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Knowledge Gaps About Obesity Medicine Seen in Primary Care
SAN ANTONIO — Despite the prevalence of obesity in primary care, there appear to be major knowledge gaps among providers regarding obesity management, new research suggests.
Anonymous surveys of 96 primary care providers at a Boston, Massachusetts, safety-net hospital revealed that participants had limited understanding of criteria for prescribing antiobesity medications (AOM), and expressed discomfort in prescribing AOMs because of knowledge concerns, especially for non–glucagon-like peptide 1 (GLP-1) receptor agonists. One third reported that they didn’t prescribe AOMs, and rates of referral for bariatric surgery were also low.
The findings were presented at the Obesity Society’s annual Obesity Week meeting by Alejandro Campos, MD, a third-year resident in the section of internal medicine, Boston Medical Center, and the Department of Medicine, Boston University.
“I think it comes down to education. ... Not only training primary care physicians or residents about criteria and pathophysiology, but also stigma. Perceptions need to be addressed from the start of training in the healthcare field,” Campos told this news organization in an interview.
During his presentation, Campos noted this is the first such study in the setting of a safety-net hospital, which cares for lower-income people who experience disproportionate rates of obesity. But, “these findings are similar to ones observed from non–safety-net settings, which can indicate some potential transferability.”
Asked to comment, session moderator John D. Clark, MD, PhD, Chief Population Health Officer at Sharp Rees-Stealy Medical Group, San Diego, California, told this news organization that the findings didn’t surprise him. “I’d say that medical education around obesity has definitely improved, and training is improving but it’s not uniform. The treatment of obesity as a disease, especially with some of the newer medical treatments, is not standard of care and practiced widely.”
The study involved a standard-model Knowledge, Attitudes, and Practices questionnaire, distributed electronically for anonymous responses among both trained and in-training primary care providers. It contained a total of 43 items, 7 of them demographic, 11 on knowledge, 9 regarding attitudes, and 16 asking about practices.
The hospital is the largest safety-net hospital in New England, with a patient population that includes 58% enrolled in Medicaid, 32% Black/African American individuals, 24% identifying as Hispanic/Latino individuals, and 37% living below the poverty line.
The 96 responding providers (from a total 350 invited) all worked in either family medicine or internal medicine. The trained providers included both attending MDs and nurse practitioners, while those in-training were residents in one of those two specialties. Two thirds were women. The majority were aged 20-30 years (49.45%) or 31-40 years (27.47%).
Overall, 73.63% reported having received some type of obesity training. Just over half (52.08%) reported receiving that training during medical or nursing school, while 43.75% reported receiving it during residency.
When asked to choose from a list of conditions to pick which are considered weight-related comorbidities, between 80% and 90% choose type 2 diabetes, obstructive sleep apnea (OSA), hypertension, hyperlipidemia, nonalcoholic fatty liver disease, and coronary artery disease. Fewer, but still a majority, also listed osteoarthritis and gastroesophageal reflux disease. However, respondents were less likely to cite cancer, mood disorders, or chronic kidney disease as being related to obesity.
Asked to list benefits of a 10% body weight loss, most recognized reductions in OSA, glycemia, cardiovascular disease risk, osteoarthritis, and hepatic steatosis. But, only about half knew weight loss could also improve urinary incontinence.
Only 25% could correctly name both indications for AOMs. Just 27.1% knew that one was a body mass index (BMI) ≥ 27 with comorbidities, while 46.9% knew BMI ≥ 30 without comorbidities was an AOM indication. Only 9.4% were correct on both of those indications for bariatric surgery.
“Reassuringly,” Campos said, the majority either “disagreed” or “strongly disagreed” that “lack of will power” contributes to obesity. However, more than 20% agreed that “lack of exercise or physical activity” contributed.
Overall, 73% of the trained providers and 59% of those in training reported that they prescribe AOMs. Asked about their comfort level in prescribing specific types of AOMs, many more endorsed semaglutide and liraglutide than older medications such as bupropion/naltrexone and phentermine/topiramate.
Asked about factors that influence their comfort with prescribing AOMs, the top five factors selected, in order, were side-effect knowledge, insurance coverage, safety issues, and dosing knowledge. Fewer respondents endorsed “patient’s ideas, concerns, and expectations,” cost, or efficacy.
Referrals to nutrition services were endorsed more often than to obesity medicine specialists or bariatric surgery.
Asked about barriers to obesity treatment in their practices, “time constraints” was the most frequently endorsed, followed by “lack of training or knowledge,” “patient adherence and motivation,” and “limited resources.”
“What are the future directives? We feel we have the need to provide ongoing obesity management, education and assistance to primary care providers, including support for securing coverage for treatments,” Campos said.
He added that Boston Medical Center is now developing and implementing an embedded weight management program within primary care “to assist the front line of obesity care.”
Asked by this news organization whether he believes the rise of GLP-1 drugs will make a difference, Campos said “Definitely, I think with that momentum obesity medicine as a whole will gain more attention and hopefully more implementation in the curricula for medical and nursing schools, because in the end it requires a multidisciplinary approach.”
Campos and Clark had no disclosures.
A version of this article first appeared on Medscape.com.
SAN ANTONIO — Despite the prevalence of obesity in primary care, there appear to be major knowledge gaps among providers regarding obesity management, new research suggests.
Anonymous surveys of 96 primary care providers at a Boston, Massachusetts, safety-net hospital revealed that participants had limited understanding of criteria for prescribing antiobesity medications (AOM), and expressed discomfort in prescribing AOMs because of knowledge concerns, especially for non–glucagon-like peptide 1 (GLP-1) receptor agonists. One third reported that they didn’t prescribe AOMs, and rates of referral for bariatric surgery were also low.
The findings were presented at the Obesity Society’s annual Obesity Week meeting by Alejandro Campos, MD, a third-year resident in the section of internal medicine, Boston Medical Center, and the Department of Medicine, Boston University.
“I think it comes down to education. ... Not only training primary care physicians or residents about criteria and pathophysiology, but also stigma. Perceptions need to be addressed from the start of training in the healthcare field,” Campos told this news organization in an interview.
During his presentation, Campos noted this is the first such study in the setting of a safety-net hospital, which cares for lower-income people who experience disproportionate rates of obesity. But, “these findings are similar to ones observed from non–safety-net settings, which can indicate some potential transferability.”
Asked to comment, session moderator John D. Clark, MD, PhD, Chief Population Health Officer at Sharp Rees-Stealy Medical Group, San Diego, California, told this news organization that the findings didn’t surprise him. “I’d say that medical education around obesity has definitely improved, and training is improving but it’s not uniform. The treatment of obesity as a disease, especially with some of the newer medical treatments, is not standard of care and practiced widely.”
The study involved a standard-model Knowledge, Attitudes, and Practices questionnaire, distributed electronically for anonymous responses among both trained and in-training primary care providers. It contained a total of 43 items, 7 of them demographic, 11 on knowledge, 9 regarding attitudes, and 16 asking about practices.
The hospital is the largest safety-net hospital in New England, with a patient population that includes 58% enrolled in Medicaid, 32% Black/African American individuals, 24% identifying as Hispanic/Latino individuals, and 37% living below the poverty line.
The 96 responding providers (from a total 350 invited) all worked in either family medicine or internal medicine. The trained providers included both attending MDs and nurse practitioners, while those in-training were residents in one of those two specialties. Two thirds were women. The majority were aged 20-30 years (49.45%) or 31-40 years (27.47%).
Overall, 73.63% reported having received some type of obesity training. Just over half (52.08%) reported receiving that training during medical or nursing school, while 43.75% reported receiving it during residency.
When asked to choose from a list of conditions to pick which are considered weight-related comorbidities, between 80% and 90% choose type 2 diabetes, obstructive sleep apnea (OSA), hypertension, hyperlipidemia, nonalcoholic fatty liver disease, and coronary artery disease. Fewer, but still a majority, also listed osteoarthritis and gastroesophageal reflux disease. However, respondents were less likely to cite cancer, mood disorders, or chronic kidney disease as being related to obesity.
Asked to list benefits of a 10% body weight loss, most recognized reductions in OSA, glycemia, cardiovascular disease risk, osteoarthritis, and hepatic steatosis. But, only about half knew weight loss could also improve urinary incontinence.
Only 25% could correctly name both indications for AOMs. Just 27.1% knew that one was a body mass index (BMI) ≥ 27 with comorbidities, while 46.9% knew BMI ≥ 30 without comorbidities was an AOM indication. Only 9.4% were correct on both of those indications for bariatric surgery.
“Reassuringly,” Campos said, the majority either “disagreed” or “strongly disagreed” that “lack of will power” contributes to obesity. However, more than 20% agreed that “lack of exercise or physical activity” contributed.
Overall, 73% of the trained providers and 59% of those in training reported that they prescribe AOMs. Asked about their comfort level in prescribing specific types of AOMs, many more endorsed semaglutide and liraglutide than older medications such as bupropion/naltrexone and phentermine/topiramate.
Asked about factors that influence their comfort with prescribing AOMs, the top five factors selected, in order, were side-effect knowledge, insurance coverage, safety issues, and dosing knowledge. Fewer respondents endorsed “patient’s ideas, concerns, and expectations,” cost, or efficacy.
Referrals to nutrition services were endorsed more often than to obesity medicine specialists or bariatric surgery.
Asked about barriers to obesity treatment in their practices, “time constraints” was the most frequently endorsed, followed by “lack of training or knowledge,” “patient adherence and motivation,” and “limited resources.”
“What are the future directives? We feel we have the need to provide ongoing obesity management, education and assistance to primary care providers, including support for securing coverage for treatments,” Campos said.
He added that Boston Medical Center is now developing and implementing an embedded weight management program within primary care “to assist the front line of obesity care.”
Asked by this news organization whether he believes the rise of GLP-1 drugs will make a difference, Campos said “Definitely, I think with that momentum obesity medicine as a whole will gain more attention and hopefully more implementation in the curricula for medical and nursing schools, because in the end it requires a multidisciplinary approach.”
Campos and Clark had no disclosures.
A version of this article first appeared on Medscape.com.
SAN ANTONIO — Despite the prevalence of obesity in primary care, there appear to be major knowledge gaps among providers regarding obesity management, new research suggests.
Anonymous surveys of 96 primary care providers at a Boston, Massachusetts, safety-net hospital revealed that participants had limited understanding of criteria for prescribing antiobesity medications (AOM), and expressed discomfort in prescribing AOMs because of knowledge concerns, especially for non–glucagon-like peptide 1 (GLP-1) receptor agonists. One third reported that they didn’t prescribe AOMs, and rates of referral for bariatric surgery were also low.
The findings were presented at the Obesity Society’s annual Obesity Week meeting by Alejandro Campos, MD, a third-year resident in the section of internal medicine, Boston Medical Center, and the Department of Medicine, Boston University.
“I think it comes down to education. ... Not only training primary care physicians or residents about criteria and pathophysiology, but also stigma. Perceptions need to be addressed from the start of training in the healthcare field,” Campos told this news organization in an interview.
During his presentation, Campos noted this is the first such study in the setting of a safety-net hospital, which cares for lower-income people who experience disproportionate rates of obesity. But, “these findings are similar to ones observed from non–safety-net settings, which can indicate some potential transferability.”
Asked to comment, session moderator John D. Clark, MD, PhD, Chief Population Health Officer at Sharp Rees-Stealy Medical Group, San Diego, California, told this news organization that the findings didn’t surprise him. “I’d say that medical education around obesity has definitely improved, and training is improving but it’s not uniform. The treatment of obesity as a disease, especially with some of the newer medical treatments, is not standard of care and practiced widely.”
The study involved a standard-model Knowledge, Attitudes, and Practices questionnaire, distributed electronically for anonymous responses among both trained and in-training primary care providers. It contained a total of 43 items, 7 of them demographic, 11 on knowledge, 9 regarding attitudes, and 16 asking about practices.
The hospital is the largest safety-net hospital in New England, with a patient population that includes 58% enrolled in Medicaid, 32% Black/African American individuals, 24% identifying as Hispanic/Latino individuals, and 37% living below the poverty line.
The 96 responding providers (from a total 350 invited) all worked in either family medicine or internal medicine. The trained providers included both attending MDs and nurse practitioners, while those in-training were residents in one of those two specialties. Two thirds were women. The majority were aged 20-30 years (49.45%) or 31-40 years (27.47%).
Overall, 73.63% reported having received some type of obesity training. Just over half (52.08%) reported receiving that training during medical or nursing school, while 43.75% reported receiving it during residency.
When asked to choose from a list of conditions to pick which are considered weight-related comorbidities, between 80% and 90% choose type 2 diabetes, obstructive sleep apnea (OSA), hypertension, hyperlipidemia, nonalcoholic fatty liver disease, and coronary artery disease. Fewer, but still a majority, also listed osteoarthritis and gastroesophageal reflux disease. However, respondents were less likely to cite cancer, mood disorders, or chronic kidney disease as being related to obesity.
Asked to list benefits of a 10% body weight loss, most recognized reductions in OSA, glycemia, cardiovascular disease risk, osteoarthritis, and hepatic steatosis. But, only about half knew weight loss could also improve urinary incontinence.
Only 25% could correctly name both indications for AOMs. Just 27.1% knew that one was a body mass index (BMI) ≥ 27 with comorbidities, while 46.9% knew BMI ≥ 30 without comorbidities was an AOM indication. Only 9.4% were correct on both of those indications for bariatric surgery.
“Reassuringly,” Campos said, the majority either “disagreed” or “strongly disagreed” that “lack of will power” contributes to obesity. However, more than 20% agreed that “lack of exercise or physical activity” contributed.
Overall, 73% of the trained providers and 59% of those in training reported that they prescribe AOMs. Asked about their comfort level in prescribing specific types of AOMs, many more endorsed semaglutide and liraglutide than older medications such as bupropion/naltrexone and phentermine/topiramate.
Asked about factors that influence their comfort with prescribing AOMs, the top five factors selected, in order, were side-effect knowledge, insurance coverage, safety issues, and dosing knowledge. Fewer respondents endorsed “patient’s ideas, concerns, and expectations,” cost, or efficacy.
Referrals to nutrition services were endorsed more often than to obesity medicine specialists or bariatric surgery.
Asked about barriers to obesity treatment in their practices, “time constraints” was the most frequently endorsed, followed by “lack of training or knowledge,” “patient adherence and motivation,” and “limited resources.”
“What are the future directives? We feel we have the need to provide ongoing obesity management, education and assistance to primary care providers, including support for securing coverage for treatments,” Campos said.
He added that Boston Medical Center is now developing and implementing an embedded weight management program within primary care “to assist the front line of obesity care.”
Asked by this news organization whether he believes the rise of GLP-1 drugs will make a difference, Campos said “Definitely, I think with that momentum obesity medicine as a whole will gain more attention and hopefully more implementation in the curricula for medical and nursing schools, because in the end it requires a multidisciplinary approach.”
Campos and Clark had no disclosures.
A version of this article first appeared on Medscape.com.
FROM OBESITY WEEK 2024
AI-Assisted Colonoscopy Linked to Higher Rate of Benign Lesion Removal
PHILADELPHIA — according to a study presented at the annual meeting of the American College of Gastroenterology (ACG).
In particular, AIAC led to a statistically and clinically significant increase in the proportion of exams that detected lesions that after resection were all found to be benign, compared with unassisted colonoscopy.
