Conference Coverage

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, the landscape of DMD therapy is transforming at a rapid pace. “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, the landscape of DMD therapy is transforming at a rapid pace. “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, the landscape of DMD therapy is transforming at a rapid pace. “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.

Individuals with hidradenitis suppurativa (HS) face a significantly increased risk for bulimia nervosa and binge eating disorder, an analysis of national data showed.

“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.

Chrisopher Guirguis

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.

Individuals with hidradenitis suppurativa (HS) face a significantly increased risk for bulimia nervosa and binge eating disorder, an analysis of national data showed.

“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.

Chrisopher Guirguis

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.

Individuals with hidradenitis suppurativa (HS) face a significantly increased risk for bulimia nervosa and binge eating disorder, an analysis of national data showed.

“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.

Chrisopher Guirguis

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.

VIENNA — Guselkumab has been shown to be efficacious vs placebo in patients with moderately to severely active Crohn’s disease (CD), regardless of prior biologic therapy exposure, 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 — Guselkumab has been shown to be efficacious vs placebo in patients with moderately to severely active Crohn’s disease (CD), regardless of prior biologic therapy exposure, 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 — Guselkumab has been shown to be efficacious vs placebo in patients with moderately to severely active Crohn’s disease (CD), regardless of prior biologic therapy exposure, 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 — Researchers have determined a bidirectional relationship between inflammatory bowel disease (IBD) and type 1 diabetes (T1D), using both a cohort and case-control approach to analysis.

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 — Researchers have determined a bidirectional relationship between inflammatory bowel disease (IBD) and type 1 diabetes (T1D), using both a cohort and case-control approach to analysis.

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 — Researchers have determined a bidirectional relationship between inflammatory bowel disease (IBD) and type 1 diabetes (T1D), using both a cohort and case-control approach to analysis.

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.

AMSTERDAM — The monoclonal antibody bimekizumab (Bimzelx) achieves significant and clinically meaningful improvements in moderate to severe hidradenitis suppurativa (HS) that are maintained well beyond the initial 1-year clinical trial treatment period, 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 bimekizumab (Bimzelx) achieves significant and clinically meaningful improvements in moderate to severe hidradenitis suppurativa (HS) that are maintained well beyond the initial 1-year clinical trial treatment period, 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 bimekizumab (Bimzelx) achieves significant and clinically meaningful improvements in moderate to severe hidradenitis suppurativa (HS) that are maintained well beyond the initial 1-year clinical trial treatment period, 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.

SAN ANTONIO, TEXAS — An upcoming document will entirely reframe obesity as a “condition of excess adiposity” that constitutes a disease called “clinical obesity” when related tissue and organ abnormalities are present.

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 — An upcoming document will entirely reframe obesity as a “condition of excess adiposity” that constitutes a disease called “clinical obesity” when related tissue and organ abnormalities are present.

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 — An upcoming document will entirely reframe obesity as a “condition of excess adiposity” that constitutes a disease called “clinical obesity” when related tissue and organ abnormalities are present.

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.

Misinformation and outdated protocols contribute to the suboptimal management of anaphylaxis by patients and healthcare professionals, based on data from two new studies presented at the American College of Allergy, Asthma and Immunology Annual Scientific Meeting.

Anaphylaxis can strike suddenly, and many patients and caregivers at risk do not know which symptoms to treat with epinephrine, said Joni Chow, DO, of Baylor College of Medicine, San Antonio, Texas, in her presentation at the meeting.

“Early identification of anaphylaxis and early intervention with epinephrine are critical for improving patient outcomes,” Chow said in an interview.

“Many allergic reactions occur in community settings, where written action plans serve to instruct patients and caregivers on how to recognize and respond to these emergencies,” she said. “Currently, anaphylaxis action plans are developed based on the consensus of healthcare professionals, with limited information available on the preferences of patients and caregivers,” she noted. However, even with action plans, many patients and families struggle to recognize and manage severe allergic reactions effectively, she added.

In response to this issue, Chow and colleagues created a survey designed to assess the understanding of anaphylaxis recognition and management by patients and caregivers and to identify their preferences regarding the elements included in the action plans.

In the study, Chow and colleagues surveyed 96 patients and caregivers in an allergy clinic waiting room. The majority (95%) of the patients were prescribed epinephrine. Although 73% said they were comfortable identifying signs of anaphylaxis, only 14% said they were likely to use epinephrine as a first-line treatment.

The most common reason given for avoiding epinephrine was uncertainty over which symptoms to treat (40.6%), followed by hesitancy to visit an emergency department (24%), hesitancy to call 911 (17.7%), uncertainty about how to use epinephrine auto-injectors (11.5%), and fear of needles (5.2%).

Although 85% of the respondents understood that antihistamine use does not prevent the need for epinephrine in cases of anaphylactic reactions, 23.7% said they would use an antihistamine as the first treatment in these cases.

For patients with rash and wheezing after a suspected allergen exposure, approximately two thirds (64.5%) of the respondents said they would inject epinephrine and 10.8% would drive to the emergency room before taking any action, Chow said in her presentation.

The relatively low impact of fear of needles was unexpected, as fear of needles is considered a significant deterrent to epinephrine use, Chow told this news organization. “However, our respondents were more inclined to acknowledge a reluctance to escalate to emergency response as the major barrier to treatment,” she said.

The survey also asked patients what features of an anaphylaxis action plan would be most helpful. A majority of respondents (93%) rated a section for the management of mild (non-anaphylactic) allergic reaction symptoms as somewhat or very important. Visual aids for injection of epinephrine and visuals of anaphylaxis symptoms also ranked as somewhat or very important for 87.6% and 81% of respondents, respectively.

The study highlights the importance of educating allergy patients on recognizing and treating anaphylaxis and demonstrates that visuals were preferred in this survey population, Chow said. “Most patients and caregivers from our surveyed population report knowing how to treat anaphylaxis, but many would not use epinephrine as the first treatment,” she noted.

“The study focused on a single community clinic, and it would be beneficial to gather feedback from patients and caregivers representing a wider variety of educational, cultural, social, and socioeconomic backgrounds,” Chow told this news organization. “Additionally, input from other stakeholders, such as school nurses, would enhance knowledge,” she said.
 

Clinical Anaphylaxis Protocols Fall Short

A second study presented at the meeting showed the need to improve anaphylaxis education for clinicians.

Discrepancies in anaphylaxis management include variations in the definition and treatment of the condition, according to Carly Gunderson, DO, of Memorial Healthcare System, Pembroke Pines, Florida, who presented the study at the meeting.

“So often, we see patients in our office with a history of symptoms that meet criteria for anaphylaxis, yet when they call 911 and emergency medical services (EMS) arrive, they never receive epinephrine,” Gunderson said in an interview. “They receive antihistamines, steroids, everything except epinephrine, which is incredibly concerning given that epinephrine is always the first-line treatment for anaphylaxis,” she said.

“Because EMS providers are often the first healthcare professionals to assess patients experiencing anaphylaxis, their ability to recognize and appropriately treat anaphylaxis is essential,” Gunderson emphasized.

Gunderson and colleagues analyzed data from 30 states with mandatory Advanced Cardiac Life Support protocols to identify gaps in recognizing anaphylaxis and areas for improvement in prehospital management.

Only 15 states (50%) included gastrointestinal symptoms in the definition of anaphylaxis, 40% included neurologic manifestations, and 47% used a two-organ system definition, Gunderson noted in her presentation.

All 30 state protocols recommended diphenhydramine and epinephrine for anaphylactic reactions, 90% recommended albuterol if respiratory symptoms were present, 73% recommended intravenous fluids, and 60% recommended steroids. All but one of the state protocols listed epinephrine as the first-line recommendation for anaphylaxis; 25 states allowed epinephrine autoinjectors and 17 provided autoinjectors.

“We were shocked by how many protocols didn’t include gastrointestinal (abdominal pain, vomiting) or neurologic (lethargy, altered mental status) manifestations, when these are common presenting symptoms of anaphylaxis,” Gunderson told this news organization.

“We were also disappointed by how many protocols continue to recommend outdated interventions such as first-generation antihistamines and corticosteroids in the treatment of anaphylaxis,” she said.

Although anaphylaxis management has come a long way, the current study suggests that there is clearly room for improvement in the education of healthcare providers on how to identify and treat anaphylaxis, said Gunderson. “Most people think of anaphylaxis as the typical ‘face swelling up, throat closing’ type of reaction, which it can be, but in reality, there are so many other ways that it can present,” she said. “Healthcare providers must be aware of all of these possible manifestations so that we can treat in a timely manner to improve outcomes,” she added.

Limitations of the study included the focus only on states with mandatory or model EMS protocols, Gunderson told this news organization. As for additional research, the most important next steps are practical ones, namely, identifying ways to realistically implement necessary protocol changes, she said.
 

