle its way through a mother’s placenta and into a fetus. Once there, it can multiply furiously, invading every part of the body.

The effects on a newborn can be devastating. Philip Cheng, MD, is a neonatologist at St. Joseph’s Medical Center in Stockton, a city in San Joaquin County in California’s Central Valley. Twenty-six babies were infected last year in San Joaquin County, according to state data.

The brain of one of Cheng’s patients didn’t develop properly and the baby died shortly after birth. Other young patients survive but battle blood abnormalities, bone deformities, and organ damage. Congenital syphilis can cause blindness and excruciating pain.

Public health departments across the Central Valley, a largely rural expanse, report similar experiences. Following the release of the CDC report Tuesday, the California Department of Public Health released its county-by-county numbers for 2018. The report showed syphilis, gonorrhea, and chlamydia levels at their highest in 30 years, and attributed 22 stillbirths or neonatal deaths to congenital syphilis.

For the past several years, Fresno County, which had 63 cases of congenital syphilis in 2017, had the highest rate in California. In 2018, Fresno fell to fourth, behind Yuba, Kern, and San Joaquin counties. But the epidemic is far from under control. “I couldn’t even tell you how soon I think we’re going to see a decrease,” said Jena Adams, who oversees HIV and STD programs for Fresno County.

Syphilis was once a prolific and widely feared STD. But by the 1940s, penicillin was found to have a near-perfect cure rate for the disease. By 2000, syphilis rates were so low in the U.S. that the federal government launched a plan to eliminate the disease. Today, that goal is a distant memory.

Health departments once tracked down every person who tested positive for chlamydia, gonorrhea, or syphilis, to make sure they and their partners got treatment. With limited funds and climbing caseloads, many states now devote resources only to tracking syphilis. The caseloads are so high in some California counties that they track only women of childbearing age or just pregnant women.

“A lot of the funding for day-to-day public health work isn’t there,” said Jeffrey Klausner, MD, a professor at the University of California-Los Angeles who ran San Francisco’s STD program for more than a decade.

The bulk of STD prevention funding is appropriated by Congress to the CDC, which passes it on to states. That funding has been largely flat since 2003, according to data from the National Coalition of STD Directors, which represents health departments across the country. Take into account inflation and the growing caseloads, and the money is spread thinner. “It takes money, it takes training, it takes resources,” Dr. Klausner said, “and policymakers have just not prioritized that.”

A report this year by Trust for America’s Health, a public health policy research and advocacy group, estimated that 55,000 jobs were cut from local public health departments from 2008 to 2017. “We have our hands tied as much as [states] do,” said Dr. Bowen of the CDC. “We take what we’re given and try to distribute it as fairly as we can.”

San Joaquin County health officials have reorganized the department and applied for grants to increase the number of investigators available while congenital syphilis has spiked, said Hemal Parikh, county coordinator for STD control. But even with new hires and cutting back to tracking only women of childbearing age with syphilis, an investigator can have anywhere from 20 to 30 open cases at a time. In other counties, the caseload can be double that.

In 2018, Jennifer Wagman, PhD, a UCLA professor who studies infectious diseases and gender inequality, was part of a group that received CDC funding to look into what is causing the spike in congenital syphilis in California’s Central Valley.

Dr. Wagman said that, after years of studying health systems in other countries, she was shocked to see how much basic public health infrastructure has crumbled in California. In many parts of the Central Valley, county walk-in clinics that tested for and treated STDs were shuttered in the wake of the recession. That left few places for drop-in care, and investigators with no place to take someone for immediate treatment. Investigators or their patients must make appointments at one of the few providers who carry the right kind of treatment and hope the patients can keep the appointment when the time comes.

In focus groups, women told Dr. Wagman that working hourly jobs, or dealing with chaotic lives involving homelessness, abusive partners, and drug use, can make it all but impossible to stick to the appointments required at private clinics.

Dr. Wagman found that women in these high-risk groups were seeking care, though sometimes late in their pregnancy. They were just more likely to visit an emergency room, urgent care, or even a methadone clinic – places that take drop-ins but don’t necessarily routinely test for or treat syphilis.

“These people already have a million barriers,” said Jenny Malone, the public health nurse for San Joaquin County. “Now there are more.”

The most challenging cases in California are wrapped up with the state’s growing housing crisis and a methamphetamine epidemic with few treatment options. Women who are homeless often have unreliable contact information and are unlikely to have a primary care doctor. That makes them tough to track down to give a positive diagnosis or to follow up on a treatment plan.

Louisiana had the highest rate of congenital syphilis in the country for several years – until 2018. After a 22% drop in its rate, combined with increases in other states, Louisiana now ranks behind Texas and Nevada. That drop is the direct result of $550 million in temporary supplemental funding that the CDC gave the state to combat the epidemic, said Chaquetta Johnson, DNP, deputy director of operations for the state’s STD/HIV/hepatitis program. The money helped bolster the state’s lagging public health infrastructure. It was used to host two conferences for providers in the hardest-hit areas, hire two case managers and a nurse educator, create a program for in-home treatment, and improve data systems to track cases, among other things.

In California, more than 40% of pregnant women with syphilis passed it on to their baby in 2016, the most recent year for which data is available. Gov. Gavin Newsom (D) made additional funding available this year, but it’s a “drop in the bucket,” said Sergio Morales of Essential Access Health, a nonprofit that focuses on sexual and reproductive health and is working with Kern County on congenital syphilis. “We are seeing the results of years of inaction and a lack of prioritization of STD prevention, and we’re now paying the price.”
 

This KHN story first published on California Healthline, a service of the California Health Care Foundation. Kaiser Health News is a nonprofit national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation that is not affiliated with Kaiser Permanente.

[Update: This story was revised at 6:50 p.m. ET on Oct. 8 to reflect news developments.]
 

One of the nation’s most preventable diseases is killing newborns in ever-increasing numbers.

Christoph Burgstedt/iStock/Getty Images Plus

Nationwide, 1,306 infants acquired syphilis from their mother in 2018, a 40% rise over 2017, according to federal data released Oct. 8. Seventy-eight of those babies were stillborn, and 16 died after birth.

In California, cases of congenital syphilis – the term used when a mother passes the infection to her baby during pregnancy – continued a stark 7-year climb, to 332 cases, an 18.1% increase from 2017, according to the federal data. Only Texas, Nevada, Louisiana, and Arizona had congenital syphilis rates higher than California’s. Those five states combined made up nearly two-thirds of total cases, although all but 17 states saw increases in their congenital syphilis rates.

The state-by-state numbers were released as part of a broader report from the Centers for Disease Control and Prevention tracking trends in sexually transmitted diseases. Cases of syphilis, gonorrhea, and chlamydia combined reached an all-time high in 2018. Cases of the most infectious stage of syphilis rose 14% to more than 35,000 cases; gonorrhea increased 5% to more than 580,000 cases; and chlamydia increased 3% to more than 1.7 million cases.

For veteran public health workers, the upward trend in congenital syphilis numbers is particularly disturbing because the condition is so easy to prevent. Blood tests can identify infection in pregnant women. The treatment is relatively simple and effective. When caught during pregnancy, transmission from mother to baby generally can be stopped.

“When we see a case of congenital syphilis, it is a hallmark of a health system and a health care failure,” said Virginia Bowen, PhD, an epidemiologist with the CDC and an author of the report.