“The potential implications include increased procedural risks, as well as costs, such as pathology costs and other healthcare expenditures, without any additional colorectal cancer prevention benefit,” said lead author Tessa Herman, MD, chief resident of internal medicine at the University of Minnesota, Minneapolis, and Minneapolis Veterans Affairs Health Care System.
In a previous implementation trial at the Minneapolis VA Medical Center, Herman and colleagues compared ADR between a group of patients undergoing AIAC and a historical cohort of patients who had non–AI-assisted colonoscopy.
In this subsequent study, the research team conducted an ad hoc analysis of data from the previous trial to determine the proportion of colonoscopies for screening, surveillance, and positive fecal immunochemical tests which detect lesions that after resection are all found to be benign. They excluded colonoscopies conducted for diagnostic indications or inflammatory bowel disease, as well as incomplete colonoscopies, and for those with inadequate bowel preparation.
Overall, they studied 441 non-AIAC colonoscopies (between November 2022 and April 2023) and 599 AIAC colonoscopies (between May 2023 and October 2023). The groups were balanced, and there were no significant differences in patient demographics, endoscopists, AI technology, procedure time, or average number of polyps detected.
In the non-AIAC cohort, 37 cases (8.4%) had polypectomies that revealed only benign lesions, as compared with 74 cases (12.4%) in the AIAC cohort. The most common resected lesions were benign colonic mucosa, lymphoid aggregates, and hyperplastic polyps.
Applied to the 15 million colonoscopies conducted in the United States per year, the findings indicate that full adoption of AIAC could result in about 600,000 more colonoscopies in which only benign, nonadenomatous lesions are removed, compared with traditional colonoscopy, Herman said.
More study of AIAC is needed, said Daniel Pambianco, MD, managing partner of GastroHealth-Charlottesville in Virginia and the 2023 ACG president. “This technology is in a fledging stage, and the more data we have, the more helpful it’ll be to know if we’re removing the right lesions at a better rate.”
“There’s a hope that assistance will improve detection, removal of polyps, and ultimately, colon cancer,” added Pambianco, who comoderated the session on colorectal cancer prevention.
Future longitudinal studies should monitor both ADR and benign lesion resection rates with AIAC, and modeling studies could determine the benefits and costs of the technology, Herman said. In addition, development of hybrid CADe and computer-aided diagnosis systems could mitigate concerns about excessive benign lesion resection with AI tools.
Clinicians already are able to find colon mucosa that are polypoid or lymphoid aggregates during colonoscopy without AI assistance, said the session’s comoderator, Sita Chokhavatia, MD, AGAF, a gastroenterologist with Valley Medical Group in Ridgewood, New Jersey.
“Instead, we need a tool that can help us to not remove these polyps that are not neoplastic,” she said. “With future developments, we may be able to take it to the next step where the algorithm tells us that it’s benign and not to touch it.”
The study was named an ACG Newsworthy Abstract. Herman, Pambianco, and Chokhavatia reported no relevant disclosures.
A version of this article first appeared on Medscape.com.
PHILADELPHIA — according to a study presented at the annual meeting of the American College of Gastroenterology (ACG).
In particular, AIAC led to a statistically and clinically significant increase in the proportion of exams that detected lesions that after resection were all found to be benign, compared with unassisted colonoscopy.
“The potential implications include increased procedural risks, as well as costs, such as pathology costs and other healthcare expenditures, without any additional colorectal cancer prevention benefit,” said lead author Tessa Herman, MD, chief resident of internal medicine at the University of Minnesota, Minneapolis, and Minneapolis Veterans Affairs Health Care System.
In a previous implementation trial at the Minneapolis VA Medical Center, Herman and colleagues compared ADR between a group of patients undergoing AIAC and a historical cohort of patients who had non–AI-assisted colonoscopy.
In this subsequent study, the research team conducted an ad hoc analysis of data from the previous trial to determine the proportion of colonoscopies for screening, surveillance, and positive fecal immunochemical tests which detect lesions that after resection are all found to be benign. They excluded colonoscopies conducted for diagnostic indications or inflammatory bowel disease, as well as incomplete colonoscopies, and for those with inadequate bowel preparation.
Overall, they studied 441 non-AIAC colonoscopies (between November 2022 and April 2023) and 599 AIAC colonoscopies (between May 2023 and October 2023). The groups were balanced, and there were no significant differences in patient demographics, endoscopists, AI technology, procedure time, or average number of polyps detected.
In the non-AIAC cohort, 37 cases (8.4%) had polypectomies that revealed only benign lesions, as compared with 74 cases (12.4%) in the AIAC cohort. The most common resected lesions were benign colonic mucosa, lymphoid aggregates, and hyperplastic polyps.
Applied to the 15 million colonoscopies conducted in the United States per year, the findings indicate that full adoption of AIAC could result in about 600,000 more colonoscopies in which only benign, nonadenomatous lesions are removed, compared with traditional colonoscopy, Herman said.
More study of AIAC is needed, said Daniel Pambianco, MD, managing partner of GastroHealth-Charlottesville in Virginia and the 2023 ACG president. “This technology is in a fledging stage, and the more data we have, the more helpful it’ll be to know if we’re removing the right lesions at a better rate.”
“There’s a hope that assistance will improve detection, removal of polyps, and ultimately, colon cancer,” added Pambianco, who comoderated the session on colorectal cancer prevention.
Future longitudinal studies should monitor both ADR and benign lesion resection rates with AIAC, and modeling studies could determine the benefits and costs of the technology, Herman said. In addition, development of hybrid CADe and computer-aided diagnosis systems could mitigate concerns about excessive benign lesion resection with AI tools.
Clinicians already are able to find colon mucosa that are polypoid or lymphoid aggregates during colonoscopy without AI assistance, said the session’s comoderator, Sita Chokhavatia, MD, AGAF, a gastroenterologist with Valley Medical Group in Ridgewood, New Jersey.
“Instead, we need a tool that can help us to not remove these polyps that are not neoplastic,” she said. “With future developments, we may be able to take it to the next step where the algorithm tells us that it’s benign and not to touch it.”
The study was named an ACG Newsworthy Abstract. Herman, Pambianco, and Chokhavatia reported no relevant disclosures.
A version of this article first appeared on Medscape.com.
PHILADELPHIA — according to a study presented at the annual meeting of the American College of Gastroenterology (ACG).
In particular, AIAC led to a statistically and clinically significant increase in the proportion of exams that detected lesions that after resection were all found to be benign, compared with unassisted colonoscopy.
“The potential implications include increased procedural risks, as well as costs, such as pathology costs and other healthcare expenditures, without any additional colorectal cancer prevention benefit,” said lead author Tessa Herman, MD, chief resident of internal medicine at the University of Minnesota, Minneapolis, and Minneapolis Veterans Affairs Health Care System.
In a previous implementation trial at the Minneapolis VA Medical Center, Herman and colleagues compared ADR between a group of patients undergoing AIAC and a historical cohort of patients who had non–AI-assisted colonoscopy.
In this subsequent study, the research team conducted an ad hoc analysis of data from the previous trial to determine the proportion of colonoscopies for screening, surveillance, and positive fecal immunochemical tests which detect lesions that after resection are all found to be benign. They excluded colonoscopies conducted for diagnostic indications or inflammatory bowel disease, as well as incomplete colonoscopies, and for those with inadequate bowel preparation.
Overall, they studied 441 non-AIAC colonoscopies (between November 2022 and April 2023) and 599 AIAC colonoscopies (between May 2023 and October 2023). The groups were balanced, and there were no significant differences in patient demographics, endoscopists, AI technology, procedure time, or average number of polyps detected.
In the non-AIAC cohort, 37 cases (8.4%) had polypectomies that revealed only benign lesions, as compared with 74 cases (12.4%) in the AIAC cohort. The most common resected lesions were benign colonic mucosa, lymphoid aggregates, and hyperplastic polyps.
Applied to the 15 million colonoscopies conducted in the United States per year, the findings indicate that full adoption of AIAC could result in about 600,000 more colonoscopies in which only benign, nonadenomatous lesions are removed, compared with traditional colonoscopy, Herman said.
More study of AIAC is needed, said Daniel Pambianco, MD, managing partner of GastroHealth-Charlottesville in Virginia and the 2023 ACG president. “This technology is in a fledging stage, and the more data we have, the more helpful it’ll be to know if we’re removing the right lesions at a better rate.”
“There’s a hope that assistance will improve detection, removal of polyps, and ultimately, colon cancer,” added Pambianco, who comoderated the session on colorectal cancer prevention.
Future longitudinal studies should monitor both ADR and benign lesion resection rates with AIAC, and modeling studies could determine the benefits and costs of the technology, Herman said. In addition, development of hybrid CADe and computer-aided diagnosis systems could mitigate concerns about excessive benign lesion resection with AI tools.
Clinicians already are able to find colon mucosa that are polypoid or lymphoid aggregates during colonoscopy without AI assistance, said the session’s comoderator, Sita Chokhavatia, MD, AGAF, a gastroenterologist with Valley Medical Group in Ridgewood, New Jersey.
“Instead, we need a tool that can help us to not remove these polyps that are not neoplastic,” she said. “With future developments, we may be able to take it to the next step where the algorithm tells us that it’s benign and not to touch it.”
The study was named an ACG Newsworthy Abstract. Herman, Pambianco, and Chokhavatia reported no relevant disclosures.
A version of this article first appeared on Medscape.com.
FROM ACG 2024
Short-Course Vasoconstrictors After EVL: Time for a New Standard of Care?
PHILADELPHIA — without raising the risk for rebleeding, if the initial ligation successfully controls bleeding.
“This approach would allow earlier discharge from the hospital and reduce the risk of adverse events, all without sacrificing treatment efficacy or compromising patient safety,” Sushrut Ingawale, MD, MBBS, Quinnipiac University School of Medicine, North Haven, and St. Vincent’s Medical Center, Bridgeport, both in Connecticut, said in a presentation at the annual meeting of the American College of Gastroenterology (ACG).
Ingawale called for a “re-evaluation of existing protocols, emphasizing the potential to update current protocols to reflect shorter, more personalized” duration of vasoconstrictor therapy in these patients.
Commenting on this research, Nancy Reau, MD, AGAF, of Rush University in Chicago, Illinois, said: “We should always question the standard of care.”
“Vasoconstrictors for 5 days is the standard of care, but this could lead to prolonged hospitalization in patients who are otherwise doing well after endoscopic intervention. Recognizing that a shorter course of vasoconstrictor treatment may have equal outcome is very important though it may not be appropriate for all patients, especially those at high risk for rebleeding,” said Reau.
Outdated Guidelines?
In his presentation, Ingawale noted that current guidelines that recommend continuing vasoconstrictors, like octreotide or terlipressin, for at least 3-5 days after EVL for acute variceal bleeding are based primarily on old studies in which sclerotherapy was the primary hemostatic method.
The study team assessed comparative outcomes based on the duration of vasoconstrictors after EVL for acute variceal bleeding in a systematic review and network meta-analysis of 11 randomized controlled trials.
The studies had a total of 816 patients who were grouped based on the duration vasoconstrictor therapy: 24 hours or less (group 1), 24-72 hours (group 2), and 72-120 hours (group 3).
There was no significant difference in the risk for rebleeding in group 1 (risk ratio [RR], 1.36; 95% CI, 0.48-3.52) and group 2 (RR, 1.34; 95% CI, 0.42-4.54) vs group 3.
“This finding was even consistent when we compared individual durations” of 0, 12, 24, 48, and 72 hours vs 120 hours, Ingawale said.
There was also no statistically significant difference in the 5-day mortality risk between group 1 (RR, 0.66; 95% CI, 0.09-2.52) and group 2 (RR, 1.08; 95% CI, 0.15-6.43) or the 30-day mortality risk between group 1 (RR, 1.18; 95% CI, 0.51-2.51) and group 2 (RR, 0.98; 95% CI, 0.36-2.52) vs group 3.
Rapidly Evolving Area
“Our network meta-analysis did not show any benefit of continuing vasoconstrictors after EVL,” the researchers wrote in their conference abstract. Despite historical precedent, shorter durations may be adequate, “potentially enabling earlier hospital discharge without compromising patient outcomes.”
Ingawale suggested future research should look to identify the subset of patients at a risk for failure to control bleeding who might benefit from the continuation of vasoconstrictors.
“Management of complications of portal hypertension are rapidly evolving and this study will add to the data that drives our guidelines. Seeing this data in a peer reviewed publication will add the necessary validity to impact a change in the treatment paradigm,” Reau said.
The study had no specific funding. Ingawale had no relevant financial relationships. Reau disclosed various relationships with AbbVie, Gilead, Arbutus, Intercept, and Salix.
A version of this article first appeared on Medscape.com.
PHILADELPHIA — without raising the risk for rebleeding, if the initial ligation successfully controls bleeding.
“This approach would allow earlier discharge from the hospital and reduce the risk of adverse events, all without sacrificing treatment efficacy or compromising patient safety,” Sushrut Ingawale, MD, MBBS, Quinnipiac University School of Medicine, North Haven, and St. Vincent’s Medical Center, Bridgeport, both in Connecticut, said in a presentation at the annual meeting of the American College of Gastroenterology (ACG).
Ingawale called for a “re-evaluation of existing protocols, emphasizing the potential to update current protocols to reflect shorter, more personalized” duration of vasoconstrictor therapy in these patients.
Commenting on this research, Nancy Reau, MD, AGAF, of Rush University in Chicago, Illinois, said: “We should always question the standard of care.”
“Vasoconstrictors for 5 days is the standard of care, but this could lead to prolonged hospitalization in patients who are otherwise doing well after endoscopic intervention. Recognizing that a shorter course of vasoconstrictor treatment may have equal outcome is very important though it may not be appropriate for all patients, especially those at high risk for rebleeding,” said Reau.
Outdated Guidelines?
In his presentation, Ingawale noted that current guidelines that recommend continuing vasoconstrictors, like octreotide or terlipressin, for at least 3-5 days after EVL for acute variceal bleeding are based primarily on old studies in which sclerotherapy was the primary hemostatic method.
The study team assessed comparative outcomes based on the duration of vasoconstrictors after EVL for acute variceal bleeding in a systematic review and network meta-analysis of 11 randomized controlled trials.
The studies had a total of 816 patients who were grouped based on the duration vasoconstrictor therapy: 24 hours or less (group 1), 24-72 hours (group 2), and 72-120 hours (group 3).
There was no significant difference in the risk for rebleeding in group 1 (risk ratio [RR], 1.36; 95% CI, 0.48-3.52) and group 2 (RR, 1.34; 95% CI, 0.42-4.54) vs group 3.
“This finding was even consistent when we compared individual durations” of 0, 12, 24, 48, and 72 hours vs 120 hours, Ingawale said.
There was also no statistically significant difference in the 5-day mortality risk between group 1 (RR, 0.66; 95% CI, 0.09-2.52) and group 2 (RR, 1.08; 95% CI, 0.15-6.43) or the 30-day mortality risk between group 1 (RR, 1.18; 95% CI, 0.51-2.51) and group 2 (RR, 0.98; 95% CI, 0.36-2.52) vs group 3.
Rapidly Evolving Area
“Our network meta-analysis did not show any benefit of continuing vasoconstrictors after EVL,” the researchers wrote in their conference abstract. Despite historical precedent, shorter durations may be adequate, “potentially enabling earlier hospital discharge without compromising patient outcomes.”
Ingawale suggested future research should look to identify the subset of patients at a risk for failure to control bleeding who might benefit from the continuation of vasoconstrictors.
“Management of complications of portal hypertension are rapidly evolving and this study will add to the data that drives our guidelines. Seeing this data in a peer reviewed publication will add the necessary validity to impact a change in the treatment paradigm,” Reau said.