Real-World Data Support Need for Education

Real-world studies are important to identify current practice and opportunities for improvement, S. Shahzad Mustafa, MD, lead physician in allergy, immunology, and rheumatology at Rochester Regional Health and clinical associate professor of medicine at the University of Rochester School of Medicine and Dentistry, Rochester, New York, said in an interview.

“Management of anaphylaxis continues to evolve, and studies like these can help standardize evidence-based care across different medical settings, such as emergency medical services, urgent care, and emergency departments,” said Mustafa, who was not involved in either study.

The findings of the two studies were not unexpected, Mustafa said. “Heterogeneity in medical care is well recognized in numerous conditions, and anaphylaxis is no different. Patients and healthcare providers continue to have hesitation to use epinephrine and continue to overly rely on antihistamines and/or systemic steroids,” he noted.

For both studies, the takeaway message is that education is paramount to optimize anaphylaxis management, Mustafa told this news organization. “Education needs to focus on timely recognition of anaphylaxis, including atypical features such as gastrointestinal symptoms, and appropriate therapy with epinephrine,” he said.

Looking ahead, “research demonstrating differences in clinical outcomes with differing approaches to anaphylaxis may highlight the importance of early recognition and treatment with epinephrine,” said Mustafa. Management of anaphylaxis also lends itself to quality improvement studies, he added.

Neither of the studies received any outside funding. The researchers had no financial conflicts to disclose. Mustafa had no disclosures related to anaphylaxis but disclosed serving on the speakers’ bureau for Genentech, GSK, AstraZeneca, Regeneron/Sanofi, and CSL Behring and received grants from Takeda.
 

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

Misinformation and outdated protocols contribute to the suboptimal management of anaphylaxis by patients and healthcare professionals, based on data from two new studies presented at the American College of Allergy, Asthma and Immunology Annual Scientific Meeting.

Anaphylaxis can strike suddenly, and many patients and caregivers at risk do not know which symptoms to treat with epinephrine, said Joni Chow, DO, of Baylor College of Medicine, San Antonio, Texas, in her presentation at the meeting.

“Early identification of anaphylaxis and early intervention with epinephrine are critical for improving patient outcomes,” Chow said in an interview.

“Many allergic reactions occur in community settings, where written action plans serve to instruct patients and caregivers on how to recognize and respond to these emergencies,” she said. “Currently, anaphylaxis action plans are developed based on the consensus of healthcare professionals, with limited information available on the preferences of patients and caregivers,” she noted. However, even with action plans, many patients and families struggle to recognize and manage severe allergic reactions effectively, she added.

In response to this issue, Chow and colleagues created a survey designed to assess the understanding of anaphylaxis recognition and management by patients and caregivers and to identify their preferences regarding the elements included in the action plans.

In the study, Chow and colleagues surveyed 96 patients and caregivers in an allergy clinic waiting room. The majority (95%) of the patients were prescribed epinephrine. Although 73% said they were comfortable identifying signs of anaphylaxis, only 14% said they were likely to use epinephrine as a first-line treatment.

The most common reason given for avoiding epinephrine was uncertainty over which symptoms to treat (40.6%), followed by hesitancy to visit an emergency department (24%), hesitancy to call 911 (17.7%), uncertainty about how to use epinephrine auto-injectors (11.5%), and fear of needles (5.2%).

Although 85% of the respondents understood that antihistamine use does not prevent the need for epinephrine in cases of anaphylactic reactions, 23.7% said they would use an antihistamine as the first treatment in these cases.

For patients with rash and wheezing after a suspected allergen exposure, approximately two thirds (64.5%) of the respondents said they would inject epinephrine and 10.8% would drive to the emergency room before taking any action, Chow said in her presentation.

The relatively low impact of fear of needles was unexpected, as fear of needles is considered a significant deterrent to epinephrine use, Chow told this news organization. “However, our respondents were more inclined to acknowledge a reluctance to escalate to emergency response as the major barrier to treatment,” she said.

The survey also asked patients what features of an anaphylaxis action plan would be most helpful. A majority of respondents (93%) rated a section for the management of mild (non-anaphylactic) allergic reaction symptoms as somewhat or very important. Visual aids for injection of epinephrine and visuals of anaphylaxis symptoms also ranked as somewhat or very important for 87.6% and 81% of respondents, respectively.

The study highlights the importance of educating allergy patients on recognizing and treating anaphylaxis and demonstrates that visuals were preferred in this survey population, Chow said. “Most patients and caregivers from our surveyed population report knowing how to treat anaphylaxis, but many would not use epinephrine as the first treatment,” she noted.

“The study focused on a single community clinic, and it would be beneficial to gather feedback from patients and caregivers representing a wider variety of educational, cultural, social, and socioeconomic backgrounds,” Chow told this news organization. “Additionally, input from other stakeholders, such as school nurses, would enhance knowledge,” she said.
 

Clinical Anaphylaxis Protocols Fall Short

A second study presented at the meeting showed the need to improve anaphylaxis education for clinicians.

Discrepancies in anaphylaxis management include variations in the definition and treatment of the condition, according to Carly Gunderson, DO, of Memorial Healthcare System, Pembroke Pines, Florida, who presented the study at the meeting.

“So often, we see patients in our office with a history of symptoms that meet criteria for anaphylaxis, yet when they call 911 and emergency medical services (EMS) arrive, they never receive epinephrine,” Gunderson said in an interview. “They receive antihistamines, steroids, everything except epinephrine, which is incredibly concerning given that epinephrine is always the first-line treatment for anaphylaxis,” she said.

“Because EMS providers are often the first healthcare professionals to assess patients experiencing anaphylaxis, their ability to recognize and appropriately treat anaphylaxis is essential,” Gunderson emphasized.

Gunderson and colleagues analyzed data from 30 states with mandatory Advanced Cardiac Life Support protocols to identify gaps in recognizing anaphylaxis and areas for improvement in prehospital management.

Only 15 states (50%) included gastrointestinal symptoms in the definition of anaphylaxis, 40% included neurologic manifestations, and 47% used a two-organ system definition, Gunderson noted in her presentation.

All 30 state protocols recommended diphenhydramine and epinephrine for anaphylactic reactions, 90% recommended albuterol if respiratory symptoms were present, 73% recommended intravenous fluids, and 60% recommended steroids. All but one of the state protocols listed epinephrine as the first-line recommendation for anaphylaxis; 25 states allowed epinephrine autoinjectors and 17 provided autoinjectors.

“We were shocked by how many protocols didn’t include gastrointestinal (abdominal pain, vomiting) or neurologic (lethargy, altered mental status) manifestations, when these are common presenting symptoms of anaphylaxis,” Gunderson told this news organization.

“We were also disappointed by how many protocols continue to recommend outdated interventions such as first-generation antihistamines and corticosteroids in the treatment of anaphylaxis,” she said.

Although anaphylaxis management has come a long way, the current study suggests that there is clearly room for improvement in the education of healthcare providers on how to identify and treat anaphylaxis, said Gunderson. “Most people think of anaphylaxis as the typical ‘face swelling up, throat closing’ type of reaction, which it can be, but in reality, there are so many other ways that it can present,” she said. “Healthcare providers must be aware of all of these possible manifestations so that we can treat in a timely manner to improve outcomes,” she added.

Limitations of the study included the focus only on states with mandatory or model EMS protocols, Gunderson told this news organization. As for additional research, the most important next steps are practical ones, namely, identifying ways to realistically implement necessary protocol changes, she said.
 

Real-World Data Support Need for Education

Real-world studies are important to identify current practice and opportunities for improvement, S. Shahzad Mustafa, MD, lead physician in allergy, immunology, and rheumatology at Rochester Regional Health and clinical associate professor of medicine at the University of Rochester School of Medicine and Dentistry, Rochester, New York, said in an interview.

“Management of anaphylaxis continues to evolve, and studies like these can help standardize evidence-based care across different medical settings, such as emergency medical services, urgent care, and emergency departments,” said Mustafa, who was not involved in either study.

The findings of the two studies were not unexpected, Mustafa said. “Heterogeneity in medical care is well recognized in numerous conditions, and anaphylaxis is no different. Patients and healthcare providers continue to have hesitation to use epinephrine and continue to overly rely on antihistamines and/or systemic steroids,” he noted.

For both studies, the takeaway message is that education is paramount to optimize anaphylaxis management, Mustafa told this news organization. “Education needs to focus on timely recognition of anaphylaxis, including atypical features such as gastrointestinal symptoms, and appropriate therapy with epinephrine,” he said.

Looking ahead, “research demonstrating differences in clinical outcomes with differing approaches to anaphylaxis may highlight the importance of early recognition and treatment with epinephrine,” said Mustafa. Management of anaphylaxis also lends itself to quality improvement studies, he added.