It takes just a few shots of antibiotics to prevent a baby from getting syphilis from its mother. Left untreated, Treponema pallidum, the corkscrew-shaped organism that causes syphilis, can wiggle its way through a mother’s placenta and into a fetus. Once there, it can multiply furiously, invading every part of the body.

The effects on a newborn can be devastating. Philip Cheng, MD, is a neonatologist at St. Joseph’s Medical Center in Stockton, a city in San Joaquin County in California’s Central Valley. Twenty-six babies were infected last year in San Joaquin County, according to state data.

The brain of one of Cheng’s patients didn’t develop properly and the baby died shortly after birth. Other young patients survive but battle blood abnormalities, bone deformities, and organ damage. Congenital syphilis can cause blindness and excruciating pain.

Public health departments across the Central Valley, a largely rural expanse, report similar experiences. Following the release of the CDC report Tuesday, the California Department of Public Health released its county-by-county numbers for 2018. The report showed syphilis, gonorrhea, and chlamydia levels at their highest in 30 years, and attributed 22 stillbirths or neonatal deaths to congenital syphilis.

For the past several years, Fresno County, which had 63 cases of congenital syphilis in 2017, had the highest rate in California. In 2018, Fresno fell to fourth, behind Yuba, Kern, and San Joaquin counties. But the epidemic is far from under control. “I couldn’t even tell you how soon I think we’re going to see a decrease,” said Jena Adams, who oversees HIV and STD programs for Fresno County.

Syphilis was once a prolific and widely feared STD. But by the 1940s, penicillin was found to have a near-perfect cure rate for the disease. By 2000, syphilis rates were so low in the U.S. that the federal government launched a plan to eliminate the disease. Today, that goal is a distant memory.

Health departments once tracked down every person who tested positive for chlamydia, gonorrhea, or syphilis, to make sure they and their partners got treatment. With limited funds and climbing caseloads, many states now devote resources only to tracking syphilis. The caseloads are so high in some California counties that they track only women of childbearing age or just pregnant women.

“A lot of the funding for day-to-day public health work isn’t there,” said Jeffrey Klausner, MD, a professor at the University of California-Los Angeles who ran San Francisco’s STD program for more than a decade.

The bulk of STD prevention funding is appropriated by Congress to the CDC, which passes it on to states. That funding has been largely flat since 2003, according to data from the National Coalition of STD Directors, which represents health departments across the country. Take into account inflation and the growing caseloads, and the money is spread thinner. “It takes money, it takes training, it takes resources,” Dr. Klausner said, “and policymakers have just not prioritized that.”

A report this year by Trust for America’s Health, a public health policy research and advocacy group, estimated that 55,000 jobs were cut from local public health departments from 2008 to 2017. “We have our hands tied as much as [states] do,” said Dr. Bowen of the CDC. “We take what we’re given and try to distribute it as fairly as we can.”

San Joaquin County health officials have reorganized the department and applied for grants to increase the number of investigators available while congenital syphilis has spiked, said Hemal Parikh, county coordinator for STD control. But even with new hires and cutting back to tracking only women of childbearing age with syphilis, an investigator can have anywhere from 20 to 30 open cases at a time. In other counties, the caseload can be double that.

In 2018, Jennifer Wagman, PhD, a UCLA professor who studies infectious diseases and gender inequality, was part of a group that received CDC funding to look into what is causing the spike in congenital syphilis in California’s Central Valley.

Dr. Wagman said that, after years of studying health systems in other countries, she was shocked to see how much basic public health infrastructure has crumbled in California. In many parts of the Central Valley, county walk-in clinics that tested for and treated STDs were shuttered in the wake of the recession. That left few places for drop-in care, and investigators with no place to take someone for immediate treatment. Investigators or their patients must make appointments at one of the few providers who carry the right kind of treatment and hope the patients can keep the appointment when the time comes.

In focus groups, women told Dr. Wagman that working hourly jobs, or dealing with chaotic lives involving homelessness, abusive partners, and drug use, can make it all but impossible to stick to the appointments required at private clinics.

Dr. Wagman found that women in these high-risk groups were seeking care, though sometimes late in their pregnancy. They were just more likely to visit an emergency room, urgent care, or even a methadone clinic – places that take drop-ins but don’t necessarily routinely test for or treat syphilis.

“These people already have a million barriers,” said Jenny Malone, the public health nurse for San Joaquin County. “Now there are more.”

The most challenging cases in California are wrapped up with the state’s growing housing crisis and a methamphetamine epidemic with few treatment options. Women who are homeless often have unreliable contact information and are unlikely to have a primary care doctor. That makes them tough to track down to give a positive diagnosis or to follow up on a treatment plan.

Louisiana had the highest rate of congenital syphilis in the country for several years – until 2018. After a 22% drop in its rate, combined with increases in other states, Louisiana now ranks behind Texas and Nevada. That drop is the direct result of $550 million in temporary supplemental funding that the CDC gave the state to combat the epidemic, said Chaquetta Johnson, DNP, deputy director of operations for the state’s STD/HIV/hepatitis program. The money helped bolster the state’s lagging public health infrastructure. It was used to host two conferences for providers in the hardest-hit areas, hire two case managers and a nurse educator, create a program for in-home treatment, and improve data systems to track cases, among other things.

In California, more than 40% of pregnant women with syphilis passed it on to their baby in 2016, the most recent year for which data is available. Gov. Gavin Newsom (D) made additional funding available this year, but it’s a “drop in the bucket,” said Sergio Morales of Essential Access Health, a nonprofit that focuses on sexual and reproductive health and is working with Kern County on congenital syphilis. “We are seeing the results of years of inaction and a lack of prioritization of STD prevention, and we’re now paying the price.”
 

This KHN story first published on California Healthline, a service of the California Health Care Foundation. Kaiser Health News is a nonprofit national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation that is not affiliated with Kaiser Permanente.

[Update: This story was revised at 6:50 p.m. ET on Oct. 8 to reflect news developments.]
 

One of the nation’s most preventable diseases is killing newborns in ever-increasing numbers.

Christoph Burgstedt/iStock/Getty Images Plus

Nationwide, 1,306 infants acquired syphilis from their mother in 2018, a 40% rise over 2017, according to federal data released Oct. 8. Seventy-eight of those babies were stillborn, and 16 died after birth.

In California, cases of congenital syphilis – the term used when a mother passes the infection to her baby during pregnancy – continued a stark 7-year climb, to 332 cases, an 18.1% increase from 2017, according to the federal data. Only Texas, Nevada, Louisiana, and Arizona had congenital syphilis rates higher than California’s. Those five states combined made up nearly two-thirds of total cases, although all but 17 states saw increases in their congenital syphilis rates.

The state-by-state numbers were released as part of a broader report from the Centers for Disease Control and Prevention tracking trends in sexually transmitted diseases. Cases of syphilis, gonorrhea, and chlamydia combined reached an all-time high in 2018. Cases of the most infectious stage of syphilis rose 14% to more than 35,000 cases; gonorrhea increased 5% to more than 580,000 cases; and chlamydia increased 3% to more than 1.7 million cases.