The study had no specific funding. Ingawale had no relevant financial relationships. Reau disclosed various relationships with AbbVie, Gilead, Arbutus, Intercept, and Salix.
A version of this article first appeared on Medscape.com.
PHILADELPHIA — without raising the risk for rebleeding, if the initial ligation successfully controls bleeding.
“This approach would allow earlier discharge from the hospital and reduce the risk of adverse events, all without sacrificing treatment efficacy or compromising patient safety,” Sushrut Ingawale, MD, MBBS, Quinnipiac University School of Medicine, North Haven, and St. Vincent’s Medical Center, Bridgeport, both in Connecticut, said in a presentation at the annual meeting of the American College of Gastroenterology (ACG).
Ingawale called for a “re-evaluation of existing protocols, emphasizing the potential to update current protocols to reflect shorter, more personalized” duration of vasoconstrictor therapy in these patients.
Commenting on this research, Nancy Reau, MD, AGAF, of Rush University in Chicago, Illinois, said: “We should always question the standard of care.”
“Vasoconstrictors for 5 days is the standard of care, but this could lead to prolonged hospitalization in patients who are otherwise doing well after endoscopic intervention. Recognizing that a shorter course of vasoconstrictor treatment may have equal outcome is very important though it may not be appropriate for all patients, especially those at high risk for rebleeding,” said Reau.
Outdated Guidelines?
In his presentation, Ingawale noted that current guidelines that recommend continuing vasoconstrictors, like octreotide or terlipressin, for at least 3-5 days after EVL for acute variceal bleeding are based primarily on old studies in which sclerotherapy was the primary hemostatic method.
The study team assessed comparative outcomes based on the duration of vasoconstrictors after EVL for acute variceal bleeding in a systematic review and network meta-analysis of 11 randomized controlled trials.
The studies had a total of 816 patients who were grouped based on the duration vasoconstrictor therapy: 24 hours or less (group 1), 24-72 hours (group 2), and 72-120 hours (group 3).
There was no significant difference in the risk for rebleeding in group 1 (risk ratio [RR], 1.36; 95% CI, 0.48-3.52) and group 2 (RR, 1.34; 95% CI, 0.42-4.54) vs group 3.
“This finding was even consistent when we compared individual durations” of 0, 12, 24, 48, and 72 hours vs 120 hours, Ingawale said.
There was also no statistically significant difference in the 5-day mortality risk between group 1 (RR, 0.66; 95% CI, 0.09-2.52) and group 2 (RR, 1.08; 95% CI, 0.15-6.43) or the 30-day mortality risk between group 1 (RR, 1.18; 95% CI, 0.51-2.51) and group 2 (RR, 0.98; 95% CI, 0.36-2.52) vs group 3.
Rapidly Evolving Area
“Our network meta-analysis did not show any benefit of continuing vasoconstrictors after EVL,” the researchers wrote in their conference abstract. Despite historical precedent, shorter durations may be adequate, “potentially enabling earlier hospital discharge without compromising patient outcomes.”
Ingawale suggested future research should look to identify the subset of patients at a risk for failure to control bleeding who might benefit from the continuation of vasoconstrictors.
“Management of complications of portal hypertension are rapidly evolving and this study will add to the data that drives our guidelines. Seeing this data in a peer reviewed publication will add the necessary validity to impact a change in the treatment paradigm,” Reau said.
The study had no specific funding. Ingawale had no relevant financial relationships. Reau disclosed various relationships with AbbVie, Gilead, Arbutus, Intercept, and Salix.
A version of this article first appeared on Medscape.com.
FROM ACG 2024
Myasthenia Gravis: Where Does Traditional Therapy Fit In?
SAVANNAH, GEORGIA —
In a debate at American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024, a pair of neurologists who specialize in neuromuscular disorders laid out opposing evidence for each approach.
On one hand, Benjamin Claytor, MD, of Cleveland Clinic, Cleveland, argued that “traditional therapy is very effective for the majority of myasthenia gravis patients,” and he said it should be considered first-line.
But Amanda C. Guidon, MD, MPH, of Massachusetts General Hospital and Harvard Medical School, both in Boston, responded that “the immunosuppression of traditional therapies is too broad: The time to benefit is too long, the burden of side effects is too high, and the cancer risk is too elevated.”
Traditional Therapy: Affordable, Tolerable, and Safe?
Claytor said ideal myasthenia gravis therapies are effective, tolerable, and safe. They’re also affordable, convenient (such as a pill), lead to sustained remission, and can have dosages reduced.
Only traditional therapies — corticosteroids, azathioprine, mycophenolate, and rituximab — meet those last three criteria, he said. Newer therapies, he said, do not.
Claytor highlighted a 2023 Duke University study that tracked 367 patients with MG who were treated with traditional therapies after the year 2000. Of those, 72% reached the treatment goal of minimal manifestations in a median of less than 2 years.
In addition, Claytor noted that the percentage of patients with myasthenia gravis who reach minimal symptom expression ranges from 45% (6 months) to 60% or more (2 years), while studies suggest that newer treatments such as eculizumab (Soliris), efgartigimod (Vyvgart), rozanolixizumab (Rystiggo), and zilucoplan (Zilbrysq) haven’t reached those levels.
As for specific traditional therapies, Claytor said the corticosteroid prednisone is “extremely affordable,” effective, and takes fewer than 2 weeks to work. All patients with myasthenia gravis can take it, he said, and at least 75% of those with mild/moderate disease respond to low doses.
Nonsteroidal Agents, Immune Globulin, Rituximab
He acknowledged side effects from corticosteroids but said doses can be tapered once severity improves. Calcium and vitamin D can be helpful to support bone health, he added.
As for nonsteroidal immunosuppressive treatments, he said they’re easy to administer, increase the likelihood of reaching minimal manifestation status, can be effective at lower doses, and may allow patients to discontinue steroids.
Two other traditional therapies, immune globulin and plasmapheresis, can be appropriate in crisis or impending crisis situations, he said, or as an add-on therapy if steroids and nonsteroidal immunosuppressive therapies don’t work.
What about rituximab? “We’re learning that patients with new-onset disease and younger patients seem to respond better,” Claytor said. While rituximab is expensive, it’s “not even in the same realm” as newer agents if only a dose or two are given, he said.
Steroids Are Ideal in MG? Not So Fast
In her response, Guidon noted that she was assigned to offer a counter-perspective in her presentation, and “personal opinions are not being represented here fully.” She then listed the weaknesses of traditional therapy in myasthenia gravis.
For one thing, she said the drugs don’t work well. She highlighted a 2019 registry study that found “many myasthenia gravis patients remain negatively impacted despite treatment.”
In addition, “we can’t predict who will respond to which therapy. ... We start drugs and don’t know if we’ll have benefit from 6 months up to 18 months. We also can’t determine minimally effective dose a priori. Some patients require higher doses, and some subtherapeutic doses are actually therapeutic for our patients.”
Broad immunosuppression, she added, boosts the risk for serious infections. “We’ve all heard from our patients that the side effects can be worse than the myasthenia, and next we’re going to talk about the role of corticosteroids in myasthenia.”
As for corticosteroids in particular, “they’re really the best treatment and also the worst treatment.” Efficacy and side effects battle for supremacy in patients, she said, “and you don’t know which is going to win out.”
Kicking Traditional Therapy to the Curb
There are many possible side effects from steroids, she said, including steroid-induced diabetes, which is “profound.” Some patients never recover from it.
On top of all these risks, she said, 20%-30% of patients are resistant to steroids.
As for other treatments, immune globulin and plasmapheresis “aren’t really benign,” Guidon said. They come with potentially serious side effects of their own, as do nonsteroidal immunosuppressive treatments.
Guidon said better treatments are needed to minimize the risks from traditional therapies. “We need targeted therapies that drive disease into remission, can be tapered, are delivered orally or with infrequent self-injections, and don’t require frequent lab monitoring.”
In addition, ideal treatments should “have a good safety data in pregnancy and for breastfeeding and have a favorable side effect profile with no significant long-term cancer risks.”
Claytor had no disclosures. Guidon disclosed consulting/medical advisory board (Alexion Pharmaceuticals, argenx, Regeneron, and UCB), publishing royalties (Oakstone), and other research support (Myasthenia Gravis Foundation of America, Myasthenia Gravis Rare Disease Network, National Institutes of Health, and National Institute of Neurological Disorders and Stroke/BioSensics).
A version of this article appeared on Medscape.com.
SAVANNAH, GEORGIA —
In a debate at American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024, a pair of neurologists who specialize in neuromuscular disorders laid out opposing evidence for each approach.
On one hand, Benjamin Claytor, MD, of Cleveland Clinic, Cleveland, argued that “traditional therapy is very effective for the majority of myasthenia gravis patients,” and he said it should be considered first-line.
But Amanda C. Guidon, MD, MPH, of Massachusetts General Hospital and Harvard Medical School, both in Boston, responded that “the immunosuppression of traditional therapies is too broad: The time to benefit is too long, the burden of side effects is too high, and the cancer risk is too elevated.”
Traditional Therapy: Affordable, Tolerable, and Safe?
Claytor said ideal myasthenia gravis therapies are effective, tolerable, and safe. They’re also affordable, convenient (such as a pill), lead to sustained remission, and can have dosages reduced.
Only traditional therapies — corticosteroids, azathioprine, mycophenolate, and rituximab — meet those last three criteria, he said. Newer therapies, he said, do not.
Claytor highlighted a 2023 Duke University study that tracked 367 patients with MG who were treated with traditional therapies after the year 2000. Of those, 72% reached the treatment goal of minimal manifestations in a median of less than 2 years.
In addition, Claytor noted that the percentage of patients with myasthenia gravis who reach minimal symptom expression ranges from 45% (6 months) to 60% or more (2 years), while studies suggest that newer treatments such as eculizumab (Soliris), efgartigimod (Vyvgart), rozanolixizumab (Rystiggo), and zilucoplan (Zilbrysq) haven’t reached those levels.
As for specific traditional therapies, Claytor said the corticosteroid prednisone is “extremely affordable,” effective, and takes fewer than 2 weeks to work. All patients with myasthenia gravis can take it, he said, and at least 75% of those with mild/moderate disease respond to low doses.
Nonsteroidal Agents, Immune Globulin, Rituximab
He acknowledged side effects from corticosteroids but said doses can be tapered once severity improves. Calcium and vitamin D can be helpful to support bone health, he added.
As for nonsteroidal immunosuppressive treatments, he said they’re easy to administer, increase the likelihood of reaching minimal manifestation status, can be effective at lower doses, and may allow patients to discontinue steroids.
Two other traditional therapies, immune globulin and plasmapheresis, can be appropriate in crisis or impending crisis situations, he said, or as an add-on therapy if steroids and nonsteroidal immunosuppressive therapies don’t work.
What about rituximab? “We’re learning that patients with new-onset disease and younger patients seem to respond better,” Claytor said. While rituximab is expensive, it’s “not even in the same realm” as newer agents if only a dose or two are given, he said.
Steroids Are Ideal in MG? Not So Fast
In her response, Guidon noted that she was assigned to offer a counter-perspective in her presentation, and “personal opinions are not being represented here fully.” She then listed the weaknesses of traditional therapy in myasthenia gravis.
For one thing, she said the drugs don’t work well. She highlighted a 2019 registry study that found “many myasthenia gravis patients remain negatively impacted despite treatment.”
In addition, “we can’t predict who will respond to which therapy. ... We start drugs and don’t know if we’ll have benefit from 6 months up to 18 months. We also can’t determine minimally effective dose a priori. Some patients require higher doses, and some subtherapeutic doses are actually therapeutic for our patients.”
Broad immunosuppression, she added, boosts the risk for serious infections. “We’ve all heard from our patients that the side effects can be worse than the myasthenia, and next we’re going to talk about the role of corticosteroids in myasthenia.”
As for corticosteroids in particular, “they’re really the best treatment and also the worst treatment.” Efficacy and side effects battle for supremacy in patients, she said, “and you don’t know which is going to win out.”
Kicking Traditional Therapy to the Curb
There are many possible side effects from steroids, she said, including steroid-induced diabetes, which is “profound.” Some patients never recover from it.
On top of all these risks, she said, 20%-30% of patients are resistant to steroids.
As for other treatments, immune globulin and plasmapheresis “aren’t really benign,” Guidon said. They come with potentially serious side effects of their own, as do nonsteroidal immunosuppressive treatments.
Guidon said better treatments are needed to minimize the risks from traditional therapies. “We need targeted therapies that drive disease into remission, can be tapered, are delivered orally or with infrequent self-injections, and don’t require frequent lab monitoring.”
In addition, ideal treatments should “have a good safety data in pregnancy and for breastfeeding and have a favorable side effect profile with no significant long-term cancer risks.”
Claytor had no disclosures. Guidon disclosed consulting/medical advisory board (Alexion Pharmaceuticals, argenx, Regeneron, and UCB), publishing royalties (Oakstone), and other research support (Myasthenia Gravis Foundation of America, Myasthenia Gravis Rare Disease Network, National Institutes of Health, and National Institute of Neurological Disorders and Stroke/BioSensics).
A version of this article appeared on Medscape.com.
SAVANNAH, GEORGIA —
In a debate at American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024, a pair of neurologists who specialize in neuromuscular disorders laid out opposing evidence for each approach.
On one hand, Benjamin Claytor, MD, of Cleveland Clinic, Cleveland, argued that “traditional therapy is very effective for the majority of myasthenia gravis patients,” and he said it should be considered first-line.
But Amanda C. Guidon, MD, MPH, of Massachusetts General Hospital and Harvard Medical School, both in Boston, responded that “the immunosuppression of traditional therapies is too broad: The time to benefit is too long, the burden of side effects is too high, and the cancer risk is too elevated.”
Traditional Therapy: Affordable, Tolerable, and Safe?
Claytor said ideal myasthenia gravis therapies are effective, tolerable, and safe. They’re also affordable, convenient (such as a pill), lead to sustained remission, and can have dosages reduced.
Only traditional therapies — corticosteroids, azathioprine, mycophenolate, and rituximab — meet those last three criteria, he said. Newer therapies, he said, do not.
Claytor highlighted a 2023 Duke University study that tracked 367 patients with MG who were treated with traditional therapies after the year 2000. Of those, 72% reached the treatment goal of minimal manifestations in a median of less than 2 years.
In addition, Claytor noted that the percentage of patients with myasthenia gravis who reach minimal symptom expression ranges from 45% (6 months) to 60% or more (2 years), while studies suggest that newer treatments such as eculizumab (Soliris), efgartigimod (Vyvgart), rozanolixizumab (Rystiggo), and zilucoplan (Zilbrysq) haven’t reached those levels.
As for specific traditional therapies, Claytor said the corticosteroid prednisone is “extremely affordable,” effective, and takes fewer than 2 weeks to work. All patients with myasthenia gravis can take it, he said, and at least 75% of those with mild/moderate disease respond to low doses.
Nonsteroidal Agents, Immune Globulin, Rituximab
He acknowledged side effects from corticosteroids but said doses can be tapered once severity improves. Calcium and vitamin D can be helpful to support bone health, he added.
As for nonsteroidal immunosuppressive treatments, he said they’re easy to administer, increase the likelihood of reaching minimal manifestation status, can be effective at lower doses, and may allow patients to discontinue steroids.
Two other traditional therapies, immune globulin and plasmapheresis, can be appropriate in crisis or impending crisis situations, he said, or as an add-on therapy if steroids and nonsteroidal immunosuppressive therapies don’t work.
