Semaglutide for Treating Paediatric Craniopharyngioma-Related Obesity: A Multicentre Case Series

Obesity is a complication of childhood craniopharyngioma that remains difficult to treat. We investigated whether semaglutide was associated with a decrease in BMI among adolescents with craniopharyngioma-related obesity.

This multicentre retrospective study included 20 patients < 18 years old with craniopharyngioma and obesity (BMI > 2 WHO Z-score) from 2019 to 2024 (source population: 139 children with craniopharyngioma, including 36 children with obesity). They received off-label semaglutide with dose titration (0.25-2 mg/week), tailored to tolerance and clinical response. The primary outcome was the within-patient comparison of BMI change from -12 months to treatment initiation and from initiation to +12 months.

At semaglutide initiation, patients were 14.0 ± 3.9 years old, the interval since craniopharyngioma diagnosis was 6.3 ± 4.7 years, and BMI was 33.4 ± 3.9 kg/m² (3.2 ± 0.7 Z-score). Tumour treatment was surgery alone (8/20), surgery and proton therapy (11/20), or proton therapy alone (1/20). All patients had growth hormone and TSH deficiency; 19/20 had ACTH and AVP deficiency. In the year before treatment, patients gained 3.5 ± 4.4 kg/m² in BMI and 0.6 ± 1.6 in BMI Z-score; after 1 year of semaglutide therapy, they lost 2.5 ± 3.9 kg/m² in BMI and 0.5 ± 0.5 in BMI Z-score (paired comparisons, p < 0.001 and p = 0.004, respectively). During treatment, BMI decreased by 10% or more in 40% of patients (95% CI: 19%-64%). Gastrointestinal side effects occurred in 30% of patients but were mild. No cases of treatment discontinuation, pituitary insufficiency decompensation or tumour recurrence were observed.

Semaglutide was associated with reversal of weight gain among adolescents with craniopharyngioma-related obesity.