For veteran public health workers, the upward trend in congenital syphilis numbers is particularly disturbing because the condition is so easy to prevent. Blood tests can identify infection in pregnant women. The treatment is relatively simple and effective. When caught during pregnancy, transmission from mother to baby generally can be stopped.

“When we see a case of congenital syphilis, it is a hallmark of a health system and a health care failure,” said Virginia Bowen, PhD, an epidemiologist with the CDC and an author of the report.

It takes just a few shots of antibiotics to prevent a baby from getting syphilis from its mother. Left untreated, Treponema pallidum, the corkscrew-shaped organism that causes syphilis, can wiggle its way through a mother’s placenta and into a fetus. Once there, it can multiply furiously, invading every part of the body.

The effects on a newborn can be devastating. Philip Cheng, MD, is a neonatologist at St. Joseph’s Medical Center in Stockton, a city in San Joaquin County in California’s Central Valley. Twenty-six babies were infected last year in San Joaquin County, according to state data.

The brain of one of Cheng’s patients didn’t develop properly and the baby died shortly after birth. Other young patients survive but battle blood abnormalities, bone deformities, and organ damage. Congenital syphilis can cause blindness and excruciating pain.

Public health departments across the Central Valley, a largely rural expanse, report similar experiences. Following the release of the CDC report Tuesday, the California Department of Public Health released its county-by-county numbers for 2018. The report showed syphilis, gonorrhea, and chlamydia levels at their highest in 30 years, and attributed 22 stillbirths or neonatal deaths to congenital syphilis.

For the past several years, Fresno County, which had 63 cases of congenital syphilis in 2017, had the highest rate in California. In 2018, Fresno fell to fourth, behind Yuba, Kern, and San Joaquin counties. But the epidemic is far from under control. “I couldn’t even tell you how soon I think we’re going to see a decrease,” said Jena Adams, who oversees HIV and STD programs for Fresno County.

Syphilis was once a prolific and widely feared STD. But by the 1940s, penicillin was found to have a near-perfect cure rate for the disease. By 2000, syphilis rates were so low in the U.S. that the federal government launched a plan to eliminate the disease. Today, that goal is a distant memory.

Health departments once tracked down every person who tested positive for chlamydia, gonorrhea, or syphilis, to make sure they and their partners got treatment. With limited funds and climbing caseloads, many states now devote resources only to tracking syphilis. The caseloads are so high in some California counties that they track only women of childbearing age or just pregnant women.

“A lot of the funding for day-to-day public health work isn’t there,” said Jeffrey Klausner, MD, a professor at the University of California-Los Angeles who ran San Francisco’s STD program for more than a decade.

The bulk of STD prevention funding is appropriated by Congress to the CDC, which passes it on to states. That funding has been largely flat since 2003, according to data from the National Coalition of STD Directors, which represents health departments across the country. Take into account inflation and the growing caseloads, and the money is spread thinner. “It takes money, it takes training, it takes resources,” Dr. Klausner said, “and policymakers have just not prioritized that.”

A report this year by Trust for America’s Health, a public health policy research and advocacy group, estimated that 55,000 jobs were cut from local public health departments from 2008 to 2017. “We have our hands tied as much as [states] do,” said Dr. Bowen of the CDC. “We take what we’re given and try to distribute it as fairly as we can.”

San Joaquin County health officials have reorganized the department and applied for grants to increase the number of investigators available while congenital syphilis has spiked, said Hemal Parikh, county coordinator for STD control. But even with new hires and cutting back to tracking only women of childbearing age with syphilis, an investigator can have anywhere from 20 to 30 open cases at a time. In other counties, the caseload can be double that.

In 2018, Jennifer Wagman, PhD, a UCLA professor who studies infectious diseases and gender inequality, was part of a group that received CDC funding to look into what is causing the spike in congenital syphilis in California’s Central Valley.

Dr. Wagman said that, after years of studying health systems in other countries, she was shocked to see how much basic public health infrastructure has crumbled in California. In many parts of the Central Valley, county walk-in clinics that tested for and treated STDs were shuttered in the wake of the recession. That left few places for drop-in care, and investigators with no place to take someone for immediate treatment. Investigators or their patients must make appointments at one of the few providers who carry the right kind of treatment and hope the patients can keep the appointment when the time comes.

In focus groups, women told Dr. Wagman that working hourly jobs, or dealing with chaotic lives involving homelessness, abusive partners, and drug use, can make it all but impossible to stick to the appointments required at private clinics.

Dr. Wagman found that women in these high-risk groups were seeking care, though sometimes late in their pregnancy. They were just more likely to visit an emergency room, urgent care, or even a methadone clinic – places that take drop-ins but don’t necessarily routinely test for or treat syphilis.

“These people already have a million barriers,” said Jenny Malone, the public health nurse for San Joaquin County. “Now there are more.”

The most challenging cases in California are wrapped up with the state’s growing housing crisis and a methamphetamine epidemic with few treatment options. Women who are homeless often have unreliable contact information and are unlikely to have a primary care doctor. That makes them tough to track down to give a positive diagnosis or to follow up on a treatment plan.

Louisiana had the highest rate of congenital syphilis in the country for several years – until 2018. After a 22% drop in its rate, combined with increases in other states, Louisiana now ranks behind Texas and Nevada. That drop is the direct result of $550 million in temporary supplemental funding that the CDC gave the state to combat the epidemic, said Chaquetta Johnson, DNP, deputy director of operations for the state’s STD/HIV/hepatitis program. The money helped bolster the state’s lagging public health infrastructure. It was used to host two conferences for providers in the hardest-hit areas, hire two case managers and a nurse educator, create a program for in-home treatment, and improve data systems to track cases, among other things.

In California, more than 40% of pregnant women with syphilis passed it on to their baby in 2016, the most recent year for which data is available. Gov. Gavin Newsom (D) made additional funding available this year, but it’s a “drop in the bucket,” said Sergio Morales of Essential Access Health, a nonprofit that focuses on sexual and reproductive health and is working with Kern County on congenital syphilis. “We are seeing the results of years of inaction and a lack of prioritization of STD prevention, and we’re now paying the price.”
 

This KHN story first published on California Healthline, a service of the California Health Care Foundation. Kaiser Health News is a nonprofit national health policy news service. It is an editorially independent program of the Henry J. Kaiser Family Foundation that is not affiliated with Kaiser Permanente.

[Update: This story was revised at 6:50 p.m. ET on Oct. 8 to reflect news developments.]
 

A study of dupilumab in adolescents with atopic dermatitis (AD) has provided longer term efficacy and safety data, with no new safety signals and continued evidence of efficacy for up to 52 weeks, reported the authors of the study, published online Oct. 9 in the British Journal of Dermatology.

The phase 2a open-label, ascending-dose cohort study of dupilumab in 40 adolescents with moderate to severe AD was followed by a 48-week phase 3 open-label extension study in 36 of those participants. Dupilumab is a monoclonal antibody that inhibits signaling of interleukin (IL)-4 and IL-13.