What about rituximab? “We’re learning that patients with new-onset disease and younger patients seem to respond better,” Claytor said. While rituximab is expensive, it’s “not even in the same realm” as newer agents if only a dose or two are given, he said.
Steroids Are Ideal in MG? Not So Fast
In her response, Guidon noted that she was assigned to offer a counter-perspective in her presentation, and “personal opinions are not being represented here fully.” She then listed the weaknesses of traditional therapy in myasthenia gravis.
For one thing, she said the drugs don’t work well. She highlighted a 2019 registry study that found “many myasthenia gravis patients remain negatively impacted despite treatment.”
In addition, “we can’t predict who will respond to which therapy. ... We start drugs and don’t know if we’ll have benefit from 6 months up to 18 months. We also can’t determine minimally effective dose a priori. Some patients require higher doses, and some subtherapeutic doses are actually therapeutic for our patients.”
Broad immunosuppression, she added, boosts the risk for serious infections. “We’ve all heard from our patients that the side effects can be worse than the myasthenia, and next we’re going to talk about the role of corticosteroids in myasthenia.”
As for corticosteroids in particular, “they’re really the best treatment and also the worst treatment.” Efficacy and side effects battle for supremacy in patients, she said, “and you don’t know which is going to win out.”
Kicking Traditional Therapy to the Curb
There are many possible side effects from steroids, she said, including steroid-induced diabetes, which is “profound.” Some patients never recover from it.
On top of all these risks, she said, 20%-30% of patients are resistant to steroids.
As for other treatments, immune globulin and plasmapheresis “aren’t really benign,” Guidon said. They come with potentially serious side effects of their own, as do nonsteroidal immunosuppressive treatments.
Guidon said better treatments are needed to minimize the risks from traditional therapies. “We need targeted therapies that drive disease into remission, can be tapered, are delivered orally or with infrequent self-injections, and don’t require frequent lab monitoring.”
In addition, ideal treatments should “have a good safety data in pregnancy and for breastfeeding and have a favorable side effect profile with no significant long-term cancer risks.”
Claytor had no disclosures. Guidon disclosed consulting/medical advisory board (Alexion Pharmaceuticals, argenx, Regeneron, and UCB), publishing royalties (Oakstone), and other research support (Myasthenia Gravis Foundation of America, Myasthenia Gravis Rare Disease Network, National Institutes of Health, and National Institute of Neurological Disorders and Stroke/BioSensics).
A version of this article appeared on Medscape.com.
FROM AANEM 2024
New Drug Options Abound for Duchenne Muscular Dystrophy
SAVANNAH, GEORGIA — When Ann & Robert H. Lurie Children’s Hospital of Chicago pediatric neurologist Nancy L. Kuntz, MD, was a fellow about 45 years ago, there were few more devastating diagnoses than Duchenne muscular dystrophy (DMD).
“The rule of thumb was that they would stop walking by age 10 and probably die around age 20, and there was not much we could do,” Kuntz told colleagues at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024.
Now, “In the last 8 years, we’ve seen eight different therapies that are FDA-approved specifically for Duchenne, and many more are in the pipeline,” said session moderator Kathryn Mosher, MD, a pediatric physical medicine and rehabilitation physician at Akron Children’s Hospital, Akron, Ohio.
This is both good news and a new challenge for clinicians: Which of these treatments are best for which patients? Kuntz said the traditional therapy of corticosteroids is still crucial. However, “there are still families begging to not use steroids, or refusing to use steroids, just not filling the prescriptions,” she said.
Beware of Parents Who Reject Steroids
The failure to use steroids “breaks your heart” because data show their impact on “really important functions like walking and being able to get up from the ground,” she said. “You can add months and years to life with this treatment.”
However, “while we have shown that using corticosteroids makes a difference, I don’t think that we’ve really worked out the best age at which to start the steroids, or the dosing schedule, or even the type of steroids,” she cautioned.
In an accompanying presentation about therapy for DMD, pediatric neurologist Craig M. Zaidman, MD, of Washington University in St. Louis, Missouri, cautioned that “daily steroids make a big impact on your growth and particularly on your height.”
In particular, the corticosteroid deflazacort has been linked to more cataracts than prednisone and less weight gain and height growth. “They really don’t grow, they don’t get taller, and they also don’t gain weight. They look like little boys when they’re 13 years old.”
Deflazacort or Vamorolone?
Vamorolone (Agamree) is a cheaper corticosteroid alternative to deflazacort (Emflaza), and a 2024 study showed no difference in functional outcomes over 48 weeks, he said. Also, daily vamorolone does a better job of preserving height growth than daily prednisone, he said, and he’s seen less risk for vertebral fractures.
Where do newer drugs fit in? One crucial thing to know about the new generation of targeted therapies is that they’re often mutation-dependent, Kuntz said. They may only work in patients with certain mutations, or mutations may lead to more side effects.
“You should have the exact mutation of your patient, and then you can look and see what they’re eligible for,” she said.
$700,000 a Year for Givinostat
Zaidman highlighted the newly approved givinostat (Duvyzat), a histone deacetylase inhibitor approved for boys 6 years or older. The cost is $700,000 a year, he said, and it’s been linked to less decline in four-stair climb per a double-blind, placebo-controlled, phase 3 trial.
The drug can cause side effects such as reducing platelets, boosting triglycerides, and inducing gastrointestinal problems. “When you drop the dose, these problems go away,” he said.
Does givinostat work? While trial data are challenging to interpret, they do suggest that patients “will lose skill, but they might not lose two or three skills they otherwise would have,” Zaidman said. “To me, that’s quite compelling.”
As for exon-skipping therapies, another new-generation option for DMD, he noted that “these drugs are on the market based on their accelerated approval. We will never have the perfect phase 3, randomized, controlled, long-term trial for these. It’s just not going to come. This is what we get.”
Mosher disclosed the advisory board (Sarepta Therapeutics, Pfizer, Reata Pharmaceuticals, and PTC). Kuntz disclosed advisory board (Astellas Pharma, Inc., argenx, Catalyst, Entrada Therapeutics, Genentech, and Novartis), exchange expert on-demand program (Sarepta Therapeutics), speaker (Genentech, Sarepta Therapeutics, and Solid), and research funding (Astellas Pharma, Inc., argenx, Biogen, Catalyst, Genentech, Novartis, and Sarepta Therapeutics). Zaidman disclosed speaking/advisor/consulting (Sarepta Therapeutics and Optum) and research funding (Novartis and Biogen).
A version of this article appeared on Medscape.com.
SAVANNAH, GEORGIA — When Ann & Robert H. Lurie Children’s Hospital of Chicago pediatric neurologist Nancy L. Kuntz, MD, was a fellow about 45 years ago, there were few more devastating diagnoses than Duchenne muscular dystrophy (DMD).
“The rule of thumb was that they would stop walking by age 10 and probably die around age 20, and there was not much we could do,” Kuntz told colleagues at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024.
Now, “In the last 8 years, we’ve seen eight different therapies that are FDA-approved specifically for Duchenne, and many more are in the pipeline,” said session moderator Kathryn Mosher, MD, a pediatric physical medicine and rehabilitation physician at Akron Children’s Hospital, Akron, Ohio.
This is both good news and a new challenge for clinicians: Which of these treatments are best for which patients? Kuntz said the traditional therapy of corticosteroids is still crucial. However, “there are still families begging to not use steroids, or refusing to use steroids, just not filling the prescriptions,” she said.
Beware of Parents Who Reject Steroids
The failure to use steroids “breaks your heart” because data show their impact on “really important functions like walking and being able to get up from the ground,” she said. “You can add months and years to life with this treatment.”
However, “while we have shown that using corticosteroids makes a difference, I don’t think that we’ve really worked out the best age at which to start the steroids, or the dosing schedule, or even the type of steroids,” she cautioned.
In an accompanying presentation about therapy for DMD, pediatric neurologist Craig M. Zaidman, MD, of Washington University in St. Louis, Missouri, cautioned that “daily steroids make a big impact on your growth and particularly on your height.”
In particular, the corticosteroid deflazacort has been linked to more cataracts than prednisone and less weight gain and height growth. “They really don’t grow, they don’t get taller, and they also don’t gain weight. They look like little boys when they’re 13 years old.”
Deflazacort or Vamorolone?
Vamorolone (Agamree) is a cheaper corticosteroid alternative to deflazacort (Emflaza), and a 2024 study showed no difference in functional outcomes over 48 weeks, he said. Also, daily vamorolone does a better job of preserving height growth than daily prednisone, he said, and he’s seen less risk for vertebral fractures.
Where do newer drugs fit in? One crucial thing to know about the new generation of targeted therapies is that they’re often mutation-dependent, Kuntz said. They may only work in patients with certain mutations, or mutations may lead to more side effects.
“You should have the exact mutation of your patient, and then you can look and see what they’re eligible for,” she said.
$700,000 a Year for Givinostat
Zaidman highlighted the newly approved givinostat (Duvyzat), a histone deacetylase inhibitor approved for boys 6 years or older. The cost is $700,000 a year, he said, and it’s been linked to less decline in four-stair climb per a double-blind, placebo-controlled, phase 3 trial.
The drug can cause side effects such as reducing platelets, boosting triglycerides, and inducing gastrointestinal problems. “When you drop the dose, these problems go away,” he said.
Does givinostat work? While trial data are challenging to interpret, they do suggest that patients “will lose skill, but they might not lose two or three skills they otherwise would have,” Zaidman said. “To me, that’s quite compelling.”
As for exon-skipping therapies, another new-generation option for DMD, he noted that “these drugs are on the market based on their accelerated approval. We will never have the perfect phase 3, randomized, controlled, long-term trial for these. It’s just not going to come. This is what we get.”
Mosher disclosed the advisory board (Sarepta Therapeutics, Pfizer, Reata Pharmaceuticals, and PTC). Kuntz disclosed advisory board (Astellas Pharma, Inc., argenx, Catalyst, Entrada Therapeutics, Genentech, and Novartis), exchange expert on-demand program (Sarepta Therapeutics), speaker (Genentech, Sarepta Therapeutics, and Solid), and research funding (Astellas Pharma, Inc., argenx, Biogen, Catalyst, Genentech, Novartis, and Sarepta Therapeutics). Zaidman disclosed speaking/advisor/consulting (Sarepta Therapeutics and Optum) and research funding (Novartis and Biogen).
A version of this article appeared on Medscape.com.
SAVANNAH, GEORGIA — When Ann & Robert H. Lurie Children’s Hospital of Chicago pediatric neurologist Nancy L. Kuntz, MD, was a fellow about 45 years ago, there were few more devastating diagnoses than Duchenne muscular dystrophy (DMD).
“The rule of thumb was that they would stop walking by age 10 and probably die around age 20, and there was not much we could do,” Kuntz told colleagues at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) 2024.
Now, “In the last 8 years, we’ve seen eight different therapies that are FDA-approved specifically for Duchenne, and many more are in the pipeline,” said session moderator Kathryn Mosher, MD, a pediatric physical medicine and rehabilitation physician at Akron Children’s Hospital, Akron, Ohio.
This is both good news and a new challenge for clinicians: Which of these treatments are best for which patients? Kuntz said the traditional therapy of corticosteroids is still crucial. However, “there are still families begging to not use steroids, or refusing to use steroids, just not filling the prescriptions,” she said.
Beware of Parents Who Reject Steroids
The failure to use steroids “breaks your heart” because data show their impact on “really important functions like walking and being able to get up from the ground,” she said. “You can add months and years to life with this treatment.”
However, “while we have shown that using corticosteroids makes a difference, I don’t think that we’ve really worked out the best age at which to start the steroids, or the dosing schedule, or even the type of steroids,” she cautioned.
In an accompanying presentation about therapy for DMD, pediatric neurologist Craig M. Zaidman, MD, of Washington University in St. Louis, Missouri, cautioned that “daily steroids make a big impact on your growth and particularly on your height.”
In particular, the corticosteroid deflazacort has been linked to more cataracts than prednisone and less weight gain and height growth. “They really don’t grow, they don’t get taller, and they also don’t gain weight. They look like little boys when they’re 13 years old.”
Deflazacort or Vamorolone?
Vamorolone (Agamree) is a cheaper corticosteroid alternative to deflazacort (Emflaza), and a 2024 study showed no difference in functional outcomes over 48 weeks, he said. Also, daily vamorolone does a better job of preserving height growth than daily prednisone, he said, and he’s seen less risk for vertebral fractures.
Where do newer drugs fit in? One crucial thing to know about the new generation of targeted therapies is that they’re often mutation-dependent, Kuntz said. They may only work in patients with certain mutations, or mutations may lead to more side effects.
“You should have the exact mutation of your patient, and then you can look and see what they’re eligible for,” she said.
$700,000 a Year for Givinostat
Zaidman highlighted the newly approved givinostat (Duvyzat), a histone deacetylase inhibitor approved for boys 6 years or older. The cost is $700,000 a year, he said, and it’s been linked to less decline in four-stair climb per a double-blind, placebo-controlled, phase 3 trial.
The drug can cause side effects such as reducing platelets, boosting triglycerides, and inducing gastrointestinal problems. “When you drop the dose, these problems go away,” he said.
Does givinostat work? While trial data are challenging to interpret, they do suggest that patients “will lose skill, but they might not lose two or three skills they otherwise would have,” Zaidman said. “To me, that’s quite compelling.”
As for exon-skipping therapies, another new-generation option for DMD, he noted that “these drugs are on the market based on their accelerated approval. We will never have the perfect phase 3, randomized, controlled, long-term trial for these. It’s just not going to come. This is what we get.”
Mosher disclosed the advisory board (Sarepta Therapeutics, Pfizer, Reata Pharmaceuticals, and PTC). Kuntz disclosed advisory board (Astellas Pharma, Inc., argenx, Catalyst, Entrada Therapeutics, Genentech, and Novartis), exchange expert on-demand program (Sarepta Therapeutics), speaker (Genentech, Sarepta Therapeutics, and Solid), and research funding (Astellas Pharma, Inc., argenx, Biogen, Catalyst, Genentech, Novartis, and Sarepta Therapeutics). Zaidman disclosed speaking/advisor/consulting (Sarepta Therapeutics and Optum) and research funding (Novartis and Biogen).
A version of this article appeared on Medscape.com.
FROM AANEM 2024
Study Finds Link to Increased Risk for Bulimia, Binge Eating and HS
“Clinicians should actively screen for eating disorders,” particularly bulimia nervosa and binge eating disorder, in patients with HS,” lead study author Christopher Guirguis, DMD, a student at Georgetown University School of Medicine, Washington, DC, told this news organization in advance of the annual Symposium on Hidradenitis Suppurative Advances, where the study was presented during an oral abstract session. “The significant psychological burden in these patients requires a holistic approach that integrates both dermatologic and psychosocial care. Addressing their mental health needs is essential for improving overall patient outcomes and quality of life,” he added.
In collaboration with fellow Georgetown medical student and first author Lauren Chin and Mikael Horissian, MD, a dermatologist and director of the HS Clinic at Gesinger Health System, Danville, Pennsylvania, Guirguis drew from the National Institutes of Health’s All of Us Research Program to identify 1653 individuals with a diagnosis of HS and a control group of 8265 individuals without a diagnosis of HS. They used the Observational Medical Outcomes Partnership to identify anorexia nervosa, bulimia nervosa, body dysmorphic disorder, binge eating disorder, and eating disorder, unspecified. Obsessive-compulsive disorder (OCD) was also included because of its association with bulimia. They used statistical models to compare cohorts and comorbidities. “What makes this work unique is its focus on the link between HS and eating disorders, a relationship previously underexplored,” he said.