Neither of the studies received any outside funding. The researchers had no financial conflicts to disclose. Mustafa had no disclosures related to anaphylaxis but disclosed serving on the speakers’ bureau for Genentech, GSK, AstraZeneca, Regeneron/Sanofi, and CSL Behring and received grants from Takeda.
 

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

Misinformation and outdated protocols contribute to the suboptimal management of anaphylaxis by patients and healthcare professionals, based on data from two new studies presented at the American College of Allergy, Asthma and Immunology Annual Scientific Meeting.

Anaphylaxis can strike suddenly, and many patients and caregivers at risk do not know which symptoms to treat with epinephrine, said Joni Chow, DO, of Baylor College of Medicine, San Antonio, Texas, in her presentation at the meeting.

“Early identification of anaphylaxis and early intervention with epinephrine are critical for improving patient outcomes,” Chow said in an interview.

“Many allergic reactions occur in community settings, where written action plans serve to instruct patients and caregivers on how to recognize and respond to these emergencies,” she said. “Currently, anaphylaxis action plans are developed based on the consensus of healthcare professionals, with limited information available on the preferences of patients and caregivers,” she noted. However, even with action plans, many patients and families struggle to recognize and manage severe allergic reactions effectively, she added.

In response to this issue, Chow and colleagues created a survey designed to assess the understanding of anaphylaxis recognition and management by patients and caregivers and to identify their preferences regarding the elements included in the action plans.

In the study, Chow and colleagues surveyed 96 patients and caregivers in an allergy clinic waiting room. The majority (95%) of the patients were prescribed epinephrine. Although 73% said they were comfortable identifying signs of anaphylaxis, only 14% said they were likely to use epinephrine as a first-line treatment.

The most common reason given for avoiding epinephrine was uncertainty over which symptoms to treat (40.6%), followed by hesitancy to visit an emergency department (24%), hesitancy to call 911 (17.7%), uncertainty about how to use epinephrine auto-injectors (11.5%), and fear of needles (5.2%).

Although 85% of the respondents understood that antihistamine use does not prevent the need for epinephrine in cases of anaphylactic reactions, 23.7% said they would use an antihistamine as the first treatment in these cases.

For patients with rash and wheezing after a suspected allergen exposure, approximately two thirds (64.5%) of the respondents said they would inject epinephrine and 10.8% would drive to the emergency room before taking any action, Chow said in her presentation.

The relatively low impact of fear of needles was unexpected, as fear of needles is considered a significant deterrent to epinephrine use, Chow told this news organization. “However, our respondents were more inclined to acknowledge a reluctance to escalate to emergency response as the major barrier to treatment,” she said.

The survey also asked patients what features of an anaphylaxis action plan would be most helpful. A majority of respondents (93%) rated a section for the management of mild (non-anaphylactic) allergic reaction symptoms as somewhat or very important. Visual aids for injection of epinephrine and visuals of anaphylaxis symptoms also ranked as somewhat or very important for 87.6% and 81% of respondents, respectively.

The study highlights the importance of educating allergy patients on recognizing and treating anaphylaxis and demonstrates that visuals were preferred in this survey population, Chow said. “Most patients and caregivers from our surveyed population report knowing how to treat anaphylaxis, but many would not use epinephrine as the first treatment,” she noted.

“The study focused on a single community clinic, and it would be beneficial to gather feedback from patients and caregivers representing a wider variety of educational, cultural, social, and socioeconomic backgrounds,” Chow told this news organization. “Additionally, input from other stakeholders, such as school nurses, would enhance knowledge,” she said.
 

Clinical Anaphylaxis Protocols Fall Short

A second study presented at the meeting showed the need to improve anaphylaxis education for clinicians.

Discrepancies in anaphylaxis management include variations in the definition and treatment of the condition, according to Carly Gunderson, DO, of Memorial Healthcare System, Pembroke Pines, Florida, who presented the study at the meeting.

“So often, we see patients in our office with a history of symptoms that meet criteria for anaphylaxis, yet when they call 911 and emergency medical services (EMS) arrive, they never receive epinephrine,” Gunderson said in an interview. “They receive antihistamines, steroids, everything except epinephrine, which is incredibly concerning given that epinephrine is always the first-line treatment for anaphylaxis,” she said.

“Because EMS providers are often the first healthcare professionals to assess patients experiencing anaphylaxis, their ability to recognize and appropriately treat anaphylaxis is essential,” Gunderson emphasized.

Gunderson and colleagues analyzed data from 30 states with mandatory Advanced Cardiac Life Support protocols to identify gaps in recognizing anaphylaxis and areas for improvement in prehospital management.

Only 15 states (50%) included gastrointestinal symptoms in the definition of anaphylaxis, 40% included neurologic manifestations, and 47% used a two-organ system definition, Gunderson noted in her presentation.

All 30 state protocols recommended diphenhydramine and epinephrine for anaphylactic reactions, 90% recommended albuterol if respiratory symptoms were present, 73% recommended intravenous fluids, and 60% recommended steroids. All but one of the state protocols listed epinephrine as the first-line recommendation for anaphylaxis; 25 states allowed epinephrine autoinjectors and 17 provided autoinjectors.

“We were shocked by how many protocols didn’t include gastrointestinal (abdominal pain, vomiting) or neurologic (lethargy, altered mental status) manifestations, when these are common presenting symptoms of anaphylaxis,” Gunderson told this news organization.

“We were also disappointed by how many protocols continue to recommend outdated interventions such as first-generation antihistamines and corticosteroids in the treatment of anaphylaxis,” she said.

Although anaphylaxis management has come a long way, the current study suggests that there is clearly room for improvement in the education of healthcare providers on how to identify and treat anaphylaxis, said Gunderson. “Most people think of anaphylaxis as the typical ‘face swelling up, throat closing’ type of reaction, which it can be, but in reality, there are so many other ways that it can present,” she said. “Healthcare providers must be aware of all of these possible manifestations so that we can treat in a timely manner to improve outcomes,” she added.

Limitations of the study included the focus only on states with mandatory or model EMS protocols, Gunderson told this news organization. As for additional research, the most important next steps are practical ones, namely, identifying ways to realistically implement necessary protocol changes, she said.
 

Real-World Data Support Need for Education

Real-world studies are important to identify current practice and opportunities for improvement, S. Shahzad Mustafa, MD, lead physician in allergy, immunology, and rheumatology at Rochester Regional Health and clinical associate professor of medicine at the University of Rochester School of Medicine and Dentistry, Rochester, New York, said in an interview.

“Management of anaphylaxis continues to evolve, and studies like these can help standardize evidence-based care across different medical settings, such as emergency medical services, urgent care, and emergency departments,” said Mustafa, who was not involved in either study.

The findings of the two studies were not unexpected, Mustafa said. “Heterogeneity in medical care is well recognized in numerous conditions, and anaphylaxis is no different. Patients and healthcare providers continue to have hesitation to use epinephrine and continue to overly rely on antihistamines and/or systemic steroids,” he noted.

For both studies, the takeaway message is that education is paramount to optimize anaphylaxis management, Mustafa told this news organization. “Education needs to focus on timely recognition of anaphylaxis, including atypical features such as gastrointestinal symptoms, and appropriate therapy with epinephrine,” he said.

Looking ahead, “research demonstrating differences in clinical outcomes with differing approaches to anaphylaxis may highlight the importance of early recognition and treatment with epinephrine,” said Mustafa. Management of anaphylaxis also lends itself to quality improvement studies, he added.

Neither of the studies received any outside funding. The researchers had no financial conflicts to disclose. Mustafa had no disclosures related to anaphylaxis but disclosed serving on the speakers’ bureau for Genentech, GSK, AstraZeneca, Regeneron/Sanofi, and CSL Behring and received grants from Takeda.
 

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

CHICAGO — Patients who are new users of glucagon-like peptide 1 (GLP-1) receptor agonists have a low absolute risk of thyroid cancer, according to a new study presented at the annual meeting of the American Thyroid Association (ATA).

The study, presented by Juan Brito Campana, MBBS, of the Mayo Clinic in Rochester, Minnesota, used Medicare records to perform a secondary analysis of 41,000 adults with type 2 diabetes and moderate cardiovascular risk who were new users of GLP-1 receptor agonists, compared to users of other diabetes medications. 

“We took the innovative approach of applying the methodological rigor of a randomized clinical trial to the very large dataset of observational studies,” said Brito Campana.

The results showed a low absolute risk of thyroid cancer, with only 0.17% of patients in the GLP-1 group developing the disease. However, the data also showed a potential relative increase in risk during the first year of GLP-1 receptor agonist use. 

“This is likely due to increased detection rather than true incidence, as the latency period for thyroid cancer development is typically longer,” Brito Campana said. 

“We also note the limitations of the observational study design, including the short follow-up period and lack of detailed histological data. However, we believe the benefits of GLP-1 receptor agonists likely outweigh the risk of thyroid cancer.”
 