In the phase 2a study, participants were treated with a single subcutaneous dose of dupilumab – either 2 mg/kg or 4 mg/kg – and had 8 weeks of pharmacokinetic sampling. They subsequently received that same dose weekly for 4 weeks, with an 8-week-long safety follow-up period. Those who participated in the open-label extension continued their weekly dose to a maximum of 300 mg. per kg

The most common treatment-emergent adverse events (a primary endpoint) seen in both the phase 2a and phase 3 studies were nasopharyngitis and exacerbation of AD – in the phase 2a study, exacerbations were seen in the period when patients weren’t taking the treatment. In the 2-mg and 4-mg groups, the incidence of skin infections was 29% and 42%, respectively, and the incidence of injection site reactions – which were mostly mild – were 18% and 11%, respectively. Researchers also noted conjunctivitis in 18% and 16% of the patients in the 2-mg and 4-mg groups, respectively, but none of the cases were considered serious and all resolved over the course of the study. In the phase 2a study, 50% of patients on the 2-mg/kg dose and 65% of those on the 4-mg/kg dose experienced an adverse event, while in the open-label extension all reported at least one adverse event.

There was one case of suicidal behavior and one case of systemic or severe hypersensitivity reported in the 2-mg/kg groups, both of which were considered adverse events of special interest. There were no deaths.

However none of the serious adverse events – which included infected AD, palpitations, patent ductus arteriosus, and food allergy – were linked to the study treatment, and no adverse events led to study discontinuation, the authors reported.

By week 12, 70% of participants in the 2-mg/kg group and 75% in the 4-mg/kg group had achieved a 50% or greater improvement in their Eczema Area and Severity Index (EASI) scores, which was a secondary outcome. By week 52, that had increased to 100% and 89% respectively.

More than half the patients (55%) in the 2-mg/kg group, and 40% of those in the 4-mg/kg group achieved a 75% or more improvement in their EASI scores by week 12, which increased to 88% and 78%, respectively, by week 52 in the open label phase.

“The results from these studies support use of dupilumab for the long-term management of moderate to severe AD in adolescents,” wrote Michael J. Cork, MD, professor of dermatology, University of Sheffield, England, and coauthors. No new safety signals were identified, “compared with the known safety profile of dupilumab in adults with moderate to severe AD,” and “the PK profile was characterized by nonlinear, target-mediated kinetics, consistent with the profile in adults with moderate to severe AD,” they added.

Dupilumab was approved in the United States in March 2019 for adolescents with moderate to severe AD whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable.

The study was sponsored by dupilumab manufacturers Sanofi and Regeneron Pharmaceuticals, which market dupilumab as Dupixent in the United States. Dr. Cork disclosures included those related to Sanofi Genzyme and Regeneron; other authors included employees of the companies.

SOURCE: Cork M et al. Br J Dermatol. 2019 Oct 9. doi: 10.1111/bjd.18476.

A study of dupilumab in adolescents with atopic dermatitis (AD) has provided longer term efficacy and safety data, with no new safety signals and continued evidence of efficacy for up to 52 weeks, reported the authors of the study, published online Oct. 9 in the British Journal of Dermatology.

The phase 2a open-label, ascending-dose cohort study of dupilumab in 40 adolescents with moderate to severe AD was followed by a 48-week phase 3 open-label extension study in 36 of those participants. Dupilumab is a monoclonal antibody that inhibits signaling of interleukin (IL)-4 and IL-13.

In the phase 2a study, participants were treated with a single subcutaneous dose of dupilumab – either 2 mg/kg or 4 mg/kg – and had 8 weeks of pharmacokinetic sampling. They subsequently received that same dose weekly for 4 weeks, with an 8-week-long safety follow-up period. Those who participated in the open-label extension continued their weekly dose to a maximum of 300 mg. per kg

The most common treatment-emergent adverse events (a primary endpoint) seen in both the phase 2a and phase 3 studies were nasopharyngitis and exacerbation of AD – in the phase 2a study, exacerbations were seen in the period when patients weren’t taking the treatment. In the 2-mg and 4-mg groups, the incidence of skin infections was 29% and 42%, respectively, and the incidence of injection site reactions – which were mostly mild – were 18% and 11%, respectively. Researchers also noted conjunctivitis in 18% and 16% of the patients in the 2-mg and 4-mg groups, respectively, but none of the cases were considered serious and all resolved over the course of the study. In the phase 2a study, 50% of patients on the 2-mg/kg dose and 65% of those on the 4-mg/kg dose experienced an adverse event, while in the open-label extension all reported at least one adverse event.

There was one case of suicidal behavior and one case of systemic or severe hypersensitivity reported in the 2-mg/kg groups, both of which were considered adverse events of special interest. There were no deaths.

However none of the serious adverse events – which included infected AD, palpitations, patent ductus arteriosus, and food allergy – were linked to the study treatment, and no adverse events led to study discontinuation, the authors reported.

By week 12, 70% of participants in the 2-mg/kg group and 75% in the 4-mg/kg group had achieved a 50% or greater improvement in their Eczema Area and Severity Index (EASI) scores, which was a secondary outcome. By week 52, that had increased to 100% and 89% respectively.

More than half the patients (55%) in the 2-mg/kg group, and 40% of those in the 4-mg/kg group achieved a 75% or more improvement in their EASI scores by week 12, which increased to 88% and 78%, respectively, by week 52 in the open label phase.

“The results from these studies support use of dupilumab for the long-term management of moderate to severe AD in adolescents,” wrote Michael J. Cork, MD, professor of dermatology, University of Sheffield, England, and coauthors. No new safety signals were identified, “compared with the known safety profile of dupilumab in adults with moderate to severe AD,” and “the PK profile was characterized by nonlinear, target-mediated kinetics, consistent with the profile in adults with moderate to severe AD,” they added.

Dupilumab was approved in the United States in March 2019 for adolescents with moderate to severe AD whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable.

The study was sponsored by dupilumab manufacturers Sanofi and Regeneron Pharmaceuticals, which market dupilumab as Dupixent in the United States. Dr. Cork disclosures included those related to Sanofi Genzyme and Regeneron; other authors included employees of the companies.

SOURCE: Cork M et al. Br J Dermatol. 2019 Oct 9. doi: 10.1111/bjd.18476.

A study of dupilumab in adolescents with atopic dermatitis (AD) has provided longer term efficacy and safety data, with no new safety signals and continued evidence of efficacy for up to 52 weeks, reported the authors of the study, published online Oct. 9 in the British Journal of Dermatology.

The phase 2a open-label, ascending-dose cohort study of dupilumab in 40 adolescents with moderate to severe AD was followed by a 48-week phase 3 open-label extension study in 36 of those participants. Dupilumab is a monoclonal antibody that inhibits signaling of interleukin (IL)-4 and IL-13.

In the phase 2a study, participants were treated with a single subcutaneous dose of dupilumab – either 2 mg/kg or 4 mg/kg – and had 8 weeks of pharmacokinetic sampling. They subsequently received that same dose weekly for 4 weeks, with an 8-week-long safety follow-up period. Those who participated in the open-label extension continued their weekly dose to a maximum of 300 mg. per kg

The most common treatment-emergent adverse events (a primary endpoint) seen in both the phase 2a and phase 3 studies were nasopharyngitis and exacerbation of AD – in the phase 2a study, exacerbations were seen in the period when patients weren’t taking the treatment. In the 2-mg and 4-mg groups, the incidence of skin infections was 29% and 42%, respectively, and the incidence of injection site reactions – which were mostly mild – were 18% and 11%, respectively. Researchers also noted conjunctivitis in 18% and 16% of the patients in the 2-mg and 4-mg groups, respectively, but none of the cases were considered serious and all resolved over the course of the study. In the phase 2a study, 50% of patients on the 2-mg/kg dose and 65% of those on the 4-mg/kg dose experienced an adverse event, while in the open-label extension all reported at least one adverse event.