The mean age of the overall study cohort was 46.8 years, and 78.6% were female. Univariate analysis revealed that, compared with controls, individuals in the HS cohort showed significantly increased diagnoses of bulimia, binge eating disorder, OCD, and eating disorder, unspecified, by 2.6, 5.48, 2.50, and 2.43 times, respectively (P < .05 for all associations). After adjusting for age, race, sex, and ethnicity, the researchers observed that patients with HS were 4.46 times as likely to have a diagnosis of binge eating disorder and 3.51 times as likely to have a diagnosis of bulimia as those who did not have HS (P < .05 for both associations).
Guirguis said that the absence of body dysmorphic disorder diagnoses in the HS cohort was unexpected. “Given HS’s known association with body image issues, we anticipated a higher prevalence of BDD,” he said. “This discrepancy may reflect underreporting or diagnostic overshadowing, where the physical symptoms of HS dominate clinical attention, potentially masking or complicating the identification of psychological conditions like BDD.”
He acknowledged certain limitations of the study, including the potential for variations in documentation practices in the database. “Additionally, there may be bias due to underrepresentation of certain demographic groups or underreporting of psychological comorbidities, which could influence the findings.”
Patricia M. Richey, MD, assistant professor of dermatology, at Boston University School of Medicine in Massachusetts, who was asked to comment on the study, said the results “should affect how physicians discuss lifestyle recommendations in those already at increased risk of psychiatric disease and disrupted body image.” The findings should also “prompt physicians to screen this patient population more thoroughly for eating disorders as we know they are an underrecognized and often undertreated entity,” she added.
Neither the study authors nor Richey reported having relevant disclosures.
A version of this article appeared on Medscape.com.
“Clinicians should actively screen for eating disorders,” particularly bulimia nervosa and binge eating disorder, in patients with HS,” lead study author Christopher Guirguis, DMD, a student at Georgetown University School of Medicine, Washington, DC, told this news organization in advance of the annual Symposium on Hidradenitis Suppurative Advances, where the study was presented during an oral abstract session. “The significant psychological burden in these patients requires a holistic approach that integrates both dermatologic and psychosocial care. Addressing their mental health needs is essential for improving overall patient outcomes and quality of life,” he added.
In collaboration with fellow Georgetown medical student and first author Lauren Chin and Mikael Horissian, MD, a dermatologist and director of the HS Clinic at Gesinger Health System, Danville, Pennsylvania, Guirguis drew from the National Institutes of Health’s All of Us Research Program to identify 1653 individuals with a diagnosis of HS and a control group of 8265 individuals without a diagnosis of HS. They used the Observational Medical Outcomes Partnership to identify anorexia nervosa, bulimia nervosa, body dysmorphic disorder, binge eating disorder, and eating disorder, unspecified. Obsessive-compulsive disorder (OCD) was also included because of its association with bulimia. They used statistical models to compare cohorts and comorbidities. “What makes this work unique is its focus on the link between HS and eating disorders, a relationship previously underexplored,” he said.
The mean age of the overall study cohort was 46.8 years, and 78.6% were female. Univariate analysis revealed that, compared with controls, individuals in the HS cohort showed significantly increased diagnoses of bulimia, binge eating disorder, OCD, and eating disorder, unspecified, by 2.6, 5.48, 2.50, and 2.43 times, respectively (P < .05 for all associations). After adjusting for age, race, sex, and ethnicity, the researchers observed that patients with HS were 4.46 times as likely to have a diagnosis of binge eating disorder and 3.51 times as likely to have a diagnosis of bulimia as those who did not have HS (P < .05 for both associations).
Guirguis said that the absence of body dysmorphic disorder diagnoses in the HS cohort was unexpected. “Given HS’s known association with body image issues, we anticipated a higher prevalence of BDD,” he said. “This discrepancy may reflect underreporting or diagnostic overshadowing, where the physical symptoms of HS dominate clinical attention, potentially masking or complicating the identification of psychological conditions like BDD.”
He acknowledged certain limitations of the study, including the potential for variations in documentation practices in the database. “Additionally, there may be bias due to underrepresentation of certain demographic groups or underreporting of psychological comorbidities, which could influence the findings.”
Patricia M. Richey, MD, assistant professor of dermatology, at Boston University School of Medicine in Massachusetts, who was asked to comment on the study, said the results “should affect how physicians discuss lifestyle recommendations in those already at increased risk of psychiatric disease and disrupted body image.” The findings should also “prompt physicians to screen this patient population more thoroughly for eating disorders as we know they are an underrecognized and often undertreated entity,” she added.
Neither the study authors nor Richey reported having relevant disclosures.
A version of this article appeared on Medscape.com.
“Clinicians should actively screen for eating disorders,” particularly bulimia nervosa and binge eating disorder, in patients with HS,” lead study author Christopher Guirguis, DMD, a student at Georgetown University School of Medicine, Washington, DC, told this news organization in advance of the annual Symposium on Hidradenitis Suppurative Advances, where the study was presented during an oral abstract session. “The significant psychological burden in these patients requires a holistic approach that integrates both dermatologic and psychosocial care. Addressing their mental health needs is essential for improving overall patient outcomes and quality of life,” he added.
In collaboration with fellow Georgetown medical student and first author Lauren Chin and Mikael Horissian, MD, a dermatologist and director of the HS Clinic at Gesinger Health System, Danville, Pennsylvania, Guirguis drew from the National Institutes of Health’s All of Us Research Program to identify 1653 individuals with a diagnosis of HS and a control group of 8265 individuals without a diagnosis of HS. They used the Observational Medical Outcomes Partnership to identify anorexia nervosa, bulimia nervosa, body dysmorphic disorder, binge eating disorder, and eating disorder, unspecified. Obsessive-compulsive disorder (OCD) was also included because of its association with bulimia. They used statistical models to compare cohorts and comorbidities. “What makes this work unique is its focus on the link between HS and eating disorders, a relationship previously underexplored,” he said.
The mean age of the overall study cohort was 46.8 years, and 78.6% were female. Univariate analysis revealed that, compared with controls, individuals in the HS cohort showed significantly increased diagnoses of bulimia, binge eating disorder, OCD, and eating disorder, unspecified, by 2.6, 5.48, 2.50, and 2.43 times, respectively (P < .05 for all associations). After adjusting for age, race, sex, and ethnicity, the researchers observed that patients with HS were 4.46 times as likely to have a diagnosis of binge eating disorder and 3.51 times as likely to have a diagnosis of bulimia as those who did not have HS (P < .05 for both associations).
Guirguis said that the absence of body dysmorphic disorder diagnoses in the HS cohort was unexpected. “Given HS’s known association with body image issues, we anticipated a higher prevalence of BDD,” he said. “This discrepancy may reflect underreporting or diagnostic overshadowing, where the physical symptoms of HS dominate clinical attention, potentially masking or complicating the identification of psychological conditions like BDD.”
He acknowledged certain limitations of the study, including the potential for variations in documentation practices in the database. “Additionally, there may be bias due to underrepresentation of certain demographic groups or underreporting of psychological comorbidities, which could influence the findings.”
Patricia M. Richey, MD, assistant professor of dermatology, at Boston University School of Medicine in Massachusetts, who was asked to comment on the study, said the results “should affect how physicians discuss lifestyle recommendations in those already at increased risk of psychiatric disease and disrupted body image.” The findings should also “prompt physicians to screen this patient population more thoroughly for eating disorders as we know they are an underrecognized and often undertreated entity,” she added.
Neither the study authors nor Richey reported having relevant disclosures.
A version of this article appeared on Medscape.com.
Guselkumab Efficacy in Crohn’s Disease Unaffected by Prior Biologic Use
VIENNA — according to a pooled analysis of the two phase 3 double-blind GALAXI 2 and 3 studies.
“We found that guselkumab was effective in both biologic-naive and biologic-inadequate subpopulations,” said coinvestigator Bruce E. Sands, MD, AGAF, gastroenterologist from Icahn School of Medicine at Mount Sinai, New York City.
These latest results add to the primary results of these studies reported earlier in 2024 that guselkumab was shown to be superior to both placebo and ustekinumab in the same patient population with moderately to severely active CD.
Sands reported the new data in a presentation at the United European Gastroenterology (UEG) Week 2024.
Guselkumab potently blocks interleukin (IL)–23 and binds to CD64, a receptor on cells that produce IL-23. The dual-acting IL-23p19 subunit inhibitor agent is currently under review by the Food and Drug Administration (FDA) for moderately to severely active CD. In September, guselkumab (Tremfya, Johnson & Johnson) was approved for use in moderately to severely active ulcerative colitis.
GALAXI 2 and 3 Pooled Dataset
In the two independent, identically designed GALAXI 2 and 3 studies, patients were randomized to guselkumab treatment at either 200 mg intravenous (IV) induction at weeks 0, 4, and 8, followed by 200 mg subcutaneous maintenance every 4 weeks, starting at week 12, or 200 mg IV induction at weeks 0, 4, and 8, followed by 100 mg subcutaneous maintenance every 8 weeks, starting at week 16; or to ustekinumab; or to placebo.
Participants were required to remain on their treatment of initial randomization for a long-term extension study (up to 5 years) looking at clinical, endoscopic, and safety outcomes, except for participants on placebo who were allowed to switch to ustekinumab if clinical response was not met at week 12.
Inclusion criteria for the studies comprised a Crohn’s Disease Activity Index score between 220 and 450, a mean daily stool frequency count > 3 or an abdominal pain score > 1, and a simple endoscopic score for CD score ≥ 6. Participants were also required to have shown an inadequate response or intolerance to oral corticosteroids, 6-mercaptopurine/azathioprine/methotrexate, or biologic therapies.
The pooled dataset included patients on either dose of guselkumab and patients on placebo (total n = 730). Of these, 52% of participants had shown a prior inadequate response to a biologic, 42% were biologic naive, and 6% had prior exposure to biologics but no documented failure. Patients on ustekinumab were not included in this analysis.
Almost all patients (97%) in the biologic-inadequate response group had previously received at least one anti–tumor necrosis factor agent, and around 15% had received vedolizumab. As expected, the biologic-inadequate responders were a lot sicker than the biologic-naive patients, Sands reported.
The composite co–primary endpoints for each guselkumab regimen vs placebo were clinical response at week 12 plus clinical remission at week 48, and clinical response at week 12 plus endoscopic response at week 48.
The major secondary endpoints comprised clinical remission at week 12 and endoscopic response also at week 12.
Short- and Long-Term Endpoints in Both Subgroups
In the biologic-naive subgroup, 54.7% of patients receiving the 200-mg dose regimen of guselkumab and 51.7% of those receiving the 100-mg dose regimen showed a clinical response at week 12 plus clinical remission at week 48, compared with 11.5% in the placebo group (P < .001 for both compared with placebo).
In the biologic-inadequate response group, 49.7% of those receiving the 200-mg dose regimen of guselkumab and 45.8% on the 100-mg dose regimen reached the composite endpoint, compared with the placebo response of 12.8% (P < .001 for both compared with placebo).
“You can see a slight decrease in response in the biologic-inadequate responders, but on the whole, the confidence intervals are highly overlapping,” said Sands.
Turning to major secondary endpoints at week 12, clinical remission was reached by 49.6% of the biologic-naive group on the 200-mg guselkumab regimen vs 16.4% on placebo, and by 46.0% of the biologic-inadequate group on the 200-mg regimen vs 19.2% on placebo (P < .001 for both subgroups). Endoscopic response was achieved by 46.3% of patients in the biologic-naive group and 29.0% in the biologic-inadequate group on the 200-mg regimen vs 18.0% and 6.4%, respectively, on placebo (P < .001 for both subgroups).
Sands noted that the drug has an excellent safety profile.
“These data show the drug works for naive patients who have failed conventional therapies, as well as for those who have failed biologic therapies,” so it could be used as a first- or second-line biologic, he added.
Sands reported potential conflicts of interest with AbbVie, Abivax, Adiso Therapeutics, Agomab, Alimentiv, Amgen, AnaptysBio, Arena Pharmaceuticals, Artugen Therapeutics, AstraZeneca, Biora Therapeutics, Boehringer Ingelheim, Boston Pharmaceuticals, Bristol-Myers Squibb, Calibr, Celgene, Celltrion, ClostraBio, Equillium, Enthera, Evommune, Ferring, Fresenius Kabi, Galapagos, Genentech (Roche), Gilead Sciences, GlaxoSmithKline, Gossamer Bio, Index Pharmaceuticals, Innovation Pharmaceuticals, Inotrem, Janssen, Kaleido, Kallyope, Lilly, Merck, Microbiotica, Mobius Care, Morphic Therapeutic, MRM Health, Pfizer, Nexus Therapeutics, Nimbus Discovery, Odyssey Therapeutics, Progenity, Prometheus Biosciences, Prometheus Laboratories, Protagonist Therapeutics, Q32 Bio, Rasayana Therapeutics, Recludix Pharma, Reistone Biopharma, Sun Pharma, Surrozen, Target RWE, Takeda, Teva, Theravance Biopharma, TLL Pharmaceutical, Tr1X, UNION Therapeutics, and Ventyx Biosciences.
A version of this article appeared on Medscape.com.
VIENNA — according to a pooled analysis of the two phase 3 double-blind GALAXI 2 and 3 studies.
“We found that guselkumab was effective in both biologic-naive and biologic-inadequate subpopulations,” said coinvestigator Bruce E. Sands, MD, AGAF, gastroenterologist from Icahn School of Medicine at Mount Sinai, New York City.
These latest results add to the primary results of these studies reported earlier in 2024 that guselkumab was shown to be superior to both placebo and ustekinumab in the same patient population with moderately to severely active CD.
Sands reported the new data in a presentation at the United European Gastroenterology (UEG) Week 2024.
Guselkumab potently blocks interleukin (IL)–23 and binds to CD64, a receptor on cells that produce IL-23. The dual-acting IL-23p19 subunit inhibitor agent is currently under review by the Food and Drug Administration (FDA) for moderately to severely active CD. In September, guselkumab (Tremfya, Johnson & Johnson) was approved for use in moderately to severely active ulcerative colitis.
GALAXI 2 and 3 Pooled Dataset
In the two independent, identically designed GALAXI 2 and 3 studies, patients were randomized to guselkumab treatment at either 200 mg intravenous (IV) induction at weeks 0, 4, and 8, followed by 200 mg subcutaneous maintenance every 4 weeks, starting at week 12, or 200 mg IV induction at weeks 0, 4, and 8, followed by 100 mg subcutaneous maintenance every 8 weeks, starting at week 16; or to ustekinumab; or to placebo.
Participants were required to remain on their treatment of initial randomization for a long-term extension study (up to 5 years) looking at clinical, endoscopic, and safety outcomes, except for participants on placebo who were allowed to switch to ustekinumab if clinical response was not met at week 12.
Inclusion criteria for the studies comprised a Crohn’s Disease Activity Index score between 220 and 450, a mean daily stool frequency count > 3 or an abdominal pain score > 1, and a simple endoscopic score for CD score ≥ 6. Participants were also required to have shown an inadequate response or intolerance to oral corticosteroids, 6-mercaptopurine/azathioprine/methotrexate, or biologic therapies.
The pooled dataset included patients on either dose of guselkumab and patients on placebo (total n = 730). Of these, 52% of participants had shown a prior inadequate response to a biologic, 42% were biologic naive, and 6% had prior exposure to biologics but no documented failure. Patients on ustekinumab were not included in this analysis.