Malignancy in Bethesda III and IV Thyroid Nodules

At the same ATA session, Sapir Nachum Goldberg, MD, of the University of Pennsylvania, Philadelphia, presented the results of a retrospective record review that examined the prevalence of malignancy in Bethesda III and IV thyroid nodules with negative Thyrogen Receptor Signaling (ThyroSeq) version 3 molecular testing results.

Goldberg reported that 87% of patients with ThyroSeq negative subtype results were managed nonoperatively. “Based on our data, the true prevalence of malignancy likely lies between our low and high estimates of 3% and 23%,” she said. “We believe that the prevalence of malignancy may be higher in real-world practice than validation studies.”

Additionally, nodules with “currently negative” or “negative but limited” ThyroSeq results had a higher prevalence of malignancy (7%), compared with those with a “negative” result (2%). Factors like immediate vs delayed surgery, nodule size, and ultrasound pattern did not significantly impact malignancy prevalence.

The study results also indicated that surveillance ultrasonography is not routinely performed in up to one-third of patients, Goldberg said.

She closed by suggesting that colleagues consider the negative subtype in clinical decision-making. For “negative but limited” nodules, repeat the fine needle aspiration and, for “negative” and “currently negative” nodules, consider ultrasound follow-up as per ATA guidelines for Bethesda II cytology, she said.
 

RET-Mutated Medullary Thyroid Cancer

For patients with RET-mutated medullary thyroid cancer, Julien Hadoux, MD, PhD, of Institut de Cancérologie Gustave Roussy, Villejuif, France, presented a combined analysis of the efficacy of the RET inhibitor selpercatinib from the phase 1/2 LIBRETTO-001 and phase 3 LIBRETTO-531 trials.

This post hoc analysis used a combined cohort of 509 patients with RET-mutated advanced or metastatic medullary thyroid cancer who had received selpercatinib in the two trials.

Hadoux reported that robust and durable responses were seen across all mutation groups, including M918T, extracellular cysteine, and an “other” group composed of various uncommon RET mutations. “The median [progression-free survival] PFS was not reached for either the M918T or extracellular groups and it was 51.4 months for the Other group,” he said. 

“Selpercatinib showed superior median PFS vs control, regardless of the RET mutation. This analysis constitutes the largest catalog of RET mutations in medullary thyroid cancers treated with RET-specific inhibitors.”
 

TRK-Fusion Differentiated Thyroid Cancer

Steven Waguespack, MD, of the University of Texas MD Anderson Cancer Center, Houston, shared updated efficacy and safety data from three phase 1/2 pooled clinical trials of the tropomyosin kinase receptor (TRK) inhibitor larotrectinib in thyroid cancer. These data updated results initially published in 2022.

“Larotrectinib continues to demonstrate rapid and durable responses, extended survival, and offers a favorable safety profile in patients with TRK fusion differentiated thyroid cancer, with limited activity in anaplastic thyroid cancer,” Waguespack said. 

“Additionally, in a subset of patients, we identified some acquired on-target NTRK mutations and off-target GNAS and TP53 mutations that may give further insight into mechanisms of resistance.”

The primary endpoint was the investigator-assessed objective response rate (ORR); at 48 months, the ORR was 79% by independent review. The median PFS in patients with TRK fusion differentiated thyroid cancer was 44 months, while the median duration of response was 41 months. The 4-year overall survival rate was 86%.

Waguespack closed with a cautionary note to colleagues: “While circulating tumor DNA next-generation sequencing (NGS) analysis can be used to test for NTRK gene fusions, negative results should be followed up with tissue-based NGS,” he said.

Brito Campana and Goldberg disclosed no relevant financial relationships. Hadoux reported receiving honoraria for speaker engagements, advisory roles, or funding for CME from Eli Lilly, AAA, IPSEN, Roche, Pharma Mar, and EISAI, and research grants from Novartis, Sanofi, and Eli Lilly.

A version of this article appeared on Medscape.com.

CHICAGO — Patients who are new users of glucagon-like peptide 1 (GLP-1) receptor agonists have a low absolute risk of thyroid cancer, according to a new study presented at the annual meeting of the American Thyroid Association (ATA).

The study, presented by Juan Brito Campana, MBBS, of the Mayo Clinic in Rochester, Minnesota, used Medicare records to perform a secondary analysis of 41,000 adults with type 2 diabetes and moderate cardiovascular risk who were new users of GLP-1 receptor agonists, compared to users of other diabetes medications. 

“We took the innovative approach of applying the methodological rigor of a randomized clinical trial to the very large dataset of observational studies,” said Brito Campana.

The results showed a low absolute risk of thyroid cancer, with only 0.17% of patients in the GLP-1 group developing the disease. However, the data also showed a potential relative increase in risk during the first year of GLP-1 receptor agonist use. 

“This is likely due to increased detection rather than true incidence, as the latency period for thyroid cancer development is typically longer,” Brito Campana said. 

“We also note the limitations of the observational study design, including the short follow-up period and lack of detailed histological data. However, we believe the benefits of GLP-1 receptor agonists likely outweigh the risk of thyroid cancer.”
 

Malignancy in Bethesda III and IV Thyroid Nodules

At the same ATA session, Sapir Nachum Goldberg, MD, of the University of Pennsylvania, Philadelphia, presented the results of a retrospective record review that examined the prevalence of malignancy in Bethesda III and IV thyroid nodules with negative Thyrogen Receptor Signaling (ThyroSeq) version 3 molecular testing results.

Goldberg reported that 87% of patients with ThyroSeq negative subtype results were managed nonoperatively. “Based on our data, the true prevalence of malignancy likely lies between our low and high estimates of 3% and 23%,” she said. “We believe that the prevalence of malignancy may be higher in real-world practice than validation studies.”

Additionally, nodules with “currently negative” or “negative but limited” ThyroSeq results had a higher prevalence of malignancy (7%), compared with those with a “negative” result (2%). Factors like immediate vs delayed surgery, nodule size, and ultrasound pattern did not significantly impact malignancy prevalence.

The study results also indicated that surveillance ultrasonography is not routinely performed in up to one-third of patients, Goldberg said.

She closed by suggesting that colleagues consider the negative subtype in clinical decision-making. For “negative but limited” nodules, repeat the fine needle aspiration and, for “negative” and “currently negative” nodules, consider ultrasound follow-up as per ATA guidelines for Bethesda II cytology, she said.
 

RET-Mutated Medullary Thyroid Cancer

For patients with RET-mutated medullary thyroid cancer, Julien Hadoux, MD, PhD, of Institut de Cancérologie Gustave Roussy, Villejuif, France, presented a combined analysis of the efficacy of the RET inhibitor selpercatinib from the phase 1/2 LIBRETTO-001 and phase 3 LIBRETTO-531 trials.

This post hoc analysis used a combined cohort of 509 patients with RET-mutated advanced or metastatic medullary thyroid cancer who had received selpercatinib in the two trials.

Hadoux reported that robust and durable responses were seen across all mutation groups, including M918T, extracellular cysteine, and an “other” group composed of various uncommon RET mutations. “The median [progression-free survival] PFS was not reached for either the M918T or extracellular groups and it was 51.4 months for the Other group,” he said. 

“Selpercatinib showed superior median PFS vs control, regardless of the RET mutation. This analysis constitutes the largest catalog of RET mutations in medullary thyroid cancers treated with RET-specific inhibitors.”
 

TRK-Fusion Differentiated Thyroid Cancer

Steven Waguespack, MD, of the University of Texas MD Anderson Cancer Center, Houston, shared updated efficacy and safety data from three phase 1/2 pooled clinical trials of the tropomyosin kinase receptor (TRK) inhibitor larotrectinib in thyroid cancer. These data updated results initially published in 2022.

“Larotrectinib continues to demonstrate rapid and durable responses, extended survival, and offers a favorable safety profile in patients with TRK fusion differentiated thyroid cancer, with limited activity in anaplastic thyroid cancer,” Waguespack said. 

“Additionally, in a subset of patients, we identified some acquired on-target NTRK mutations and off-target GNAS and TP53 mutations that may give further insight into mechanisms of resistance.”

The primary endpoint was the investigator-assessed objective response rate (ORR); at 48 months, the ORR was 79% by independent review. The median PFS in patients with TRK fusion differentiated thyroid cancer was 44 months, while the median duration of response was 41 months. The 4-year overall survival rate was 86%.

Waguespack closed with a cautionary note to colleagues: “While circulating tumor DNA next-generation sequencing (NGS) analysis can be used to test for NTRK gene fusions, negative results should be followed up with tissue-based NGS,” he said.

Brito Campana and Goldberg disclosed no relevant financial relationships. Hadoux reported receiving honoraria for speaker engagements, advisory roles, or funding for CME from Eli Lilly, AAA, IPSEN, Roche, Pharma Mar, and EISAI, and research grants from Novartis, Sanofi, and Eli Lilly.