There was one case of suicidal behavior and one case of systemic or severe hypersensitivity reported in the 2-mg/kg groups, both of which were considered adverse events of special interest. There were no deaths.

However none of the serious adverse events – which included infected AD, palpitations, patent ductus arteriosus, and food allergy – were linked to the study treatment, and no adverse events led to study discontinuation, the authors reported.

By week 12, 70% of participants in the 2-mg/kg group and 75% in the 4-mg/kg group had achieved a 50% or greater improvement in their Eczema Area and Severity Index (EASI) scores, which was a secondary outcome. By week 52, that had increased to 100% and 89% respectively.

More than half the patients (55%) in the 2-mg/kg group, and 40% of those in the 4-mg/kg group achieved a 75% or more improvement in their EASI scores by week 12, which increased to 88% and 78%, respectively, by week 52 in the open label phase.

“The results from these studies support use of dupilumab for the long-term management of moderate to severe AD in adolescents,” wrote Michael J. Cork, MD, professor of dermatology, University of Sheffield, England, and coauthors. No new safety signals were identified, “compared with the known safety profile of dupilumab in adults with moderate to severe AD,” and “the PK profile was characterized by nonlinear, target-mediated kinetics, consistent with the profile in adults with moderate to severe AD,” they added.

Dupilumab was approved in the United States in March 2019 for adolescents with moderate to severe AD whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable.

The study was sponsored by dupilumab manufacturers Sanofi and Regeneron Pharmaceuticals, which market dupilumab as Dupixent in the United States. Dr. Cork disclosures included those related to Sanofi Genzyme and Regeneron; other authors included employees of the companies.

SOURCE: Cork M et al. Br J Dermatol. 2019 Oct 9. doi: 10.1111/bjd.18476.

After years of relative stability, firearm-related mortality in the United States rose sharply starting in 2015, according to analysis of a national mortality database.

U.S. firearm mortality was 10.4 per 100,000 person-years during 1999-2014, with the high in that period occurring in 2012 and dropping each of the next 2 years – compared with 11.8 per 100,000 during 2015-2017, an increase of 13.8%, Jason E. Goldstick, PhD, and associates wrote Oct. 8 in Health Affairs.

The majority of the 612,310 firearm deaths over the entire study period were suicides, with the proportion rising slightly from 58.6% in 1999-2014 to 60.0% in 2015-2017. Homicides made up 38.5% of deaths in 1999-2014 and 37.9% in 2015-2017, while the combined share of unintentional and undetermined deaths dropped from 2.9% to 2.1%, the investigators reported.

“What we can see is a worsening epidemic of firearm mortality that is geographically and demographically broad,” Dr. Goldstick of the University of Michigan, Ann Arbor, said in a separate written statement.

The geographic broadness can be seen when the change in mortality from 1999-2014 to 2015-2017 was calculated for each locale: 29 states had an increase of more than 20% and only 3 states (California, New York, and Rhode Island) and the District of Columbia had a decrease of at least 12.5%, they said. The data came from the Centers for Disease Control and Prevention’s Wide-ranging Online Data for Epidemiologic Research tool.

The different trends among states and subpopulations make it difficult to offer policy-based interventions. “The epidemiology of firearm violence is complex and varies based on the mechanism of death, demographic group under study, and regionally specific culture, making a one-size-fits-all solution inappropriate,” Dr. Goldstick and associates wrote.

The study was funded mainly by a grant from the National Institute of Child Health and Human Development. The investigators did not provide any information on conflicts of interest.

rfranki@mdedge.com

SOURCE: Goldstick JE et al. Health Aff. 2019;38(10):1646-52.

After years of relative stability, firearm-related mortality in the United States rose sharply starting in 2015, according to analysis of a national mortality database.

U.S. firearm mortality was 10.4 per 100,000 person-years during 1999-2014, with the high in that period occurring in 2012 and dropping each of the next 2 years – compared with 11.8 per 100,000 during 2015-2017, an increase of 13.8%, Jason E. Goldstick, PhD, and associates wrote Oct. 8 in Health Affairs.

The majority of the 612,310 firearm deaths over the entire study period were suicides, with the proportion rising slightly from 58.6% in 1999-2014 to 60.0% in 2015-2017. Homicides made up 38.5% of deaths in 1999-2014 and 37.9% in 2015-2017, while the combined share of unintentional and undetermined deaths dropped from 2.9% to 2.1%, the investigators reported.

“What we can see is a worsening epidemic of firearm mortality that is geographically and demographically broad,” Dr. Goldstick of the University of Michigan, Ann Arbor, said in a separate written statement.

The geographic broadness can be seen when the change in mortality from 1999-2014 to 2015-2017 was calculated for each locale: 29 states had an increase of more than 20% and only 3 states (California, New York, and Rhode Island) and the District of Columbia had a decrease of at least 12.5%, they said. The data came from the Centers for Disease Control and Prevention’s Wide-ranging Online Data for Epidemiologic Research tool.

The different trends among states and subpopulations make it difficult to offer policy-based interventions. “The epidemiology of firearm violence is complex and varies based on the mechanism of death, demographic group under study, and regionally specific culture, making a one-size-fits-all solution inappropriate,” Dr. Goldstick and associates wrote.

The study was funded mainly by a grant from the National Institute of Child Health and Human Development. The investigators did not provide any information on conflicts of interest.

rfranki@mdedge.com

SOURCE: Goldstick JE et al. Health Aff. 2019;38(10):1646-52.

After years of relative stability, firearm-related mortality in the United States rose sharply starting in 2015, according to analysis of a national mortality database.

U.S. firearm mortality was 10.4 per 100,000 person-years during 1999-2014, with the high in that period occurring in 2012 and dropping each of the next 2 years – compared with 11.8 per 100,000 during 2015-2017, an increase of 13.8%, Jason E. Goldstick, PhD, and associates wrote Oct. 8 in Health Affairs.

The majority of the 612,310 firearm deaths over the entire study period were suicides, with the proportion rising slightly from 58.6% in 1999-2014 to 60.0% in 2015-2017. Homicides made up 38.5% of deaths in 1999-2014 and 37.9% in 2015-2017, while the combined share of unintentional and undetermined deaths dropped from 2.9% to 2.1%, the investigators reported.

“What we can see is a worsening epidemic of firearm mortality that is geographically and demographically broad,” Dr. Goldstick of the University of Michigan, Ann Arbor, said in a separate written statement.

The geographic broadness can be seen when the change in mortality from 1999-2014 to 2015-2017 was calculated for each locale: 29 states had an increase of more than 20% and only 3 states (California, New York, and Rhode Island) and the District of Columbia had a decrease of at least 12.5%, they said. The data came from the Centers for Disease Control and Prevention’s Wide-ranging Online Data for Epidemiologic Research tool.

The different trends among states and subpopulations make it difficult to offer policy-based interventions. “The epidemiology of firearm violence is complex and varies based on the mechanism of death, demographic group under study, and regionally specific culture, making a one-size-fits-all solution inappropriate,” Dr. Goldstick and associates wrote.