Almost all patients (97%) in the biologic-inadequate response group had previously received at least one anti–tumor necrosis factor agent, and around 15% had received vedolizumab. As expected, the biologic-inadequate responders were a lot sicker than the biologic-naive patients, Sands reported.
The composite co–primary endpoints for each guselkumab regimen vs placebo were clinical response at week 12 plus clinical remission at week 48, and clinical response at week 12 plus endoscopic response at week 48.
The major secondary endpoints comprised clinical remission at week 12 and endoscopic response also at week 12.
Short- and Long-Term Endpoints in Both Subgroups
In the biologic-naive subgroup, 54.7% of patients receiving the 200-mg dose regimen of guselkumab and 51.7% of those receiving the 100-mg dose regimen showed a clinical response at week 12 plus clinical remission at week 48, compared with 11.5% in the placebo group (P < .001 for both compared with placebo).
In the biologic-inadequate response group, 49.7% of those receiving the 200-mg dose regimen of guselkumab and 45.8% on the 100-mg dose regimen reached the composite endpoint, compared with the placebo response of 12.8% (P < .001 for both compared with placebo).
“You can see a slight decrease in response in the biologic-inadequate responders, but on the whole, the confidence intervals are highly overlapping,” said Sands.
Turning to major secondary endpoints at week 12, clinical remission was reached by 49.6% of the biologic-naive group on the 200-mg guselkumab regimen vs 16.4% on placebo, and by 46.0% of the biologic-inadequate group on the 200-mg regimen vs 19.2% on placebo (P < .001 for both subgroups). Endoscopic response was achieved by 46.3% of patients in the biologic-naive group and 29.0% in the biologic-inadequate group on the 200-mg regimen vs 18.0% and 6.4%, respectively, on placebo (P < .001 for both subgroups).
Sands noted that the drug has an excellent safety profile.
“These data show the drug works for naive patients who have failed conventional therapies, as well as for those who have failed biologic therapies,” so it could be used as a first- or second-line biologic, he added.
Sands reported potential conflicts of interest with AbbVie, Abivax, Adiso Therapeutics, Agomab, Alimentiv, Amgen, AnaptysBio, Arena Pharmaceuticals, Artugen Therapeutics, AstraZeneca, Biora Therapeutics, Boehringer Ingelheim, Boston Pharmaceuticals, Bristol-Myers Squibb, Calibr, Celgene, Celltrion, ClostraBio, Equillium, Enthera, Evommune, Ferring, Fresenius Kabi, Galapagos, Genentech (Roche), Gilead Sciences, GlaxoSmithKline, Gossamer Bio, Index Pharmaceuticals, Innovation Pharmaceuticals, Inotrem, Janssen, Kaleido, Kallyope, Lilly, Merck, Microbiotica, Mobius Care, Morphic Therapeutic, MRM Health, Pfizer, Nexus Therapeutics, Nimbus Discovery, Odyssey Therapeutics, Progenity, Prometheus Biosciences, Prometheus Laboratories, Protagonist Therapeutics, Q32 Bio, Rasayana Therapeutics, Recludix Pharma, Reistone Biopharma, Sun Pharma, Surrozen, Target RWE, Takeda, Teva, Theravance Biopharma, TLL Pharmaceutical, Tr1X, UNION Therapeutics, and Ventyx Biosciences.
A version of this article appeared on Medscape.com.
VIENNA — according to a pooled analysis of the two phase 3 double-blind GALAXI 2 and 3 studies.
“We found that guselkumab was effective in both biologic-naive and biologic-inadequate subpopulations,” said coinvestigator Bruce E. Sands, MD, AGAF, gastroenterologist from Icahn School of Medicine at Mount Sinai, New York City.
These latest results add to the primary results of these studies reported earlier in 2024 that guselkumab was shown to be superior to both placebo and ustekinumab in the same patient population with moderately to severely active CD.
Sands reported the new data in a presentation at the United European Gastroenterology (UEG) Week 2024.
Guselkumab potently blocks interleukin (IL)–23 and binds to CD64, a receptor on cells that produce IL-23. The dual-acting IL-23p19 subunit inhibitor agent is currently under review by the Food and Drug Administration (FDA) for moderately to severely active CD. In September, guselkumab (Tremfya, Johnson & Johnson) was approved for use in moderately to severely active ulcerative colitis.
GALAXI 2 and 3 Pooled Dataset
In the two independent, identically designed GALAXI 2 and 3 studies, patients were randomized to guselkumab treatment at either 200 mg intravenous (IV) induction at weeks 0, 4, and 8, followed by 200 mg subcutaneous maintenance every 4 weeks, starting at week 12, or 200 mg IV induction at weeks 0, 4, and 8, followed by 100 mg subcutaneous maintenance every 8 weeks, starting at week 16; or to ustekinumab; or to placebo.
Participants were required to remain on their treatment of initial randomization for a long-term extension study (up to 5 years) looking at clinical, endoscopic, and safety outcomes, except for participants on placebo who were allowed to switch to ustekinumab if clinical response was not met at week 12.
Inclusion criteria for the studies comprised a Crohn’s Disease Activity Index score between 220 and 450, a mean daily stool frequency count > 3 or an abdominal pain score > 1, and a simple endoscopic score for CD score ≥ 6. Participants were also required to have shown an inadequate response or intolerance to oral corticosteroids, 6-mercaptopurine/azathioprine/methotrexate, or biologic therapies.
The pooled dataset included patients on either dose of guselkumab and patients on placebo (total n = 730). Of these, 52% of participants had shown a prior inadequate response to a biologic, 42% were biologic naive, and 6% had prior exposure to biologics but no documented failure. Patients on ustekinumab were not included in this analysis.
Almost all patients (97%) in the biologic-inadequate response group had previously received at least one anti–tumor necrosis factor agent, and around 15% had received vedolizumab. As expected, the biologic-inadequate responders were a lot sicker than the biologic-naive patients, Sands reported.
The composite co–primary endpoints for each guselkumab regimen vs placebo were clinical response at week 12 plus clinical remission at week 48, and clinical response at week 12 plus endoscopic response at week 48.
The major secondary endpoints comprised clinical remission at week 12 and endoscopic response also at week 12.
Short- and Long-Term Endpoints in Both Subgroups
In the biologic-naive subgroup, 54.7% of patients receiving the 200-mg dose regimen of guselkumab and 51.7% of those receiving the 100-mg dose regimen showed a clinical response at week 12 plus clinical remission at week 48, compared with 11.5% in the placebo group (P < .001 for both compared with placebo).
In the biologic-inadequate response group, 49.7% of those receiving the 200-mg dose regimen of guselkumab and 45.8% on the 100-mg dose regimen reached the composite endpoint, compared with the placebo response of 12.8% (P < .001 for both compared with placebo).
“You can see a slight decrease in response in the biologic-inadequate responders, but on the whole, the confidence intervals are highly overlapping,” said Sands.
Turning to major secondary endpoints at week 12, clinical remission was reached by 49.6% of the biologic-naive group on the 200-mg guselkumab regimen vs 16.4% on placebo, and by 46.0% of the biologic-inadequate group on the 200-mg regimen vs 19.2% on placebo (P < .001 for both subgroups). Endoscopic response was achieved by 46.3% of patients in the biologic-naive group and 29.0% in the biologic-inadequate group on the 200-mg regimen vs 18.0% and 6.4%, respectively, on placebo (P < .001 for both subgroups).
Sands noted that the drug has an excellent safety profile.
“These data show the drug works for naive patients who have failed conventional therapies, as well as for those who have failed biologic therapies,” so it could be used as a first- or second-line biologic, he added.
Sands reported potential conflicts of interest with AbbVie, Abivax, Adiso Therapeutics, Agomab, Alimentiv, Amgen, AnaptysBio, Arena Pharmaceuticals, Artugen Therapeutics, AstraZeneca, Biora Therapeutics, Boehringer Ingelheim, Boston Pharmaceuticals, Bristol-Myers Squibb, Calibr, Celgene, Celltrion, ClostraBio, Equillium, Enthera, Evommune, Ferring, Fresenius Kabi, Galapagos, Genentech (Roche), Gilead Sciences, GlaxoSmithKline, Gossamer Bio, Index Pharmaceuticals, Innovation Pharmaceuticals, Inotrem, Janssen, Kaleido, Kallyope, Lilly, Merck, Microbiotica, Mobius Care, Morphic Therapeutic, MRM Health, Pfizer, Nexus Therapeutics, Nimbus Discovery, Odyssey Therapeutics, Progenity, Prometheus Biosciences, Prometheus Laboratories, Protagonist Therapeutics, Q32 Bio, Rasayana Therapeutics, Recludix Pharma, Reistone Biopharma, Sun Pharma, Surrozen, Target RWE, Takeda, Teva, Theravance Biopharma, TLL Pharmaceutical, Tr1X, UNION Therapeutics, and Ventyx Biosciences.
A version of this article appeared on Medscape.com.
FROM UEG 2024
Patients With IBD More Likely to Develop, or Have Prior, T1D
VIENNA —
Their findings showed that patients with IBD had a moderately increased risk for T1D and higher odds of having prior T1D than the general population. These bidirectional associations were partially independent of shared familial factors.
Although the absolute risk for T1D is low in patients with IBD, these findings suggest that if there are nonspecific symptoms, such as weight loss and fatigue, which are typical of T1D but not of IBD, then it might be reasonable to test for diabetes, lead researcher Jiangwei Sun, PhD, postdoctoral researcher at the Karolinska Institutet, Stockholm, Sweden, told this news organization.
“Patients with IBD and T1D also tend to have worse disease outcomes for both diseases, but these two diseases are not recognized as comorbidities in the clinical guidelines,” he said.
Anecdotally, “many clinicians believe there is a higher risk of autoimmune disease in patients with IBD but not much attention is paid to type 1 diabetes,” he added.
Sun presented the study at United European Gastroenterology (UEG) Week 2024. It was also published recently in The Lancet.
Exploring the Bidirectional Relationship
Prior research in the form of a systematic review found no association between IBD and T1D, which was surprising, Sun said. Further studies found an association between IBD and incident T1D; however, these studies did not explore bidirectionality between the two diseases.
These studies also did not take shared genetic and environmental factors into consideration, though “there is known to be familial co-aggregation of IBD and T1D based on previous findings,” he said.
In this current study, Sun and colleagues compared patients with IBD with the general population, as well as with siblings without IBD to consider the potential influence of shared genetics and earlier environmental factors.
The research used two approaches to look for a bidirectional association: A nationwide matched cohort study (IBD and incident T1D) and a case-control study (IBD and prior T1D).
The cohort study included 20,314 patients with IBD aged ≤ 28 years, who were identified between 1987 and 2017. Of these, 7277 had Crohn’s disease, 10,112 had ulcerative colitis, and 2925 had unclassified IBD. There were 99,200 individually matched reference individuals.
The case-control study included 87,001 patients with IBD (without age restriction) and 431,054 matched control individuals.
Risk ratios were calculated using an adjusted hazard ratio (aHR) of incident T1D in the cohort study and an adjusted odds ratio (aOR) of prior T1D in the case-control study.
In the cohort study, the median follow-up was 14 years. Over that time, 116 patients with IBD and 353 reference individuals developed T1D. The aHR for a patient with IBD developing T1D was 1.58 (95% CI, 1.27-1.95). For patients with ulcerative colitis, the aHR of developing T1D increased to 2.02 (95% CI, 1.51-2.70); however, the association was not found for Crohn’s disease or unclassified IBD possibly because of the sample size of these latter categories, noted Sun.
In the case-control study, Sun and colleagues identified 1018 (1.2%) patients with IBD and 3496 (0.8%) control individuals who had been previously diagnosed with T1D. Patients with IBD had higher odds of having prior T1D than those without IBD (aOR, 1.36; 95% CI, 1.26-1.46). This positive association was observed in all IBD subtypes, said Sun, who added that the sample size was larger in this analysis than in the cohort analysis.
Upon comparing patients with IBD with their siblings without IBD, analyses showed similar associations between IBD and T1D; the aHR was 1.44 (95% CI, 0.97-2.15) for developing T1D, and the aOR was 1.32 (95% CI, 1.18-1.49) for prior T1D.
That these positive associations between IBD and T1D exist even when comparing patients with IBD with their siblings without IBD suggests genetics and shared environmental factors do not fully explain the association, and that later environmental factors might play a role, said Sun.
“I’m not surprised with these results,” he added. “They make sense because we know that both IBD and T1D are immunity-related diseases and have some shared pathways.”
Commenting on the study, Tine Jess, MD, director, Center for Molecular Prediction of Inflammatory Bowel Disease, PREDICT, Aalborg University in Copenhagen, Denmark, said: “The really interesting finding here is that type 1 diabetes may precede IBD, which points toward common etiologies rather than one disease leading to the other.”
“This is in line with mounting evidence that IBD is measurable at the molecular level years prior to diagnosis,” she added.
Awareness of the bidirectional association may facilitate early detection of both conditions, Sun and his colleagues noted.
Sun reported no relevant financial relationships. Jess reported receiving consultancy fees from Ferring and Pfizer.
A version of this article appeared on Medscape.com.
VIENNA —
Their findings showed that patients with IBD had a moderately increased risk for T1D and higher odds of having prior T1D than the general population. These bidirectional associations were partially independent of shared familial factors.
Although the absolute risk for T1D is low in patients with IBD, these findings suggest that if there are nonspecific symptoms, such as weight loss and fatigue, which are typical of T1D but not of IBD, then it might be reasonable to test for diabetes, lead researcher Jiangwei Sun, PhD, postdoctoral researcher at the Karolinska Institutet, Stockholm, Sweden, told this news organization.
“Patients with IBD and T1D also tend to have worse disease outcomes for both diseases, but these two diseases are not recognized as comorbidities in the clinical guidelines,” he said.
Anecdotally, “many clinicians believe there is a higher risk of autoimmune disease in patients with IBD but not much attention is paid to type 1 diabetes,” he added.
Sun presented the study at United European Gastroenterology (UEG) Week 2024. It was also published recently in The Lancet.
Exploring the Bidirectional Relationship
Prior research in the form of a systematic review found no association between IBD and T1D, which was surprising, Sun said. Further studies found an association between IBD and incident T1D; however, these studies did not explore bidirectionality between the two diseases.
These studies also did not take shared genetic and environmental factors into consideration, though “there is known to be familial co-aggregation of IBD and T1D based on previous findings,” he said.
In this current study, Sun and colleagues compared patients with IBD with the general population, as well as with siblings without IBD to consider the potential influence of shared genetics and earlier environmental factors.
The research used two approaches to look for a bidirectional association: A nationwide matched cohort study (IBD and incident T1D) and a case-control study (IBD and prior T1D).
The cohort study included 20,314 patients with IBD aged ≤ 28 years, who were identified between 1987 and 2017. Of these, 7277 had Crohn’s disease, 10,112 had ulcerative colitis, and 2925 had unclassified IBD. There were 99,200 individually matched reference individuals.
The case-control study included 87,001 patients with IBD (without age restriction) and 431,054 matched control individuals.
Risk ratios were calculated using an adjusted hazard ratio (aHR) of incident T1D in the cohort study and an adjusted odds ratio (aOR) of prior T1D in the case-control study.