A version of this article appeared on Medscape.com.

CHICAGO — Patients who are new users of glucagon-like peptide 1 (GLP-1) receptor agonists have a low absolute risk of thyroid cancer, according to a new study presented at the annual meeting of the American Thyroid Association (ATA).

The study, presented by Juan Brito Campana, MBBS, of the Mayo Clinic in Rochester, Minnesota, used Medicare records to perform a secondary analysis of 41,000 adults with type 2 diabetes and moderate cardiovascular risk who were new users of GLP-1 receptor agonists, compared to users of other diabetes medications. 

“We took the innovative approach of applying the methodological rigor of a randomized clinical trial to the very large dataset of observational studies,” said Brito Campana.

The results showed a low absolute risk of thyroid cancer, with only 0.17% of patients in the GLP-1 group developing the disease. However, the data also showed a potential relative increase in risk during the first year of GLP-1 receptor agonist use. 

“This is likely due to increased detection rather than true incidence, as the latency period for thyroid cancer development is typically longer,” Brito Campana said. 

“We also note the limitations of the observational study design, including the short follow-up period and lack of detailed histological data. However, we believe the benefits of GLP-1 receptor agonists likely outweigh the risk of thyroid cancer.”
 

Malignancy in Bethesda III and IV Thyroid Nodules

At the same ATA session, Sapir Nachum Goldberg, MD, of the University of Pennsylvania, Philadelphia, presented the results of a retrospective record review that examined the prevalence of malignancy in Bethesda III and IV thyroid nodules with negative Thyrogen Receptor Signaling (ThyroSeq) version 3 molecular testing results.

Goldberg reported that 87% of patients with ThyroSeq negative subtype results were managed nonoperatively. “Based on our data, the true prevalence of malignancy likely lies between our low and high estimates of 3% and 23%,” she said. “We believe that the prevalence of malignancy may be higher in real-world practice than validation studies.”

Additionally, nodules with “currently negative” or “negative but limited” ThyroSeq results had a higher prevalence of malignancy (7%), compared with those with a “negative” result (2%). Factors like immediate vs delayed surgery, nodule size, and ultrasound pattern did not significantly impact malignancy prevalence.

The study results also indicated that surveillance ultrasonography is not routinely performed in up to one-third of patients, Goldberg said.

She closed by suggesting that colleagues consider the negative subtype in clinical decision-making. For “negative but limited” nodules, repeat the fine needle aspiration and, for “negative” and “currently negative” nodules, consider ultrasound follow-up as per ATA guidelines for Bethesda II cytology, she said.
 

RET-Mutated Medullary Thyroid Cancer

For patients with RET-mutated medullary thyroid cancer, Julien Hadoux, MD, PhD, of Institut de Cancérologie Gustave Roussy, Villejuif, France, presented a combined analysis of the efficacy of the RET inhibitor selpercatinib from the phase 1/2 LIBRETTO-001 and phase 3 LIBRETTO-531 trials.

This post hoc analysis used a combined cohort of 509 patients with RET-mutated advanced or metastatic medullary thyroid cancer who had received selpercatinib in the two trials.

Hadoux reported that robust and durable responses were seen across all mutation groups, including M918T, extracellular cysteine, and an “other” group composed of various uncommon RET mutations. “The median [progression-free survival] PFS was not reached for either the M918T or extracellular groups and it was 51.4 months for the Other group,” he said. 

“Selpercatinib showed superior median PFS vs control, regardless of the RET mutation. This analysis constitutes the largest catalog of RET mutations in medullary thyroid cancers treated with RET-specific inhibitors.”
 

TRK-Fusion Differentiated Thyroid Cancer

Steven Waguespack, MD, of the University of Texas MD Anderson Cancer Center, Houston, shared updated efficacy and safety data from three phase 1/2 pooled clinical trials of the tropomyosin kinase receptor (TRK) inhibitor larotrectinib in thyroid cancer. These data updated results initially published in 2022.

“Larotrectinib continues to demonstrate rapid and durable responses, extended survival, and offers a favorable safety profile in patients with TRK fusion differentiated thyroid cancer, with limited activity in anaplastic thyroid cancer,” Waguespack said. 

“Additionally, in a subset of patients, we identified some acquired on-target NTRK mutations and off-target GNAS and TP53 mutations that may give further insight into mechanisms of resistance.”

The primary endpoint was the investigator-assessed objective response rate (ORR); at 48 months, the ORR was 79% by independent review. The median PFS in patients with TRK fusion differentiated thyroid cancer was 44 months, while the median duration of response was 41 months. The 4-year overall survival rate was 86%.

Waguespack closed with a cautionary note to colleagues: “While circulating tumor DNA next-generation sequencing (NGS) analysis can be used to test for NTRK gene fusions, negative results should be followed up with tissue-based NGS,” he said.

Brito Campana and Goldberg disclosed no relevant financial relationships. Hadoux reported receiving honoraria for speaker engagements, advisory roles, or funding for CME from Eli Lilly, AAA, IPSEN, Roche, Pharma Mar, and EISAI, and research grants from Novartis, Sanofi, and Eli Lilly.

A version of this article appeared on Medscape.com.

BOSTON — Social adversity is associated with worse survival among patients with pulmonary hypertension (PH), according to a new retrospective study of a New York City population. Among HIV+ patients with heart failure, PH was associated with about a threefold increase in all-cause mortality, but that risk increased to about sevenfold when social adversity, identified by a licensed social worker, was also present.

A sub-analysis of both HIV+ and HIV– patients showed worse mortality outcomes with social adversity in both groups.

“Almost the majority of patients that we treat have either some social adversity or no insurance or are undocumented, so as a group of residents, we decided to study the impact of these factors on their health and the care that can be provided. We started using the two cohorts and now we keep it going with every new resident,” said Luca Biavati, MD, who presented the study at the CHEST Annual Meeting.

“The presence of any form of socioeconomic disadvantage is negatively impacting care and for a large part of the population, there are some factors that could probably be addressed by either an institutional or hospital policy,” said Dr. Biavati, who is an internal medicine resident at Jacobi Medical Center, New York.

Other factors are more difficult to address, such as lack of education. “[Some patients] don’t understand the gravity of their issue and medical condition until it’s too late, and then they’re not fit enough for the treatment, or just because of the social situation, they cannot qualify for advanced therapies,” said Dr. Biavati.

The researchers established two cohorts: One consisting of patients with HIV and heart failure who may or may not have had PH and one comprising patients with PH with or without HIV and heart failure. In the HIV/heart failure group, PH without social adversity was associated with a nearly threefold increase in all-cause mortality (hazard ratio [HR], 2.83; P = .004), whereas PH with social adversity was linked to a more than sevenfold increase in all-cause mortality (HR, 7.14; P < .001). Social adversity without PA was associated with a more than fourfold increase (HR, 4.47; P < .001).

Within the PH cohort, social adversity was associated with lower survival (P < .001). When the researchers broke down the results by types of social adversity, they found statistically significant relationships between greater mortality risk and economic instability within the HIV+ population (HR, 2.59; P = .040), transportation issues within the HIV– population (HR, 12.8; P < .001), and lack of social or family support within both the HIV– (HR, 5.49; P < .001) and the HIV+ population (HR, 2.03; P = .028). 

The research has prompted interventions, which are now being studied at the institution, according to Dr. Biavati. “We have a policy of giving medications in bags when we discharge a patient with a social adversity. We literally go to the pharmacy, bring up the bag of medication, and we [put it] in their hands before they leave the hospital. They get a 1- or 3-month supply, depending on the medication, and then we usually discharge them with a clinical appointment already scheduled with either a pulmonary or primary care provider, and we usually call them before every appointment to confirm that they’re coming. That increases the chances of some success, but there’s still a very long way to go,” said Dr. Biavati.

Dr. Biavati was blinded to the results of the intervention, so he could not report on whether it was working. “But I can tell you that I’ve had busier clinics, so hopefully that means that they’re showing up more,” he said.

The problem is complex, according to Sandeep Jain, MD, who moderated the session. “Social adversity means lack of education. Lack of education means lack of compliance. Lack of compliance means what can you do if people are not taking medications? So it’s all matched together. It’s all lack of education and lack of money, lack of family support. And these drugs they have to take every single day. It’s not that easy. It’s very easy for us to say I had antiretroviral treatment for 6 months. It is almost impossible to continue regular treatment for that long [for a patient with social adversity]. You can’t blame them if they aren’t taking treatments. It’s very difficult for them,” said Dr. Jain.

That underscores the need for interventions that can address the needs of patients with social adversity. “We have to [practice] medicine considering the social situation of the patient and not just the medicine that we study in books. That’s kind of what we are faced with every day. We have therapies, and then life happens. It’s much harder to care for those patients,” said Dr. Biavati.