The study was funded mainly by a grant from the National Institute of Child Health and Human Development. The investigators did not provide any information on conflicts of interest.

rfranki@mdedge.com

SOURCE: Goldstick JE et al. Health Aff. 2019;38(10):1646-52.

An online assessment tool can be used to identify patients with inflammatory bowel disease (IBD) who are receiving an excess of corticosteroids, and a quality improvement plan lowered the use of excess corticosteroids, according to recent research in the journal Alimentary Pharmacology & Therapeutics.

Since measurement of excess corticosteroid use can be measured through an online assessment tool in clinical practice and long-term corticosteroid use is associated with adverse outcomes, it may be a quality marker for patients with IBD, wrote Christian P. Selinger, MD, from the Leeds (England) Gastroenterology Institute and colleagues. “Such key performance indicators have previously been lacking in IBD, unlike other disease areas such as diabetes and cardiovascular disease.”

Over a period of 3 months, Dr. Selinger and colleagues collected prospective data from 2,385 patients with IBD at 19 centers in England, Wales, and Scotland who had received steroids within the last year. The researchers divided the centers into groups based on whether they participated in the quality improvement program (7 centers), were new to the process of collecting data on steroid use (11 centers), or did not participate in the program (1 center). The seven centers that participated in the intervention were part of an audit that began in 2017, while the other centers were evaluated over a 3-month period between April and July 2017. Patients were asked questions about their steroid use, including whether the steroids were prescribed for their IBD, how long the course of steroids was, how many courses of steroids they received, and if they were able to stop using steroids without their symptoms returning.

The researchers found 14.8% of patients had an excess of steroids or were dependent on steroids, and patients at centers that participated in the quality improvement programs had a lower rate of exposure (23.8% vs. 31.0%; P less than .001) and a lower rate of steroid excess (11.5% vs. 17.1%; P less than .001) than did patients at sites that did not participate in the program. Centers with the improvement program also had steroid use decrease over time, from 30.0% in 2015 to 23.8% in 2017 (P = .003), and steroid excess also decreased from 13.8% at those centers to 11.5% during that time (P equals .17). The researchers noted that, in over half of cases (50.7%), the steroid excess was “avoidable.”

In patients with Crohn’s disease, those who had reduced steroid excess were more likely to be part of an intervention center (odds ratio, 0.72; 95% confidence interval, 0.46-0.97), at a center with a multidisciplinary team (OR, 0.54; 95% CI, 0.20-0.86), or receiving maintenance anti–tumor necrosis factor therapy (OR, 0.61; 95% CI, 0.24-0.95); in contrast, patients who received aminosalicylate were more likely to have steroid excess (OR, 1.72; 95% CI, 1.24-2.09). Steroid excess in ulcerative colitis (UC) patients was more likely among those receiving thiopurine monotherapy (OR, 1.97; 95% CI, 1.19‐3.01), while UC patients at an intervention center were less likely to have steroid excess (OR; 0.72; 95% CI, 0.45‐0.95).

The researchers said the online assessment is limited in assessing the reason for steroid excess and is unable to consider variables such as patient age, sex, IBD phenotype, and disease duration, but is a “simple, pragmatic tool” that can be used in real time in a clinical setting.

“This advances the case for steroid excess as a potential key performance indicator of quality in an IBD service, although in order for clinicians to benchmark their service and provide targets for improvements, any numerical goal attached to this key performance indicator would require consideration of case mix. Further data, including from national and international contexts, is needed,” concluded Dr. Selinger and colleagues.

The authors reported AbbVie provided the funding to develop the steroid assessment tool, as well as honoraria for invited attendees of the quality improvement plan, which the company also sponsored.

To help your patients better understand their treatment options, share AGA’s IBD patient education, which is online at www.gastro.org/practice-guidance/gi-patient-center/topic/inflammatory-bowel-disease. 

SOURCE: Selinger CP et al. Aliment Pharmacol Ther. 2019. doi: 10.1111/apt.15497.

An online assessment tool can be used to identify patients with inflammatory bowel disease (IBD) who are receiving an excess of corticosteroids, and a quality improvement plan lowered the use of excess corticosteroids, according to recent research in the journal Alimentary Pharmacology & Therapeutics.

Since measurement of excess corticosteroid use can be measured through an online assessment tool in clinical practice and long-term corticosteroid use is associated with adverse outcomes, it may be a quality marker for patients with IBD, wrote Christian P. Selinger, MD, from the Leeds (England) Gastroenterology Institute and colleagues. “Such key performance indicators have previously been lacking in IBD, unlike other disease areas such as diabetes and cardiovascular disease.”

Over a period of 3 months, Dr. Selinger and colleagues collected prospective data from 2,385 patients with IBD at 19 centers in England, Wales, and Scotland who had received steroids within the last year. The researchers divided the centers into groups based on whether they participated in the quality improvement program (7 centers), were new to the process of collecting data on steroid use (11 centers), or did not participate in the program (1 center). The seven centers that participated in the intervention were part of an audit that began in 2017, while the other centers were evaluated over a 3-month period between April and July 2017. Patients were asked questions about their steroid use, including whether the steroids were prescribed for their IBD, how long the course of steroids was, how many courses of steroids they received, and if they were able to stop using steroids without their symptoms returning.

The researchers found 14.8% of patients had an excess of steroids or were dependent on steroids, and patients at centers that participated in the quality improvement programs had a lower rate of exposure (23.8% vs. 31.0%; P less than .001) and a lower rate of steroid excess (11.5% vs. 17.1%; P less than .001) than did patients at sites that did not participate in the program. Centers with the improvement program also had steroid use decrease over time, from 30.0% in 2015 to 23.8% in 2017 (P = .003), and steroid excess also decreased from 13.8% at those centers to 11.5% during that time (P equals .17). The researchers noted that, in over half of cases (50.7%), the steroid excess was “avoidable.”

In patients with Crohn’s disease, those who had reduced steroid excess were more likely to be part of an intervention center (odds ratio, 0.72; 95% confidence interval, 0.46-0.97), at a center with a multidisciplinary team (OR, 0.54; 95% CI, 0.20-0.86), or receiving maintenance anti–tumor necrosis factor therapy (OR, 0.61; 95% CI, 0.24-0.95); in contrast, patients who received aminosalicylate were more likely to have steroid excess (OR, 1.72; 95% CI, 1.24-2.09). Steroid excess in ulcerative colitis (UC) patients was more likely among those receiving thiopurine monotherapy (OR, 1.97; 95% CI, 1.19‐3.01), while UC patients at an intervention center were less likely to have steroid excess (OR; 0.72; 95% CI, 0.45‐0.95).

The researchers said the online assessment is limited in assessing the reason for steroid excess and is unable to consider variables such as patient age, sex, IBD phenotype, and disease duration, but is a “simple, pragmatic tool” that can be used in real time in a clinical setting.

“This advances the case for steroid excess as a potential key performance indicator of quality in an IBD service, although in order for clinicians to benchmark their service and provide targets for improvements, any numerical goal attached to this key performance indicator would require consideration of case mix. Further data, including from national and international contexts, is needed,” concluded Dr. Selinger and colleagues.

The authors reported AbbVie provided the funding to develop the steroid assessment tool, as well as honoraria for invited attendees of the quality improvement plan, which the company also sponsored.