In the cohort study, the median follow-up was 14 years. Over that time, 116 patients with IBD and 353 reference individuals developed T1D. The aHR for a patient with IBD developing T1D was 1.58 (95% CI, 1.27-1.95). For patients with ulcerative colitis, the aHR of developing T1D increased to 2.02 (95% CI, 1.51-2.70); however, the association was not found for Crohn’s disease or unclassified IBD possibly because of the sample size of these latter categories, noted Sun.
In the case-control study, Sun and colleagues identified 1018 (1.2%) patients with IBD and 3496 (0.8%) control individuals who had been previously diagnosed with T1D. Patients with IBD had higher odds of having prior T1D than those without IBD (aOR, 1.36; 95% CI, 1.26-1.46). This positive association was observed in all IBD subtypes, said Sun, who added that the sample size was larger in this analysis than in the cohort analysis.
Upon comparing patients with IBD with their siblings without IBD, analyses showed similar associations between IBD and T1D; the aHR was 1.44 (95% CI, 0.97-2.15) for developing T1D, and the aOR was 1.32 (95% CI, 1.18-1.49) for prior T1D.
That these positive associations between IBD and T1D exist even when comparing patients with IBD with their siblings without IBD suggests genetics and shared environmental factors do not fully explain the association, and that later environmental factors might play a role, said Sun.
“I’m not surprised with these results,” he added. “They make sense because we know that both IBD and T1D are immunity-related diseases and have some shared pathways.”
Commenting on the study, Tine Jess, MD, director, Center for Molecular Prediction of Inflammatory Bowel Disease, PREDICT, Aalborg University in Copenhagen, Denmark, said: “The really interesting finding here is that type 1 diabetes may precede IBD, which points toward common etiologies rather than one disease leading to the other.”
“This is in line with mounting evidence that IBD is measurable at the molecular level years prior to diagnosis,” she added.
Awareness of the bidirectional association may facilitate early detection of both conditions, Sun and his colleagues noted.
Sun reported no relevant financial relationships. Jess reported receiving consultancy fees from Ferring and Pfizer.
A version of this article appeared on Medscape.com.
VIENNA —
Their findings showed that patients with IBD had a moderately increased risk for T1D and higher odds of having prior T1D than the general population. These bidirectional associations were partially independent of shared familial factors.
Although the absolute risk for T1D is low in patients with IBD, these findings suggest that if there are nonspecific symptoms, such as weight loss and fatigue, which are typical of T1D but not of IBD, then it might be reasonable to test for diabetes, lead researcher Jiangwei Sun, PhD, postdoctoral researcher at the Karolinska Institutet, Stockholm, Sweden, told this news organization.
“Patients with IBD and T1D also tend to have worse disease outcomes for both diseases, but these two diseases are not recognized as comorbidities in the clinical guidelines,” he said.
Anecdotally, “many clinicians believe there is a higher risk of autoimmune disease in patients with IBD but not much attention is paid to type 1 diabetes,” he added.
Sun presented the study at United European Gastroenterology (UEG) Week 2024. It was also published recently in The Lancet.
Exploring the Bidirectional Relationship
Prior research in the form of a systematic review found no association between IBD and T1D, which was surprising, Sun said. Further studies found an association between IBD and incident T1D; however, these studies did not explore bidirectionality between the two diseases.
These studies also did not take shared genetic and environmental factors into consideration, though “there is known to be familial co-aggregation of IBD and T1D based on previous findings,” he said.
In this current study, Sun and colleagues compared patients with IBD with the general population, as well as with siblings without IBD to consider the potential influence of shared genetics and earlier environmental factors.
The research used two approaches to look for a bidirectional association: A nationwide matched cohort study (IBD and incident T1D) and a case-control study (IBD and prior T1D).
The cohort study included 20,314 patients with IBD aged ≤ 28 years, who were identified between 1987 and 2017. Of these, 7277 had Crohn’s disease, 10,112 had ulcerative colitis, and 2925 had unclassified IBD. There were 99,200 individually matched reference individuals.
The case-control study included 87,001 patients with IBD (without age restriction) and 431,054 matched control individuals.
Risk ratios were calculated using an adjusted hazard ratio (aHR) of incident T1D in the cohort study and an adjusted odds ratio (aOR) of prior T1D in the case-control study.
In the cohort study, the median follow-up was 14 years. Over that time, 116 patients with IBD and 353 reference individuals developed T1D. The aHR for a patient with IBD developing T1D was 1.58 (95% CI, 1.27-1.95). For patients with ulcerative colitis, the aHR of developing T1D increased to 2.02 (95% CI, 1.51-2.70); however, the association was not found for Crohn’s disease or unclassified IBD possibly because of the sample size of these latter categories, noted Sun.
In the case-control study, Sun and colleagues identified 1018 (1.2%) patients with IBD and 3496 (0.8%) control individuals who had been previously diagnosed with T1D. Patients with IBD had higher odds of having prior T1D than those without IBD (aOR, 1.36; 95% CI, 1.26-1.46). This positive association was observed in all IBD subtypes, said Sun, who added that the sample size was larger in this analysis than in the cohort analysis.
Upon comparing patients with IBD with their siblings without IBD, analyses showed similar associations between IBD and T1D; the aHR was 1.44 (95% CI, 0.97-2.15) for developing T1D, and the aOR was 1.32 (95% CI, 1.18-1.49) for prior T1D.
That these positive associations between IBD and T1D exist even when comparing patients with IBD with their siblings without IBD suggests genetics and shared environmental factors do not fully explain the association, and that later environmental factors might play a role, said Sun.
“I’m not surprised with these results,” he added. “They make sense because we know that both IBD and T1D are immunity-related diseases and have some shared pathways.”
Commenting on the study, Tine Jess, MD, director, Center for Molecular Prediction of Inflammatory Bowel Disease, PREDICT, Aalborg University in Copenhagen, Denmark, said: “The really interesting finding here is that type 1 diabetes may precede IBD, which points toward common etiologies rather than one disease leading to the other.”
“This is in line with mounting evidence that IBD is measurable at the molecular level years prior to diagnosis,” she added.
Awareness of the bidirectional association may facilitate early detection of both conditions, Sun and his colleagues noted.
Sun reported no relevant financial relationships. Jess reported receiving consultancy fees from Ferring and Pfizer.
A version of this article appeared on Medscape.com.
FROM UEG 2024
Effects of Bimekizumab Durable for HS Through One Year
AMSTERDAM — The monoclonal antibody according to new data from an open-label extension period.
“Efficacy and health-related quality-of-life outcomes were maintained through 2 years of treatment,” study presenter Christos C. Zouboulis, MD, professor of dermatology, venereology, and allergology, Brandenburg Medical School Theodor Fontane, Dessau, Germany, said at the European Academy of Dermatology and Venereology (EADV) 2024 Congress.
“No new safety signals were observed,” he added. “These data highlight the durability and consistency of bimekizumab treatment in patients with moderate to severe hidradenitis suppurativa,” Zouboulis concluded.
Efficacy Maintained
“This is the type of long-term data that clinicians hope to see in large phase 3 trials for hidradenitis suppurativa medications,” commented Jennifer L. Hsiao, MD, clinical associate professor of dermatology, University of Southern California, Los Angeles, who was not involved in the study.
She told this news organization that, beyond maintained improvement of patient-reported quality of life, the results are “raising the bar in terms of measuring treatment success,” with over three quarters of patients achieving a high level of response on the Hidradenitis Suppurativa Clinical Response (HiSCR) scale at the final 96-week follow-up.
“Clinicians and patients have struggled with maintaining treatment efficacy over time with the first [Food and Drug Administration]–approved class of biologics for hidradenitis suppurativa — TNF [tumor necrosis factor]–alpha antagonists,” Hsiao said. She emphasized that sustained treatment efficacy will reduce the need for continued treatment switching and “hopefully improve treatment adherence.”
“It was also helpful to see that, consistent with studies of bimekizumab in psoriasis, rates of oral candidiasis appear to decrease with prolonged exposure over 2 years, though as with any open-label extension study, study dropout is a limitation,” she said.
“The availability of long-term efficacy and safety data, such as those shown in this study, will help guide shared decision-making discussions with our patients.” Overall, Hsiao believes there is “much to be excited about in the field of hidradenitis suppurativa, with a robust pipeline of potential treatments.”
One-Year Extension Study
HS is a “chronic and debilitating inflammatory skin disease,” Zouboulis told the audience. He noted that interleukin (IL)–17F and IL-17A are highly expressed in lesional skin and play a role in the disease immunopathogenesis.
Bimekizumab is a humanized immunoglobulin G1 monoclonal antibody that selectively inhibits both IL-17F and IL-17A. It has previously demonstrated clinically meaningful improvements in patients with moderate to severe HS in the phase 3 BE HEARD I and BE HEARD II trials evaluating several dosing regimens.
Zouboulis said the current analysis combines data from the two phase 3 studies with the BE HEARD EXT open-label extension study, in which patients from both trials were continued on bimekizumab 320 mg every 2 weeks.
Of the 1014 patients initially enrolled in the two trials, 556 continued into the open-label extension. Their average age was 36.6 years, and 53.8% were women. The majority (80.6%) were White. Of the 556 patients enrolled in the extension, 446 completed the 1-year extension study.
The average draining tunnel count at baseline was 3.8, and 54.5% had Hurley stage II disease; the remaining 45.5% had stage III disease. The mean total Dermatology Life Quality Index (DLQI) score at baseline was 11.0, indicating the HS was having a very large impact on the patients’ lives.
After the 16-week initial treatment period and the maintenance treatment period out to 48 weeks, 64.0% of patients achieved HiSCR75, indicating at least a 75% reduction from baseline in the total abscess and inflammatory nodule count, rising to 77.1% at the end of the open-label extension, after a total follow-up of 96 weeks.
HiSCR100 scores, indicating a 100% reduction in total abscess and inflammatory nodule counts, were achieved by 30.2% of 556 patients after 48 weeks and 44.2% of 446 at the 96-week follow-up.
These findings were mirrored by substantial reductions on the International HS Severity Score System, with a 70.3% reduction over baseline at 48 weeks and a 79.8% reduction at the final follow-up.
There were also “clinically meaningful” reductions in the total draining tunnel count at 1 year that were further reduced at 2 years, Zouboulis reported, at a 57.5% reduction over baseline, increasing to 73.7% by 96 weeks. The mean draining tunnel count at the end of follow-up was 1.1.
Over the full 96 weeks, the mean DLQI score reduced from 11.0 to 4.7, with 33.9% of patients achieving a score of 0 or 1 on the scale, which he said is basically patients saying: “I don’t have disease now.”
Finally, the safety data showed that there were “no differences compared to what we knew before,” Zouboulis said, with the most common treatment-related adverse events being hidradenitis, coronavirus infection, and oral candidiasis. There were few serious and severe treatment-related adverse events, and few that led to treatment discontinuation.
The study was funded by UCB.Zouboulis declared relationships with AstraZeneca, Boehringer Ingelheim, Brandenburg Medical School Theodor Fontane, EAD, European Union, German Federal Ministry of Education and Research, GSK, InflaRx, MSD, Novartis, Relaxera, UCB, Almirall, Boehringer Ingelheim, Eli Lilly, Idorsia, Incyte, L’Oréal, NAOS-BIODERMA, Pfizer, PM, Sanofi. Hsiao is on the board of directors for the Hidradenitis Suppurativa Foundation and has declared relationships with AbbVie, Aclaris Therapeutics, Amgen, Boehringer Ingelheim, Incyte, Novartis, Sanofi-Regeneron, and UCB.
A version of this article appeared on Medscape.com.
AMSTERDAM — The monoclonal antibody according to new data from an open-label extension period.
“Efficacy and health-related quality-of-life outcomes were maintained through 2 years of treatment,” study presenter Christos C. Zouboulis, MD, professor of dermatology, venereology, and allergology, Brandenburg Medical School Theodor Fontane, Dessau, Germany, said at the European Academy of Dermatology and Venereology (EADV) 2024 Congress.
“No new safety signals were observed,” he added. “These data highlight the durability and consistency of bimekizumab treatment in patients with moderate to severe hidradenitis suppurativa,” Zouboulis concluded.
Efficacy Maintained
“This is the type of long-term data that clinicians hope to see in large phase 3 trials for hidradenitis suppurativa medications,” commented Jennifer L. Hsiao, MD, clinical associate professor of dermatology, University of Southern California, Los Angeles, who was not involved in the study.
She told this news organization that, beyond maintained improvement of patient-reported quality of life, the results are “raising the bar in terms of measuring treatment success,” with over three quarters of patients achieving a high level of response on the Hidradenitis Suppurativa Clinical Response (HiSCR) scale at the final 96-week follow-up.
“Clinicians and patients have struggled with maintaining treatment efficacy over time with the first [Food and Drug Administration]–approved class of biologics for hidradenitis suppurativa — TNF [tumor necrosis factor]–alpha antagonists,” Hsiao said. She emphasized that sustained treatment efficacy will reduce the need for continued treatment switching and “hopefully improve treatment adherence.”
“It was also helpful to see that, consistent with studies of bimekizumab in psoriasis, rates of oral candidiasis appear to decrease with prolonged exposure over 2 years, though as with any open-label extension study, study dropout is a limitation,” she said.
“The availability of long-term efficacy and safety data, such as those shown in this study, will help guide shared decision-making discussions with our patients.” Overall, Hsiao believes there is “much to be excited about in the field of hidradenitis suppurativa, with a robust pipeline of potential treatments.”
One-Year Extension Study
HS is a “chronic and debilitating inflammatory skin disease,” Zouboulis told the audience. He noted that interleukin (IL)–17F and IL-17A are highly expressed in lesional skin and play a role in the disease immunopathogenesis.
Bimekizumab is a humanized immunoglobulin G1 monoclonal antibody that selectively inhibits both IL-17F and IL-17A. It has previously demonstrated clinically meaningful improvements in patients with moderate to severe HS in the phase 3 BE HEARD I and BE HEARD II trials evaluating several dosing regimens.
Zouboulis said the current analysis combines data from the two phase 3 studies with the BE HEARD EXT open-label extension study, in which patients from both trials were continued on bimekizumab 320 mg every 2 weeks.
Of the 1014 patients initially enrolled in the two trials, 556 continued into the open-label extension. Their average age was 36.6 years, and 53.8% were women. The majority (80.6%) were White. Of the 556 patients enrolled in the extension, 446 completed the 1-year extension study.
The average draining tunnel count at baseline was 3.8, and 54.5% had Hurley stage II disease; the remaining 45.5% had stage III disease. The mean total Dermatology Life Quality Index (DLQI) score at baseline was 11.0, indicating the HS was having a very large impact on the patients’ lives.
After the 16-week initial treatment period and the maintenance treatment period out to 48 weeks, 64.0% of patients achieved HiSCR75, indicating at least a 75% reduction from baseline in the total abscess and inflammatory nodule count, rising to 77.1% at the end of the open-label extension, after a total follow-up of 96 weeks.
HiSCR100 scores, indicating a 100% reduction in total abscess and inflammatory nodule counts, were achieved by 30.2% of 556 patients after 48 weeks and 44.2% of 446 at the 96-week follow-up.
These findings were mirrored by substantial reductions on the International HS Severity Score System, with a 70.3% reduction over baseline at 48 weeks and a 79.8% reduction at the final follow-up.
There were also “clinically meaningful” reductions in the total draining tunnel count at 1 year that were further reduced at 2 years, Zouboulis reported, at a 57.5% reduction over baseline, increasing to 73.7% by 96 weeks. The mean draining tunnel count at the end of follow-up was 1.1.
Over the full 96 weeks, the mean DLQI score reduced from 11.0 to 4.7, with 33.9% of patients achieving a score of 0 or 1 on the scale, which he said is basically patients saying: “I don’t have disease now.”
