Effect of Topiramate on Weight Status in Young Children with Severe Obesity

Background Pharmacological options for the treatment of early severe childhood obesity are limited. This retrospective case series evaluates the effect of topiramate on weight and cardiometabolic parameters in young children with severe obesity, including those with hypothalamic etiologies. Methods This is a retrospective case series including fourteen children with severe obesity, who were treated with topiramate prior to age 12. Anthropometric and metabolic data were analyzed. Results The median age at initiation of topiramate treatment was 10.1 years (range: 7.9–11.2), with 64% were males. At baseline, the median BMI was 149% of the 95th percentile (range: 136–200). The median treatment duration was 14 months (IQR: 9.5–24), with a maximum tolerated dose of 75 mg (range: 50–150 mg). There was a median decrease in BMI% of the 95th percentile of 8% (95% CI: –15 to –1.02; p = 0.042), and a reduction in BMI z-score by 0.37 (95% CI: –0.66 to –0.05; p = 0.028). The most notable response was in a child with hypothalamic obesity who experienced a 35-point reduction in BMI% of the 95th percentile over 24 months. While 11 of 14 children showed reduction in BMI, three had no change or increase in their BMI. No significant changes were observed in cardiometabolic markers or linear growth. Conclusion Topiramate monotherapy resulted in significant BMI reductions in young children with severe obesity, including those with hypothalamic obesity. Further prospective randomized controlled trials are warranted to assess its long-term efficacy for obesity management in young children.