Dr. Biavati and Dr. Jain reported no relevant financial relationships.
 

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

BOSTON — Social adversity is associated with worse survival among patients with pulmonary hypertension (PH), according to a new retrospective study of a New York City population. Among HIV+ patients with heart failure, PH was associated with about a threefold increase in all-cause mortality, but that risk increased to about sevenfold when social adversity, identified by a licensed social worker, was also present.

A sub-analysis of both HIV+ and HIV– patients showed worse mortality outcomes with social adversity in both groups.

“Almost the majority of patients that we treat have either some social adversity or no insurance or are undocumented, so as a group of residents, we decided to study the impact of these factors on their health and the care that can be provided. We started using the two cohorts and now we keep it going with every new resident,” said Luca Biavati, MD, who presented the study at the CHEST Annual Meeting.

“The presence of any form of socioeconomic disadvantage is negatively impacting care and for a large part of the population, there are some factors that could probably be addressed by either an institutional or hospital policy,” said Dr. Biavati, who is an internal medicine resident at Jacobi Medical Center, New York.

Other factors are more difficult to address, such as lack of education. “[Some patients] don’t understand the gravity of their issue and medical condition until it’s too late, and then they’re not fit enough for the treatment, or just because of the social situation, they cannot qualify for advanced therapies,” said Dr. Biavati.

The researchers established two cohorts: One consisting of patients with HIV and heart failure who may or may not have had PH and one comprising patients with PH with or without HIV and heart failure. In the HIV/heart failure group, PH without social adversity was associated with a nearly threefold increase in all-cause mortality (hazard ratio [HR], 2.83; P = .004), whereas PH with social adversity was linked to a more than sevenfold increase in all-cause mortality (HR, 7.14; P < .001). Social adversity without PA was associated with a more than fourfold increase (HR, 4.47; P < .001).

Within the PH cohort, social adversity was associated with lower survival (P < .001). When the researchers broke down the results by types of social adversity, they found statistically significant relationships between greater mortality risk and economic instability within the HIV+ population (HR, 2.59; P = .040), transportation issues within the HIV– population (HR, 12.8; P < .001), and lack of social or family support within both the HIV– (HR, 5.49; P < .001) and the HIV+ population (HR, 2.03; P = .028). 

The research has prompted interventions, which are now being studied at the institution, according to Dr. Biavati. “We have a policy of giving medications in bags when we discharge a patient with a social adversity. We literally go to the pharmacy, bring up the bag of medication, and we [put it] in their hands before they leave the hospital. They get a 1- or 3-month supply, depending on the medication, and then we usually discharge them with a clinical appointment already scheduled with either a pulmonary or primary care provider, and we usually call them before every appointment to confirm that they’re coming. That increases the chances of some success, but there’s still a very long way to go,” said Dr. Biavati.

Dr. Biavati was blinded to the results of the intervention, so he could not report on whether it was working. “But I can tell you that I’ve had busier clinics, so hopefully that means that they’re showing up more,” he said.

The problem is complex, according to Sandeep Jain, MD, who moderated the session. “Social adversity means lack of education. Lack of education means lack of compliance. Lack of compliance means what can you do if people are not taking medications? So it’s all matched together. It’s all lack of education and lack of money, lack of family support. And these drugs they have to take every single day. It’s not that easy. It’s very easy for us to say I had antiretroviral treatment for 6 months. It is almost impossible to continue regular treatment for that long [for a patient with social adversity]. You can’t blame them if they aren’t taking treatments. It’s very difficult for them,” said Dr. Jain.

That underscores the need for interventions that can address the needs of patients with social adversity. “We have to [practice] medicine considering the social situation of the patient and not just the medicine that we study in books. That’s kind of what we are faced with every day. We have therapies, and then life happens. It’s much harder to care for those patients,” said Dr. Biavati.

Dr. Biavati and Dr. Jain reported no relevant financial relationships.
 

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

BOSTON — Social adversity is associated with worse survival among patients with pulmonary hypertension (PH), according to a new retrospective study of a New York City population. Among HIV+ patients with heart failure, PH was associated with about a threefold increase in all-cause mortality, but that risk increased to about sevenfold when social adversity, identified by a licensed social worker, was also present.

A sub-analysis of both HIV+ and HIV– patients showed worse mortality outcomes with social adversity in both groups.

“Almost the majority of patients that we treat have either some social adversity or no insurance or are undocumented, so as a group of residents, we decided to study the impact of these factors on their health and the care that can be provided. We started using the two cohorts and now we keep it going with every new resident,” said Luca Biavati, MD, who presented the study at the CHEST Annual Meeting.

“The presence of any form of socioeconomic disadvantage is negatively impacting care and for a large part of the population, there are some factors that could probably be addressed by either an institutional or hospital policy,” said Dr. Biavati, who is an internal medicine resident at Jacobi Medical Center, New York.

Other factors are more difficult to address, such as lack of education. “[Some patients] don’t understand the gravity of their issue and medical condition until it’s too late, and then they’re not fit enough for the treatment, or just because of the social situation, they cannot qualify for advanced therapies,” said Dr. Biavati.

The researchers established two cohorts: One consisting of patients with HIV and heart failure who may or may not have had PH and one comprising patients with PH with or without HIV and heart failure. In the HIV/heart failure group, PH without social adversity was associated with a nearly threefold increase in all-cause mortality (hazard ratio [HR], 2.83; P = .004), whereas PH with social adversity was linked to a more than sevenfold increase in all-cause mortality (HR, 7.14; P < .001). Social adversity without PA was associated with a more than fourfold increase (HR, 4.47; P < .001).

Within the PH cohort, social adversity was associated with lower survival (P < .001). When the researchers broke down the results by types of social adversity, they found statistically significant relationships between greater mortality risk and economic instability within the HIV+ population (HR, 2.59; P = .040), transportation issues within the HIV– population (HR, 12.8; P < .001), and lack of social or family support within both the HIV– (HR, 5.49; P < .001) and the HIV+ population (HR, 2.03; P = .028). 

The research has prompted interventions, which are now being studied at the institution, according to Dr. Biavati. “We have a policy of giving medications in bags when we discharge a patient with a social adversity. We literally go to the pharmacy, bring up the bag of medication, and we [put it] in their hands before they leave the hospital. They get a 1- or 3-month supply, depending on the medication, and then we usually discharge them with a clinical appointment already scheduled with either a pulmonary or primary care provider, and we usually call them before every appointment to confirm that they’re coming. That increases the chances of some success, but there’s still a very long way to go,” said Dr. Biavati.

Dr. Biavati was blinded to the results of the intervention, so he could not report on whether it was working. “But I can tell you that I’ve had busier clinics, so hopefully that means that they’re showing up more,” he said.

The problem is complex, according to Sandeep Jain, MD, who moderated the session. “Social adversity means lack of education. Lack of education means lack of compliance. Lack of compliance means what can you do if people are not taking medications? So it’s all matched together. It’s all lack of education and lack of money, lack of family support. And these drugs they have to take every single day. It’s not that easy. It’s very easy for us to say I had antiretroviral treatment for 6 months. It is almost impossible to continue regular treatment for that long [for a patient with social adversity]. You can’t blame them if they aren’t taking treatments. It’s very difficult for them,” said Dr. Jain.

That underscores the need for interventions that can address the needs of patients with social adversity. “We have to [practice] medicine considering the social situation of the patient and not just the medicine that we study in books. That’s kind of what we are faced with every day. We have therapies, and then life happens. It’s much harder to care for those patients,” said Dr. Biavati.

Dr. Biavati and Dr. Jain reported no relevant financial relationships.
 

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

Approximately one third of parents of children with atopic dermatitis reported little or no improvement with elimination diets, and nearly 80% reintroduced the eliminated foods, based on survey data from nearly 300 parents.

Although atopic dermatitis can be associated with an increased risk for food allergies, major allergy organizations do not currently recommend elimination diets as a treatment for atopic dermatitis, said Nadia Makkoukdji, MD, a pediatrician at Jackson Memorial Hospital, Miami, in a presentation at the American College of Allergy, Asthma, and Immunology (ACAAI) Annual Scientific Meeting.

“A fear of drastic dietary changes often prevents families from seeking the care their children need,” Makkoukdji said in an interview. In the clinical setting, Makkoukdji noted that she has seen many patients who have started food elimination diets on their own or as recommended by other doctors, and that these diets can lead to dangers such as the development of immunoglobulin E–mediated food allergies on reintroduction of eliminated foods and malnutrition. They can also produce “emotional stress in children and anxiety or depression, while also adding stress to parents and the entire family.”

Makkoukdji conducted the study to explore parents’ perceptions of these diets in management of their children’s atopic dermatitis, she said.