Finally, the safety data showed that there were “no differences compared to what we knew before,” Zouboulis said, with the most common treatment-related adverse events being hidradenitis, coronavirus infection, and oral candidiasis. There were few serious and severe treatment-related adverse events, and few that led to treatment discontinuation.
The study was funded by UCB.Zouboulis declared relationships with AstraZeneca, Boehringer Ingelheim, Brandenburg Medical School Theodor Fontane, EAD, European Union, German Federal Ministry of Education and Research, GSK, InflaRx, MSD, Novartis, Relaxera, UCB, Almirall, Boehringer Ingelheim, Eli Lilly, Idorsia, Incyte, L’Oréal, NAOS-BIODERMA, Pfizer, PM, Sanofi. Hsiao is on the board of directors for the Hidradenitis Suppurativa Foundation and has declared relationships with AbbVie, Aclaris Therapeutics, Amgen, Boehringer Ingelheim, Incyte, Novartis, Sanofi-Regeneron, and UCB.
A version of this article appeared on Medscape.com.
AMSTERDAM — The monoclonal antibody according to new data from an open-label extension period.
“Efficacy and health-related quality-of-life outcomes were maintained through 2 years of treatment,” study presenter Christos C. Zouboulis, MD, professor of dermatology, venereology, and allergology, Brandenburg Medical School Theodor Fontane, Dessau, Germany, said at the European Academy of Dermatology and Venereology (EADV) 2024 Congress.
“No new safety signals were observed,” he added. “These data highlight the durability and consistency of bimekizumab treatment in patients with moderate to severe hidradenitis suppurativa,” Zouboulis concluded.
Efficacy Maintained
“This is the type of long-term data that clinicians hope to see in large phase 3 trials for hidradenitis suppurativa medications,” commented Jennifer L. Hsiao, MD, clinical associate professor of dermatology, University of Southern California, Los Angeles, who was not involved in the study.
She told this news organization that, beyond maintained improvement of patient-reported quality of life, the results are “raising the bar in terms of measuring treatment success,” with over three quarters of patients achieving a high level of response on the Hidradenitis Suppurativa Clinical Response (HiSCR) scale at the final 96-week follow-up.
“Clinicians and patients have struggled with maintaining treatment efficacy over time with the first [Food and Drug Administration]–approved class of biologics for hidradenitis suppurativa — TNF [tumor necrosis factor]–alpha antagonists,” Hsiao said. She emphasized that sustained treatment efficacy will reduce the need for continued treatment switching and “hopefully improve treatment adherence.”
“It was also helpful to see that, consistent with studies of bimekizumab in psoriasis, rates of oral candidiasis appear to decrease with prolonged exposure over 2 years, though as with any open-label extension study, study dropout is a limitation,” she said.
“The availability of long-term efficacy and safety data, such as those shown in this study, will help guide shared decision-making discussions with our patients.” Overall, Hsiao believes there is “much to be excited about in the field of hidradenitis suppurativa, with a robust pipeline of potential treatments.”
One-Year Extension Study
HS is a “chronic and debilitating inflammatory skin disease,” Zouboulis told the audience. He noted that interleukin (IL)–17F and IL-17A are highly expressed in lesional skin and play a role in the disease immunopathogenesis.
Bimekizumab is a humanized immunoglobulin G1 monoclonal antibody that selectively inhibits both IL-17F and IL-17A. It has previously demonstrated clinically meaningful improvements in patients with moderate to severe HS in the phase 3 BE HEARD I and BE HEARD II trials evaluating several dosing regimens.
Zouboulis said the current analysis combines data from the two phase 3 studies with the BE HEARD EXT open-label extension study, in which patients from both trials were continued on bimekizumab 320 mg every 2 weeks.
Of the 1014 patients initially enrolled in the two trials, 556 continued into the open-label extension. Their average age was 36.6 years, and 53.8% were women. The majority (80.6%) were White. Of the 556 patients enrolled in the extension, 446 completed the 1-year extension study.
The average draining tunnel count at baseline was 3.8, and 54.5% had Hurley stage II disease; the remaining 45.5% had stage III disease. The mean total Dermatology Life Quality Index (DLQI) score at baseline was 11.0, indicating the HS was having a very large impact on the patients’ lives.
After the 16-week initial treatment period and the maintenance treatment period out to 48 weeks, 64.0% of patients achieved HiSCR75, indicating at least a 75% reduction from baseline in the total abscess and inflammatory nodule count, rising to 77.1% at the end of the open-label extension, after a total follow-up of 96 weeks.
HiSCR100 scores, indicating a 100% reduction in total abscess and inflammatory nodule counts, were achieved by 30.2% of 556 patients after 48 weeks and 44.2% of 446 at the 96-week follow-up.
These findings were mirrored by substantial reductions on the International HS Severity Score System, with a 70.3% reduction over baseline at 48 weeks and a 79.8% reduction at the final follow-up.
There were also “clinically meaningful” reductions in the total draining tunnel count at 1 year that were further reduced at 2 years, Zouboulis reported, at a 57.5% reduction over baseline, increasing to 73.7% by 96 weeks. The mean draining tunnel count at the end of follow-up was 1.1.
Over the full 96 weeks, the mean DLQI score reduced from 11.0 to 4.7, with 33.9% of patients achieving a score of 0 or 1 on the scale, which he said is basically patients saying: “I don’t have disease now.”
Finally, the safety data showed that there were “no differences compared to what we knew before,” Zouboulis said, with the most common treatment-related adverse events being hidradenitis, coronavirus infection, and oral candidiasis. There were few serious and severe treatment-related adverse events, and few that led to treatment discontinuation.
The study was funded by UCB.Zouboulis declared relationships with AstraZeneca, Boehringer Ingelheim, Brandenburg Medical School Theodor Fontane, EAD, European Union, German Federal Ministry of Education and Research, GSK, InflaRx, MSD, Novartis, Relaxera, UCB, Almirall, Boehringer Ingelheim, Eli Lilly, Idorsia, Incyte, L’Oréal, NAOS-BIODERMA, Pfizer, PM, Sanofi. Hsiao is on the board of directors for the Hidradenitis Suppurativa Foundation and has declared relationships with AbbVie, Aclaris Therapeutics, Amgen, Boehringer Ingelheim, Incyte, Novartis, Sanofi-Regeneron, and UCB.
A version of this article appeared on Medscape.com.
FROM EADV 2024
Coming Soon: A New Disease Definition, ‘Clinical Obesity’
SAN ANTONIO, TEXAS —
The authors of the new framework are a Lancet Commission of 56 of the world’s leading obesity experts, including academic clinicians, scientists, public health experts, patient representatives, and officers from the World Health Organization. Following peer review, it will be launched via livestream and published in Lancet Diabetes & Endocrinology in mid-January 2025, with formal endorsement from more than 75 medical societies and other relevant stakeholder organizations.
On November 4, 2024, at the Obesity Society’s Obesity Week meeting, the publication’s lead author, Francesco Rubino, MD, Chair of Bariatric and Metabolic Surgery at King’s College London in England, gave a preview. He began by noting that, despite the declaration of obesity as a chronic disease over a decade ago, the concept is still debated and not widely accepted by the public or even by all in the medical community.
“The idea of obesity as a disease remains highly controversial,” Rubino noted, adding that the current body mass index (BMI)–based definition contributes to this because it doesn’t distinguish between people whose excess adiposity place them at excess risk for disease but they’re currently healthy vs those who already have undergone bodily harm from that adiposity.
“Having a framework that distinguishes at an individual level when you are in a condition of risk and when you have a condition of disease is fundamentally important. You don’t want to blur the picture in either direction, because obviously the consequence would be quite significant. ... So, the commission focused exactly on that point,” he said.
The new paper will propose a two-part clinical approach: First, assess whether the patient has excess adiposity, with methods that will be outlined. Next, assess on an organ-by-organ basis for the presence of abnormalities related to excess adiposity, or “clinical obesity.” The document will also provide those specific criteria, Rubino said, noting that those details are under embargo until January.
However, he did say that “We are going to propose a pragmatic approach to say that BMI alone is not enough in the clinic. It’s okay as a screening tool, but when somebody potentially has obesity, then you have to add additional measures of adiposity that makes sure you decrease the level of risk… Once you have obesity, then you need to establish if it’s clinical or nonclinical.”
Asked to comment, session moderator John D. Clark, MD, PhD, Chief Population Health Officer at Sharp Rees-Stealy Medical Group, San Diego, California, said in an interview, “I think it’ll help explain and move medicine as a whole in a direction to a greater understanding of obesity actually being a disease, how to define it, and how to identify it. And will, I think, lead to a greater understanding of the underlying disease.”
And, Clark said, it should also help target individuals with preventive vs therapeutic approaches. “I would describe it as matching the right tool to the right patient. If a person has clinical obesity, they likely can and would benefit from either different or additional tools, as opposed to otherwise healthy obesity.”
Rubino said he hopes the new framework will prompt improvements in reimbursement and public policy. “Policymakers scratch their heads when they have limited resources and you need to prioritize things. Having an obesity definition that is blurry doesn’t allow you to have a fair, human, and meaningful prioritization. ... Now that we have drugs that cannot be given to 100% of people, how do you decide who gets them first? I hope this will make it easier for people to access treatment. At the moment, it is not only difficult, but it’s also unfair. It’s random. Somebody gets access, while somebody else who is very, very sick has no access. I don’t think that’s what we want.”
A version of this article appeared on Medscape.com.
SAN ANTONIO, TEXAS —
The authors of the new framework are a Lancet Commission of 56 of the world’s leading obesity experts, including academic clinicians, scientists, public health experts, patient representatives, and officers from the World Health Organization. Following peer review, it will be launched via livestream and published in Lancet Diabetes & Endocrinology in mid-January 2025, with formal endorsement from more than 75 medical societies and other relevant stakeholder organizations.
On November 4, 2024, at the Obesity Society’s Obesity Week meeting, the publication’s lead author, Francesco Rubino, MD, Chair of Bariatric and Metabolic Surgery at King’s College London in England, gave a preview. He began by noting that, despite the declaration of obesity as a chronic disease over a decade ago, the concept is still debated and not widely accepted by the public or even by all in the medical community.
“The idea of obesity as a disease remains highly controversial,” Rubino noted, adding that the current body mass index (BMI)–based definition contributes to this because it doesn’t distinguish between people whose excess adiposity place them at excess risk for disease but they’re currently healthy vs those who already have undergone bodily harm from that adiposity.
“Having a framework that distinguishes at an individual level when you are in a condition of risk and when you have a condition of disease is fundamentally important. You don’t want to blur the picture in either direction, because obviously the consequence would be quite significant. ... So, the commission focused exactly on that point,” he said.
The new paper will propose a two-part clinical approach: First, assess whether the patient has excess adiposity, with methods that will be outlined. Next, assess on an organ-by-organ basis for the presence of abnormalities related to excess adiposity, or “clinical obesity.” The document will also provide those specific criteria, Rubino said, noting that those details are under embargo until January.
However, he did say that “We are going to propose a pragmatic approach to say that BMI alone is not enough in the clinic. It’s okay as a screening tool, but when somebody potentially has obesity, then you have to add additional measures of adiposity that makes sure you decrease the level of risk… Once you have obesity, then you need to establish if it’s clinical or nonclinical.”
Asked to comment, session moderator John D. Clark, MD, PhD, Chief Population Health Officer at Sharp Rees-Stealy Medical Group, San Diego, California, said in an interview, “I think it’ll help explain and move medicine as a whole in a direction to a greater understanding of obesity actually being a disease, how to define it, and how to identify it. And will, I think, lead to a greater understanding of the underlying disease.”
And, Clark said, it should also help target individuals with preventive vs therapeutic approaches. “I would describe it as matching the right tool to the right patient. If a person has clinical obesity, they likely can and would benefit from either different or additional tools, as opposed to otherwise healthy obesity.”
Rubino said he hopes the new framework will prompt improvements in reimbursement and public policy. “Policymakers scratch their heads when they have limited resources and you need to prioritize things. Having an obesity definition that is blurry doesn’t allow you to have a fair, human, and meaningful prioritization. ... Now that we have drugs that cannot be given to 100% of people, how do you decide who gets them first? I hope this will make it easier for people to access treatment. At the moment, it is not only difficult, but it’s also unfair. It’s random. Somebody gets access, while somebody else who is very, very sick has no access. I don’t think that’s what we want.”
A version of this article appeared on Medscape.com.
SAN ANTONIO, TEXAS —
The authors of the new framework are a Lancet Commission of 56 of the world’s leading obesity experts, including academic clinicians, scientists, public health experts, patient representatives, and officers from the World Health Organization. Following peer review, it will be launched via livestream and published in Lancet Diabetes & Endocrinology in mid-January 2025, with formal endorsement from more than 75 medical societies and other relevant stakeholder organizations.
On November 4, 2024, at the Obesity Society’s Obesity Week meeting, the publication’s lead author, Francesco Rubino, MD, Chair of Bariatric and Metabolic Surgery at King’s College London in England, gave a preview. He began by noting that, despite the declaration of obesity as a chronic disease over a decade ago, the concept is still debated and not widely accepted by the public or even by all in the medical community.
“The idea of obesity as a disease remains highly controversial,” Rubino noted, adding that the current body mass index (BMI)–based definition contributes to this because it doesn’t distinguish between people whose excess adiposity place them at excess risk for disease but they’re currently healthy vs those who already have undergone bodily harm from that adiposity.
“Having a framework that distinguishes at an individual level when you are in a condition of risk and when you have a condition of disease is fundamentally important. You don’t want to blur the picture in either direction, because obviously the consequence would be quite significant. ... So, the commission focused exactly on that point,” he said.
The new paper will propose a two-part clinical approach: First, assess whether the patient has excess adiposity, with methods that will be outlined. Next, assess on an organ-by-organ basis for the presence of abnormalities related to excess adiposity, or “clinical obesity.” The document will also provide those specific criteria, Rubino said, noting that those details are under embargo until January.
However, he did say that “We are going to propose a pragmatic approach to say that BMI alone is not enough in the clinic. It’s okay as a screening tool, but when somebody potentially has obesity, then you have to add additional measures of adiposity that makes sure you decrease the level of risk… Once you have obesity, then you need to establish if it’s clinical or nonclinical.”
Asked to comment, session moderator John D. Clark, MD, PhD, Chief Population Health Officer at Sharp Rees-Stealy Medical Group, San Diego, California, said in an interview, “I think it’ll help explain and move medicine as a whole in a direction to a greater understanding of obesity actually being a disease, how to define it, and how to identify it. And will, I think, lead to a greater understanding of the underlying disease.”
And, Clark said, it should also help target individuals with preventive vs therapeutic approaches. “I would describe it as matching the right tool to the right patient. If a person has clinical obesity, they likely can and would benefit from either different or additional tools, as opposed to otherwise healthy obesity.”
Rubino said he hopes the new framework will prompt improvements in reimbursement and public policy. “Policymakers scratch their heads when they have limited resources and you need to prioritize things. Having an obesity definition that is blurry doesn’t allow you to have a fair, human, and meaningful prioritization. ... Now that we have drugs that cannot be given to 100% of people, how do you decide who gets them first? I hope this will make it easier for people to access treatment. At the moment, it is not only difficult, but it’s also unfair. It’s random. Somebody gets access, while somebody else who is very, very sick has no access. I don’t think that’s what we want.”
A version of this article appeared on Medscape.com.
FROM OBESITY WEEK