Topiramate treatment of pediatric metabolic dysfunction-associated steatotic liver disease: A descriptive cohort study.

Background: Metabolic dysfunction-associated steatotic liver disease (MASLD) is a common disease in children. Lifestyle modification is the primary treatment but difficult to achieve and maintain. Topiramate is a component of an approved weight loss medication (topiramate-phentermine) in children aged 12 years and older but is more commonly used as a single agent, off-label, for pediatric obesity. Our aim is to describe change in anthropometrics and laboratory values while providing topiramate treatment of pediatric MASLD.

Methods: Descriptive cohort study including patients aged <18 years with MASLD and body mass index (BMI) >95th percentile treated with topiramate for weight loss for ≥3 months from January 1, 2010, to December 30, 2023. The primary outcome was change in serum alanine aminotransferase (ALT) levels from baseline to 3-6 months. Secondary outcomes were changes in BMI z score, glycated hemoglobin, and lipid profile.

Results: Of 43 patients prescribed topiramate, 11 were excluded for nonadherence, leaving 32 (56% boys, 72% non-Hispanic) for further analyses. With topiramate, ALT levels improved (76 vs 50 U/L, p = 0.001). Further, 43% of patients had either ALT normalization or reduction by >50% from baseline. BMI z score decreased by 0.1 from baseline to 3-6 months. There were no improvements in glycated hemoglobin or lipids. Eight patients (25%) reported mild side effects.

Conclusion: Topiramate, as an adjunct to lifestyle intervention, may be considered in the treatment of pediatric MASLD, specifically in the context of failed lifestyle modification and inability to tolerate or qualify for other obesity pharmacotherapy.