In the study, Makkoukdji and colleagues sought to understand parents’ perceptions of the role of diet in atopic dermatitis in their children. The researchers reviewed surveys from 298 parents of children with atopic dermatitis who were seen at a single academic center. Parents completed the surveys in the emergency department or in an allergy, dermatology, and general pediatrics clinic.

Overall, 42% of parents identified food triggers for their child’s atopic dermatitis. The most commonly identified triggers were milk (32%), tree nuts/seeds/peanuts (16%), and eggs (11%).

Of the parents who reported food triggers, 23% removed the suspected trigger food from the child’s diet completely, 20% removed suspected trigger foods from their own diets while breastfeeding, and 19% changed their infant’s formula.

In the wake of the elimination diets, 38% of the parents reported no improvement in their child’s atopic dermatitis, 35% reported a 25% improvement, and 9% reported complete resolution. The majority (79%) reintroduced eliminated foods and reported no recurrence of atopic dermatitis symptoms.

The researchers were surprised by how many parents changed their child’s diet in the belief that certain foods exacerbated their child’s atopic dermatitis, “although this perception aligns with the common concern that food allergens can trigger or worsen atopic dermatitis flares,” Makkoukdji said.

The current study highlights the need for more awareness of the limited impact of dietary modifications on atopic dermatitis in the absence of confirmed food allergies, Makkoukdji said. “Our study shows that food elimination diets are still commonly being used by parents in the local Miami population.”

The findings were limited by several factors, including the use of data from a single center and the focus only on pediatric patients, but the primary goal was to assess parental perceptions of AD flares in relation to dietary choices, said Makkoukdji. “Future studies that include larger and more diverse populations would be valuable for the field.”
 

Dietary Modifications Don’t Live Up to Hype

“Food continues to be one of the most discussed aspects of atopic dermatitis,” Peter Lio, MD, clinical assistant professor of dermatology and pediatrics at Northwestern University Feinberg School of Medicine, Chicago, Illinois, said in an interview.

“Almost all of my patients and families ask about dietary modifications, even though almost all of them have experimented with it to some degree,” said Lio. In his experience, diet plays a small role, if any, in the day-to-day management of atopic dermatitis.

This lack of effect of dietary changes is often frustrating to patients because of the persistent “common wisdom” that points to diet as a root cause of atopic dermatitis, Lio said. “Many practitioners continue to recommend excluding foods such as gluten or dairy from the diet, but generally these are only of modest help,” and although patients wish that dietary changes would fix the problem, most are left wondering why these changes didn’t help them.

The current study findings “reflect my own experience after nearly 20 years of being deeply immersed in the world of atopic dermatitis,” Lio said. Although the takeaway message does not argue against eating healthy foods, some foods do seem to make AD worse in some patients and may have nonallergic pro-inflammatory effects.

“In those cases, it is reasonable to limit or avoid those foods. However, it is extremely difficult to tell what food or foods are driving flare-ups when things are out of control, so dietary modification is generally not the best place to start,” he said.

True food allergies are much more common in patients with atopic dermatitis compared with individuals without atopic dermatitis, but the current study is not addressing these types of allergies, Lio emphasized. “If someone has true allergy to peanuts, for example, they should not be eating them; we also know that they are not ‘cheating’ because these patients would not merely have an eczema flare; they would have urticaria, angioedema, or anaphylaxis. There is tremendous confusion around this point and lots of confusion around allergy testing and its limitations.”

In addition, patients with atopic dermatitis are more likely than those without atopic dermatitis to have abnormalities in the gut microbiome and gut barrier, Lio said.

Abnormalities in the gut microbiome are different from the concept of allergy and may fall into the more complex category of barrier and microbiome disruptors, he said. Therefore, “the food category may not be nearly as important as the specific preparation of the food along with the additives (such as preservatives and emulsifiers) that may actually be driving the problem.”

Although in the past many clinicians advised patients to try cutting out certain foods to see whether atopic dermatitis symptoms improved, this strategy is not without risk, said Lio. “There have been incredible advancements in understanding the role of the gut in tolerization to foods.” Recent research has shown that by eating foods regularly, particularly those such as peanuts that seem to have more allergic potential, the body becomes tolerant, and this prevents the development of true food allergies.

As for additional research, many questions remain about the effects of types of foods, processing methods, and timing of introduction of foods on atopic dermatitis, Lio noted.

“Atopic dermatitis is a systemic condition with the immune system, with the skin/gut/respiratory barriers and microbiome involved; I think we now have a broader view of how big and complex the landscape really is,” he said.

The study received no outside funding. The researchers had no financial conflicts to disclose. Lio had no disclosures relevant to elimination diets but disclosed serving on the speakers bureau for AbbVie, Arcutis Biotherapeutics, Eli Lilly, Galderma, Hyphens Pharma, Incyte, La Roche–Posay/L’Oréal, Pfizer, Pierre Fabre Dermatologie, Regeneron/Sanofi Genzyme, and Verrica Pharmaceuticals; serving on consulting/advisory boards; or having stock options for many pharmaceutical companies. Lio also disclosed a patent pending for a Theraplex product with royalties paid and is a board member and Scientific Advisory Committee member emeritus of the National Eczema Association.

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

Approximately one third of parents of children with atopic dermatitis reported little or no improvement with elimination diets, and nearly 80% reintroduced the eliminated foods, based on survey data from nearly 300 parents.

Although atopic dermatitis can be associated with an increased risk for food allergies, major allergy organizations do not currently recommend elimination diets as a treatment for atopic dermatitis, said Nadia Makkoukdji, MD, a pediatrician at Jackson Memorial Hospital, Miami, in a presentation at the American College of Allergy, Asthma, and Immunology (ACAAI) Annual Scientific Meeting.

“A fear of drastic dietary changes often prevents families from seeking the care their children need,” Makkoukdji said in an interview. In the clinical setting, Makkoukdji noted that she has seen many patients who have started food elimination diets on their own or as recommended by other doctors, and that these diets can lead to dangers such as the development of immunoglobulin E–mediated food allergies on reintroduction of eliminated foods and malnutrition. They can also produce “emotional stress in children and anxiety or depression, while also adding stress to parents and the entire family.”

Makkoukdji conducted the study to explore parents’ perceptions of these diets in management of their children’s atopic dermatitis, she said.

In the study, Makkoukdji and colleagues sought to understand parents’ perceptions of the role of diet in atopic dermatitis in their children. The researchers reviewed surveys from 298 parents of children with atopic dermatitis who were seen at a single academic center. Parents completed the surveys in the emergency department or in an allergy, dermatology, and general pediatrics clinic.

Overall, 42% of parents identified food triggers for their child’s atopic dermatitis. The most commonly identified triggers were milk (32%), tree nuts/seeds/peanuts (16%), and eggs (11%).

Of the parents who reported food triggers, 23% removed the suspected trigger food from the child’s diet completely, 20% removed suspected trigger foods from their own diets while breastfeeding, and 19% changed their infant’s formula.

In the wake of the elimination diets, 38% of the parents reported no improvement in their child’s atopic dermatitis, 35% reported a 25% improvement, and 9% reported complete resolution. The majority (79%) reintroduced eliminated foods and reported no recurrence of atopic dermatitis symptoms.

The researchers were surprised by how many parents changed their child’s diet in the belief that certain foods exacerbated their child’s atopic dermatitis, “although this perception aligns with the common concern that food allergens can trigger or worsen atopic dermatitis flares,” Makkoukdji said.

The current study highlights the need for more awareness of the limited impact of dietary modifications on atopic dermatitis in the absence of confirmed food allergies, Makkoukdji said. “Our study shows that food elimination diets are still commonly being used by parents in the local Miami population.”

The findings were limited by several factors, including the use of data from a single center and the focus only on pediatric patients, but the primary goal was to assess parental perceptions of AD flares in relation to dietary choices, said Makkoukdji. “Future studies that include larger and more diverse populations would be valuable for the field.”
 

Dietary Modifications Don’t Live Up to Hype

“Food continues to be one of the most discussed aspects of atopic dermatitis,” Peter Lio, MD, clinical assistant professor of dermatology and pediatrics at Northwestern University Feinberg School of Medicine, Chicago, Illinois, said in an interview.

“Almost all of my patients and families ask about dietary modifications, even though almost all of them have experimented with it to some degree,” said Lio. In his experience, diet plays a small role, if any, in the day-to-day management of atopic dermatitis.

This lack of effect of dietary changes is often frustrating to patients because of the persistent “common wisdom” that points to diet as a root cause of atopic dermatitis, Lio said. “Many practitioners continue to recommend excluding foods such as gluten or dairy from the diet, but generally these are only of modest help,” and although patients wish that dietary changes would fix the problem, most are left wondering why these changes didn’t help them.

