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Hypothalamic obesity is a devastating complication in children surviving brain tumors and/or cranial irradiation. These subjects are thought to exhibit autonomic dysregulation of the β-cell, with insulin hypersecretion in response to oral glucose tolerance testing (OGTT). We report the results of a randomized, double-blind, placebo-controlled trial of octreotide therapy for pediatric hypothalamic obesity. Eighteen subjects [weight, 100.6 ± 5.6 kg; body mass index (BMI), 37.1 ± 1.3 kg/m²] received octreotide (5–15 μg/kg·d sc) or placebo for 6 months.

With octreotide, Δweight (mean ± sem) was +1.6 ± 0.6 vs. +9.1 ± 1.7 kg for placebo (P < 0.001). ΔBMI was −0.2 ± 0.2 vs. +2.2 ± 0.5 kg/m², respectively (P < 0.001). OGTT documented Δinsulin response (peak − basal) of −417 ± 304 pm after octreotide vs. +216 ± 215 pm after placebo (P = 0.034). Improvement in physical activity by parent report was noted with octreotide …