To help your patients better understand their treatment options, share AGA’s IBD patient education, which is online at www.gastro.org/practice-guidance/gi-patient-center/topic/inflammatory-bowel-disease. 

SOURCE: Selinger CP et al. Aliment Pharmacol Ther. 2019. doi: 10.1111/apt.15497.

An online assessment tool can be used to identify patients with inflammatory bowel disease (IBD) who are receiving an excess of corticosteroids, and a quality improvement plan lowered the use of excess corticosteroids, according to recent research in the journal Alimentary Pharmacology & Therapeutics.

Since measurement of excess corticosteroid use can be measured through an online assessment tool in clinical practice and long-term corticosteroid use is associated with adverse outcomes, it may be a quality marker for patients with IBD, wrote Christian P. Selinger, MD, from the Leeds (England) Gastroenterology Institute and colleagues. “Such key performance indicators have previously been lacking in IBD, unlike other disease areas such as diabetes and cardiovascular disease.”

Over a period of 3 months, Dr. Selinger and colleagues collected prospective data from 2,385 patients with IBD at 19 centers in England, Wales, and Scotland who had received steroids within the last year. The researchers divided the centers into groups based on whether they participated in the quality improvement program (7 centers), were new to the process of collecting data on steroid use (11 centers), or did not participate in the program (1 center). The seven centers that participated in the intervention were part of an audit that began in 2017, while the other centers were evaluated over a 3-month period between April and July 2017. Patients were asked questions about their steroid use, including whether the steroids were prescribed for their IBD, how long the course of steroids was, how many courses of steroids they received, and if they were able to stop using steroids without their symptoms returning.

The researchers found 14.8% of patients had an excess of steroids or were dependent on steroids, and patients at centers that participated in the quality improvement programs had a lower rate of exposure (23.8% vs. 31.0%; P less than .001) and a lower rate of steroid excess (11.5% vs. 17.1%; P less than .001) than did patients at sites that did not participate in the program. Centers with the improvement program also had steroid use decrease over time, from 30.0% in 2015 to 23.8% in 2017 (P = .003), and steroid excess also decreased from 13.8% at those centers to 11.5% during that time (P equals .17). The researchers noted that, in over half of cases (50.7%), the steroid excess was “avoidable.”

In patients with Crohn’s disease, those who had reduced steroid excess were more likely to be part of an intervention center (odds ratio, 0.72; 95% confidence interval, 0.46-0.97), at a center with a multidisciplinary team (OR, 0.54; 95% CI, 0.20-0.86), or receiving maintenance anti–tumor necrosis factor therapy (OR, 0.61; 95% CI, 0.24-0.95); in contrast, patients who received aminosalicylate were more likely to have steroid excess (OR, 1.72; 95% CI, 1.24-2.09). Steroid excess in ulcerative colitis (UC) patients was more likely among those receiving thiopurine monotherapy (OR, 1.97; 95% CI, 1.19‐3.01), while UC patients at an intervention center were less likely to have steroid excess (OR; 0.72; 95% CI, 0.45‐0.95).

The researchers said the online assessment is limited in assessing the reason for steroid excess and is unable to consider variables such as patient age, sex, IBD phenotype, and disease duration, but is a “simple, pragmatic tool” that can be used in real time in a clinical setting.

“This advances the case for steroid excess as a potential key performance indicator of quality in an IBD service, although in order for clinicians to benchmark their service and provide targets for improvements, any numerical goal attached to this key performance indicator would require consideration of case mix. Further data, including from national and international contexts, is needed,” concluded Dr. Selinger and colleagues.

The authors reported AbbVie provided the funding to develop the steroid assessment tool, as well as honoraria for invited attendees of the quality improvement plan, which the company also sponsored.

To help your patients better understand their treatment options, share AGA’s IBD patient education, which is online at www.gastro.org/practice-guidance/gi-patient-center/topic/inflammatory-bowel-disease. 

SOURCE: Selinger CP et al. Aliment Pharmacol Ther. 2019. doi: 10.1111/apt.15497.

An online assessment tool can be used to identify patients with inflammatory bowel disease (IBD) who are receiving an excess of corticosteroids, and a quality improvement plan lowered the use of excess corticosteroids, according to recent research in the journal Alimentary Pharmacology & Therapeutics.

Since measurement of excess corticosteroid use can be measured through an online assessment tool in clinical practice and long-term corticosteroid use is associated with adverse outcomes, it may be a quality marker for patients with IBD, wrote Christian P. Selinger, MD, from the Leeds (England) Gastroenterology Institute and colleagues. “Such key performance indicators have previously been lacking in IBD, unlike other disease areas such as diabetes and cardiovascular disease.”

Over a period of 3 months, Dr. Selinger and colleagues collected prospective data from 2,385 patients with IBD at 19 centers in England, Wales, and Scotland who had received steroids within the last year. The researchers divided the centers into groups based on whether they participated in the quality improvement program (7 centers), were new to the process of collecting data on steroid use (11 centers), or did not participate in the program (1 center). The seven centers that participated in the intervention were part of an audit that began in 2017, while the other centers were evaluated over a 3-month period between April and July 2017. Patients were asked questions about their steroid use, including whether the steroids were prescribed for their IBD, how long the course of steroids was, how many courses of steroids they received, and if they were able to stop using steroids without their symptoms returning.

The researchers found 14.8% of patients had an excess of steroids or were dependent on steroids, and patients at centers that participated in the quality improvement programs had a lower rate of exposure (23.8% vs. 31.0%; P less than .001) and a lower rate of steroid excess (11.5% vs. 17.1%; P less than .001) than did patients at sites that did not participate in the program. Centers with the improvement program also had steroid use decrease over time, from 30.0% in 2015 to 23.8% in 2017 (P = .003), and steroid excess also decreased from 13.8% at those centers to 11.5% during that time (P equals .17). The researchers noted that, in over half of cases (50.7%), the steroid excess was “avoidable.”

In patients with Crohn’s disease, those who had reduced steroid excess were more likely to be part of an intervention center (odds ratio, 0.72; 95% confidence interval, 0.46-0.97), at a center with a multidisciplinary team (OR, 0.54; 95% CI, 0.20-0.86), or receiving maintenance anti–tumor necrosis factor therapy (OR, 0.61; 95% CI, 0.24-0.95); in contrast, patients who received aminosalicylate were more likely to have steroid excess (OR, 1.72; 95% CI, 1.24-2.09). Steroid excess in ulcerative colitis (UC) patients was more likely among those receiving thiopurine monotherapy (OR, 1.97; 95% CI, 1.19‐3.01), while UC patients at an intervention center were less likely to have steroid excess (OR; 0.72; 95% CI, 0.45‐0.95).

The researchers said the online assessment is limited in assessing the reason for steroid excess and is unable to consider variables such as patient age, sex, IBD phenotype, and disease duration, but is a “simple, pragmatic tool” that can be used in real time in a clinical setting.

“This advances the case for steroid excess as a potential key performance indicator of quality in an IBD service, although in order for clinicians to benchmark their service and provide targets for improvements, any numerical goal attached to this key performance indicator would require consideration of case mix. Further data, including from national and international contexts, is needed,” concluded Dr. Selinger and colleagues.