The current study findings “reflect my own experience after nearly 20 years of being deeply immersed in the world of atopic dermatitis,” Lio said. Although the takeaway message does not argue against eating healthy foods, some foods do seem to make AD worse in some patients and may have nonallergic pro-inflammatory effects.

“In those cases, it is reasonable to limit or avoid those foods. However, it is extremely difficult to tell what food or foods are driving flare-ups when things are out of control, so dietary modification is generally not the best place to start,” he said.

True food allergies are much more common in patients with atopic dermatitis compared with individuals without atopic dermatitis, but the current study is not addressing these types of allergies, Lio emphasized. “If someone has true allergy to peanuts, for example, they should not be eating them; we also know that they are not ‘cheating’ because these patients would not merely have an eczema flare; they would have urticaria, angioedema, or anaphylaxis. There is tremendous confusion around this point and lots of confusion around allergy testing and its limitations.”

In addition, patients with atopic dermatitis are more likely than those without atopic dermatitis to have abnormalities in the gut microbiome and gut barrier, Lio said.

Abnormalities in the gut microbiome are different from the concept of allergy and may fall into the more complex category of barrier and microbiome disruptors, he said. Therefore, “the food category may not be nearly as important as the specific preparation of the food along with the additives (such as preservatives and emulsifiers) that may actually be driving the problem.”

Although in the past many clinicians advised patients to try cutting out certain foods to see whether atopic dermatitis symptoms improved, this strategy is not without risk, said Lio. “There have been incredible advancements in understanding the role of the gut in tolerization to foods.” Recent research has shown that by eating foods regularly, particularly those such as peanuts that seem to have more allergic potential, the body becomes tolerant, and this prevents the development of true food allergies.

As for additional research, many questions remain about the effects of types of foods, processing methods, and timing of introduction of foods on atopic dermatitis, Lio noted.

“Atopic dermatitis is a systemic condition with the immune system, with the skin/gut/respiratory barriers and microbiome involved; I think we now have a broader view of how big and complex the landscape really is,” he said.

The study received no outside funding. The researchers had no financial conflicts to disclose. Lio had no disclosures relevant to elimination diets but disclosed serving on the speakers bureau for AbbVie, Arcutis Biotherapeutics, Eli Lilly, Galderma, Hyphens Pharma, Incyte, La Roche–Posay/L’Oréal, Pfizer, Pierre Fabre Dermatologie, Regeneron/Sanofi Genzyme, and Verrica Pharmaceuticals; serving on consulting/advisory boards; or having stock options for many pharmaceutical companies. Lio also disclosed a patent pending for a Theraplex product with royalties paid and is a board member and Scientific Advisory Committee member emeritus of the National Eczema Association.

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

Approximately one third of parents of children with atopic dermatitis reported little or no improvement with elimination diets, and nearly 80% reintroduced the eliminated foods, based on survey data from nearly 300 parents.

Although atopic dermatitis can be associated with an increased risk for food allergies, major allergy organizations do not currently recommend elimination diets as a treatment for atopic dermatitis, said Nadia Makkoukdji, MD, a pediatrician at Jackson Memorial Hospital, Miami, in a presentation at the American College of Allergy, Asthma, and Immunology (ACAAI) Annual Scientific Meeting.

“A fear of drastic dietary changes often prevents families from seeking the care their children need,” Makkoukdji said in an interview. In the clinical setting, Makkoukdji noted that she has seen many patients who have started food elimination diets on their own or as recommended by other doctors, and that these diets can lead to dangers such as the development of immunoglobulin E–mediated food allergies on reintroduction of eliminated foods and malnutrition. They can also produce “emotional stress in children and anxiety or depression, while also adding stress to parents and the entire family.”

Makkoukdji conducted the study to explore parents’ perceptions of these diets in management of their children’s atopic dermatitis, she said.

In the study, Makkoukdji and colleagues sought to understand parents’ perceptions of the role of diet in atopic dermatitis in their children. The researchers reviewed surveys from 298 parents of children with atopic dermatitis who were seen at a single academic center. Parents completed the surveys in the emergency department or in an allergy, dermatology, and general pediatrics clinic.

Overall, 42% of parents identified food triggers for their child’s atopic dermatitis. The most commonly identified triggers were milk (32%), tree nuts/seeds/peanuts (16%), and eggs (11%).

Of the parents who reported food triggers, 23% removed the suspected trigger food from the child’s diet completely, 20% removed suspected trigger foods from their own diets while breastfeeding, and 19% changed their infant’s formula.

In the wake of the elimination diets, 38% of the parents reported no improvement in their child’s atopic dermatitis, 35% reported a 25% improvement, and 9% reported complete resolution. The majority (79%) reintroduced eliminated foods and reported no recurrence of atopic dermatitis symptoms.

The researchers were surprised by how many parents changed their child’s diet in the belief that certain foods exacerbated their child’s atopic dermatitis, “although this perception aligns with the common concern that food allergens can trigger or worsen atopic dermatitis flares,” Makkoukdji said.

The current study highlights the need for more awareness of the limited impact of dietary modifications on atopic dermatitis in the absence of confirmed food allergies, Makkoukdji said. “Our study shows that food elimination diets are still commonly being used by parents in the local Miami population.”

The findings were limited by several factors, including the use of data from a single center and the focus only on pediatric patients, but the primary goal was to assess parental perceptions of AD flares in relation to dietary choices, said Makkoukdji. “Future studies that include larger and more diverse populations would be valuable for the field.”
 

Dietary Modifications Don’t Live Up to Hype

“Food continues to be one of the most discussed aspects of atopic dermatitis,” Peter Lio, MD, clinical assistant professor of dermatology and pediatrics at Northwestern University Feinberg School of Medicine, Chicago, Illinois, said in an interview.

“Almost all of my patients and families ask about dietary modifications, even though almost all of them have experimented with it to some degree,” said Lio. In his experience, diet plays a small role, if any, in the day-to-day management of atopic dermatitis.

This lack of effect of dietary changes is often frustrating to patients because of the persistent “common wisdom” that points to diet as a root cause of atopic dermatitis, Lio said. “Many practitioners continue to recommend excluding foods such as gluten or dairy from the diet, but generally these are only of modest help,” and although patients wish that dietary changes would fix the problem, most are left wondering why these changes didn’t help them.

The current study findings “reflect my own experience after nearly 20 years of being deeply immersed in the world of atopic dermatitis,” Lio said. Although the takeaway message does not argue against eating healthy foods, some foods do seem to make AD worse in some patients and may have nonallergic pro-inflammatory effects.

“In those cases, it is reasonable to limit or avoid those foods. However, it is extremely difficult to tell what food or foods are driving flare-ups when things are out of control, so dietary modification is generally not the best place to start,” he said.

True food allergies are much more common in patients with atopic dermatitis compared with individuals without atopic dermatitis, but the current study is not addressing these types of allergies, Lio emphasized. “If someone has true allergy to peanuts, for example, they should not be eating them; we also know that they are not ‘cheating’ because these patients would not merely have an eczema flare; they would have urticaria, angioedema, or anaphylaxis. There is tremendous confusion around this point and lots of confusion around allergy testing and its limitations.”

In addition, patients with atopic dermatitis are more likely than those without atopic dermatitis to have abnormalities in the gut microbiome and gut barrier, Lio said.

Abnormalities in the gut microbiome are different from the concept of allergy and may fall into the more complex category of barrier and microbiome disruptors, he said. Therefore, “the food category may not be nearly as important as the specific preparation of the food along with the additives (such as preservatives and emulsifiers) that may actually be driving the problem.”

Although in the past many clinicians advised patients to try cutting out certain foods to see whether atopic dermatitis symptoms improved, this strategy is not without risk, said Lio. “There have been incredible advancements in understanding the role of the gut in tolerization to foods.” Recent research has shown that by eating foods regularly, particularly those such as peanuts that seem to have more allergic potential, the body becomes tolerant, and this prevents the development of true food allergies.

As for additional research, many questions remain about the effects of types of foods, processing methods, and timing of introduction of foods on atopic dermatitis, Lio noted.

“Atopic dermatitis is a systemic condition with the immune system, with the skin/gut/respiratory barriers and microbiome involved; I think we now have a broader view of how big and complex the landscape really is,” he said.

The study received no outside funding. The researchers had no financial conflicts to disclose. Lio had no disclosures relevant to elimination diets but disclosed serving on the speakers bureau for AbbVie, Arcutis Biotherapeutics, Eli Lilly, Galderma, Hyphens Pharma, Incyte, La Roche–Posay/L’Oréal, Pfizer, Pierre Fabre Dermatologie, Regeneron/Sanofi Genzyme, and Verrica Pharmaceuticals; serving on consulting/advisory boards; or having stock options for many pharmaceutical companies. Lio also disclosed a patent pending for a Theraplex product with royalties paid and is a board member and Scientific Advisory Committee member emeritus of the National Eczema Association.

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