Susan Furth, MD, PhD Executive Vice President and Chief Scientific Officer | Children's Hospital of Philadelphia
Susan Furth, MD, PhD Executive Vice President and Chief Scientific Officer | Children's Hospital of Philadelphia
A study conducted at Children’s Hospital of Philadelphia (CHOP) explored the efficacy of glucagon-like peptide-1 (GLP-1) receptor agonists in treating metabolic dysfunction-associated steatotic liver disease (MASLD) in children. The study found significant reductions in alanine aminotransferase (ALT) levels, especially in children also diagnosed with type 2 diabetes.
MASLD, previously called non-alcoholic fatty liver disease, is currently the most prevalent chronic liver disease among children in the United States, affecting about 10% of the population. It is also becoming a leading cause of liver transplants in adults.
Lifestyle changes, including a balanced diet and regular exercise, are regarded as the first line of treatment for pediatric MASLD. However, implementing these changes can be challenging due to the rising rates of obesity and type 2 diabetes among children.
The study, led by Dr. Jennifer Panganiban, a gastroenterologist and director of CHOP’s Metabolic Dysfunction-Associated Steatotic Liver Disease Program, was retrospective and conducted in a real-world setting. The single-center study included patients 21 and younger who began treatment with GLP-1 receptor agonists for either obesity or diabetes between January 2018 and January 2024.
Out of 111 patients, half were male, and the cohort exhibited a diverse racial composition. The study measured several parameters, including ALT levels, body mass index (BMI), and various other MASLD-associated biomarkers.
The primary outcome was a significant reduction in ALT, defined as at least 17 U/L. Study results indicated an ALT improvement of 23 U/L after six months and 18 U/L by the end of the treatment. Patients with a higher baseline ALT level experienced the most considerable reductions.
Secondary outcomes focused on other MASLD-associated biomarkers. Although there were no significant changes in BMI measurements, improvements were observed in glycated hemoglobin, aspartate aminotransferase, gamma-glutamyl transferase, and triglyceride levels.
Dr. Andrea M. Tou, a fellow at CHOP, highlighted the potential of GLP-1 receptor agonists as a powerful option for pediatric MASLD patients, particularly those with type 2 diabetes. "GLP-1 RAs are therefore a potentially powerful option for our pediatric patients with MASLD in conjunction with lifestyle changes, especially for patients with T2DM, who have the highest risk for progression if left untreated." This study offers a strong basis for future investigations into GLP-1RAs as a therapeutic approach for pediatric MASLD.
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