The authors reported AbbVie provided the funding to develop the steroid assessment tool, as well as honoraria for invited attendees of the quality improvement plan, which the company also sponsored.

SOURCE: Selinger CP et al. Aliment Pharmacol Ther. 2019. doi: 10.1111/apt.15497.

An online assessment tool can be used to identify patients with inflammatory bowel disease (IBD) who are receiving an excess of corticosteroids, and a quality improvement plan lowered the use of excess corticosteroids, according to recent research in the journal Alimentary Pharmacology & Therapeutics.

Since measurement of excess corticosteroid use can be measured through an online assessment tool in clinical practice and long-term corticosteroid use is associated with adverse outcomes, it may be a quality marker for patients with IBD, wrote Christian P. Selinger, MD, from the Leeds (England) Gastroenterology Institute and colleagues. “Such key performance indicators have previously been lacking in IBD, unlike other disease areas such as diabetes and cardiovascular disease.”

Over a period of 3 months, Dr. Selinger and colleagues collected prospective data from 2,385 patients with IBD at 19 centers in England, Wales, and Scotland who had received steroids within the last year. The researchers divided the centers into groups based on whether they participated in the quality improvement program (7 centers), were new to the process of collecting data on steroid use (11 centers), or did not participate in the program (1 center). The seven centers that participated in the intervention were part of an audit that began in 2017, while the other centers were evaluated over a 3-month period between April and July 2017. Patients were asked questions about their steroid use, including whether the steroids were prescribed for their IBD, how long the course of steroids was, how many courses of steroids they received, and if they were able to stop using steroids without their symptoms returning.

The researchers found 14.8% of patients had an excess of steroids or were dependent on steroids, and patients at centers that participated in the quality improvement programs had a lower rate of exposure (23.8% vs. 31.0%; P less than .001) and a lower rate of steroid excess (11.5% vs. 17.1%; P less than .001) than did patients at sites that did not participate in the program. Centers with the improvement program also had steroid use decrease over time, from 30.0% in 2015 to 23.8% in 2017 (P = .003), and steroid excess also decreased from 13.8% at those centers to 11.5% during that time (P equals .17). The researchers noted that, in over half of cases (50.7%), the steroid excess was “avoidable.”

In patients with Crohn’s disease, those who had reduced steroid excess were more likely to be part of an intervention center (odds ratio, 0.72; 95% confidence interval, 0.46-0.97), at a center with a multidisciplinary team (OR, 0.54; 95% CI, 0.20-0.86), or receiving maintenance anti–tumor necrosis factor therapy (OR, 0.61; 95% CI, 0.24-0.95); in contrast, patients who received aminosalicylate were more likely to have steroid excess (OR, 1.72; 95% CI, 1.24-2.09). Steroid excess in ulcerative colitis (UC) patients was more likely among those receiving thiopurine monotherapy (OR, 1.97; 95% CI, 1.19‐3.01), while UC patients at an intervention center were less likely to have steroid excess (OR; 0.72; 95% CI, 0.45‐0.95).

The researchers said the online assessment is limited in assessing the reason for steroid excess and is unable to consider variables such as patient age, sex, IBD phenotype, and disease duration, but is a “simple, pragmatic tool” that can be used in real time in a clinical setting.

“This advances the case for steroid excess as a potential key performance indicator of quality in an IBD service, although in order for clinicians to benchmark their service and provide targets for improvements, any numerical goal attached to this key performance indicator would require consideration of case mix. Further data, including from national and international contexts, is needed,” concluded Dr. Selinger and colleagues.

The authors reported AbbVie provided the funding to develop the steroid assessment tool, as well as honoraria for invited attendees of the quality improvement plan, which the company also sponsored.

SOURCE: Selinger CP et al. Aliment Pharmacol Ther. 2019. doi: 10.1111/apt.15497.

An online assessment tool can be used to identify patients with inflammatory bowel disease (IBD) who are receiving an excess of corticosteroids, and a quality improvement plan lowered the use of excess corticosteroids, according to recent research in the journal Alimentary Pharmacology & Therapeutics.

Since measurement of excess corticosteroid use can be measured through an online assessment tool in clinical practice and long-term corticosteroid use is associated with adverse outcomes, it may be a quality marker for patients with IBD, wrote Christian P. Selinger, MD, from the Leeds (England) Gastroenterology Institute and colleagues. “Such key performance indicators have previously been lacking in IBD, unlike other disease areas such as diabetes and cardiovascular disease.”

Over a period of 3 months, Dr. Selinger and colleagues collected prospective data from 2,385 patients with IBD at 19 centers in England, Wales, and Scotland who had received steroids within the last year. The researchers divided the centers into groups based on whether they participated in the quality improvement program (7 centers), were new to the process of collecting data on steroid use (11 centers), or did not participate in the program (1 center). The seven centers that participated in the intervention were part of an audit that began in 2017, while the other centers were evaluated over a 3-month period between April and July 2017. Patients were asked questions about their steroid use, including whether the steroids were prescribed for their IBD, how long the course of steroids was, how many courses of steroids they received, and if they were able to stop using steroids without their symptoms returning.

The researchers found 14.8% of patients had an excess of steroids or were dependent on steroids, and patients at centers that participated in the quality improvement programs had a lower rate of exposure (23.8% vs. 31.0%; P less than .001) and a lower rate of steroid excess (11.5% vs. 17.1%; P less than .001) than did patients at sites that did not participate in the program. Centers with the improvement program also had steroid use decrease over time, from 30.0% in 2015 to 23.8% in 2017 (P = .003), and steroid excess also decreased from 13.8% at those centers to 11.5% during that time (P equals .17). The researchers noted that, in over half of cases (50.7%), the steroid excess was “avoidable.”

In patients with Crohn’s disease, those who had reduced steroid excess were more likely to be part of an intervention center (odds ratio, 0.72; 95% confidence interval, 0.46-0.97), at a center with a multidisciplinary team (OR, 0.54; 95% CI, 0.20-0.86), or receiving maintenance anti–tumor necrosis factor therapy (OR, 0.61; 95% CI, 0.24-0.95); in contrast, patients who received aminosalicylate were more likely to have steroid excess (OR, 1.72; 95% CI, 1.24-2.09). Steroid excess in ulcerative colitis (UC) patients was more likely among those receiving thiopurine monotherapy (OR, 1.97; 95% CI, 1.19‐3.01), while UC patients at an intervention center were less likely to have steroid excess (OR; 0.72; 95% CI, 0.45‐0.95).

The researchers said the online assessment is limited in assessing the reason for steroid excess and is unable to consider variables such as patient age, sex, IBD phenotype, and disease duration, but is a “simple, pragmatic tool” that can be used in real time in a clinical setting.

“This advances the case for steroid excess as a potential key performance indicator of quality in an IBD service, although in order for clinicians to benchmark their service and provide targets for improvements, any numerical goal attached to this key performance indicator would require consideration of case mix. Further data, including from national and international contexts, is needed,” concluded Dr. Selinger and colleagues.

The authors reported AbbVie provided the funding to develop the steroid assessment tool, as well as honoraria for invited attendees of the quality improvement plan, which the company also sponsored.

SOURCE: Selinger CP et al. Aliment Pharmacol Ther. 2019. doi: 10.1111/apt.15497.