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We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com. Back to Healio Key takeaways: Treatment options remain limited for pediatric MASLD, a condition \u201cas common as asthma.\u201d Early data are promising, but clinical trials are needed before adult MASH therapies can be approved for use in children. The recent FDA approvals of pharmacologic therapies for metabolic dysfunction-associated steatohepatitis were \u201cbreakthrough moments in history\u201d for a disease whose management had mostly relied on lifestyle interventions. However, the two drugs that drastically changed the treatment of MASH in adults \u2014 resmetirom (Rezdiffra, Madrigal Pharmaceuticals) and semaglutide (Wegovy, Novo Nordisk) \u2014 have not been approved for the treatment of MASH among patients aged younger than 18 years.
\u201cI don\u2019t think people understand how common [pediatric metabolic dysfunction\u2013associated steatotic liver disease] is,\u201d Jennifer Panganiban, MD, gastroenterologist and director of the MASLD program at Children\u2019s Hospital of Philadelphia, told Healio. \u201cIt\u2019s as common as asthma, and people are shocked when they hear that in clinic.\u201d The global prevalence of pediatric MASLD is estimated to be roughly 13% among children and adolescents, according to data published in World Journal of Pediatrics. Nearly half (47%) of children with obesity have comorbid fatty liver disease. Pediatric MASLD can compromise children\u2019s liver health. \u201cAdvanced fibrosis is already present in approximately 10% to 15% of children with MASH at the time of diagnosis,\u201d Panganiban said. The current standard of care, centered on lifestyle intervention, is often not effective for many children with MASLD. In a 2025 study of 440 children with biopsy-confirmed MASLD receiving standard clinical care, 22% demonstrated improvement after 1 year, which increased to 31% after 2 years. At the same time, approximately 20% experienced worsening liver disease despite standard-of-care management, underscoring the need for more effective therapeutic options. \u2018Promising\u2019 results The health risks and treatment challenges associated with pediatric MASLD and MASH highlight the need for randomized clinical trials evaluating the efficacy of pharmacologic therapies in young patients, according to Panganiban. \u201cAfter we accumulate safety data and look at [drug] interactions, we start working on trials in younger individuals,\u201d Rohit Loomba, MD, MHSc, director of UC San Diego MASLD Research Center, told Healio. \u201cThere are discussions regarding potential studies in a pediatric population with MASH-related fibrosis, and we hope to see those in the future, develop the efficacy and safety [data] in that population, and potentially utilize those medications.\u201d Several GLP-1 receptor agonists, including semaglutide, tirzepatide, liraglutide, dulaglutide and exenatide, have pediatric approval for obesity and type 2 diabetes. Researchers have conducted retrospective studies to evaluate the impact of GLP-1s on markers of liver health in children with FDA-approved indications for GLP-1 use, and the results appear \u201cpromising,\u201d according to Loomba. One study, published in Journal of Pediatric Gastroenterology and Nutrition, included children diagnosed with MASLD (mean age, 15 years; 64% boys; 55% white) who were prescribed a GLP-1 at Children\u2019s Hospital of Philadelphia. \u201cWe looked at 42 patients that met the official diagnosis [criteria] for pediatric MASLD based on hepatic imaging, as well as cardiometabolic risk factors,\u201d Panganiban, one of the study investigators, said. Results showed alanine aminotransferase levels improved by a mean 56 U\/L at 6 months (P = .04) and by 37 U\/L at end of treatment (P = .004). The effect on ALT levels was greater in children with type 2 diabetes, compared with those with obesity. The researchers also observed improvements in aspartate aminotransferase, gamma-glutamyl transpeptidase, HbA1c and triglycerides among children treated with a GLP-1. \u201cAll the available data from GLP-1 receptor agonist trials in obesity and type 2 diabetes point to safe use and meaningful improvements in weight and BMI, but these studies were not designed to assess liver outcomes,\u201d Panganiban said. \u201cThat gap highlights the urgent need for dedicated pediatric MASLD trials.\u201d As a follow-up to those encouraging findings, Panganiban noted that her team has been conducting a prospective, observational study of children and adolescents with MASLD. Preliminary results were presented in October at NASPGHAN Annual Meeting. \u201c[The results] demonstrated significant and sustained improvements in liver enzymes, with imaging changes suggesting improvements in steatohepatitis following semaglutide treatment,\u201d she said. \u201c Notably, these improvements were observed as early as 3 months and continued through 12 months of follow-up, providing further evidence of the potential role of GLP-1 receptor agonists in treating pediatric MASLD.\u201d Panganiban and colleagues also presented promising data on semaglutide at The Liver Meeting this month. In a retrospective study of 145 children with MASLD from 2023 to 2026, 93% of those who received semaglutide achieved at least 30% relative fat fraction reduction compared with 25% who did not receive the drug. \u201cWe show that semaglutide was associated with significantly higher improvement of steatosis, NAFLD Activity Score and MASH resolution, without worsening of fibrosis per biopsy, compared with contemporaneous controls,\u201d Panganiban said. A phase 2 trial of resmetirom, which is approved to treat MASH in adults, is also under way. The multicenter, open-label study is evaluating the safety, pharmacokinetics and pharmacodynamics of the drug among children aged 6 to 17 years with MASH with stage F1 to F3 fibrosis. Panganiban called the research \u201cexciting\u201d progress. \u201cIn pediatrics, you want to impact change early, before [fibrosis] progression occurs,\u201d she said. \u2018Core treatment\u2019 Should pharmacologic therapies be approved to treat pediatric MASLD and MASH, Loomba emphasized the importance of identifying patients\u2019 individual risk and developing tailored treatment plans. \u201cThe majority of individuals with MASLD in the pediatric age group \u2014 younger than 18 years of age \u2014 will not have significant fibrosis, which is stage two, stage three or stage four,\u201d Loomba said. He added that the focus should be on risk reduction in patients with<\/p>\n","protected":false},"author":1,"featured_media":4321,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[1],"tags":[],"class_list":["post-4320","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-uncategorized"],"_links":{"self":[{"href":"https:\/\/drsoniafawad.com\/index.php?rest_route=\/wp\/v2\/posts\/4320","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/drsoniafawad.com\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/drsoniafawad.com\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/drsoniafawad.com\/index.php?rest_route=\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/drsoniafawad.com\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=4320"}],"version-history":[{"count":0,"href":"https:\/\/drsoniafawad.com\/index.php?rest_route=\/wp\/v2\/posts\/4320\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/drsoniafawad.com\/index.php?rest_route=\/wp\/v2\/media\/4321"}],"wp:attachment":[{"href":"https:\/\/drsoniafawad.com\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=4320"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/drsoniafawad.com\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=4320"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/drsoniafawad.com\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=4320"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}
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