Octreotide therapy of pediatric hypothalamic obesity: a double-blind, placebo-controlled trial.
Level 2 - randomized trial
Randomized, double-blind, placebo-controlled trial
PubMed 12788859 · doi:10.1210/jc.2002-030003
What was done
Eighteen pediatric subjects with hypothalamic obesity (mean baseline weight 100.6 ± 5.6 kg, BMI 37.1 ± 1.3 kg/m²) following brain tumors or cranial irradiation were enrolled in a randomized, double-blind, placebo-controlled trial. Participants received subcutaneous octreotide (5–15 µg/kg/day) or placebo for 6 months. Changes in weight, BMI, oral glucose tolerance test (OGTT) insulin response, parent-reported physical activity, quality of life, and adverse events were assessed.
What was found
Compared to placebo, octreotide resulted in significantly less weight gain (+1.6 ± 0.6 kg vs. +9.1 ± 1.7 kg, P < 0.001) and stabilized BMI (-0.2 ± 0.2 kg/m² vs. +2.2 ± 0.5 kg/m², P < 0.001). The OGTT insulin response (peak minus basal) decreased by -417 ± 304 pM with octreotide compared to +216 ± 215 pM with placebo (P = 0.034). Parent-reported physical activity improved with octreotide but not placebo (P = 0.03). Improvements in quality of life in the octreotide group positively correlated with insulin suppression (P = 0.041). Adverse events were mild and self-limited.
Why it matters
It demonstrates that suppressing autonomic beta-cell hypersecretion with octreotide can arrest the rapid, severe weight gain typically observed in pediatric patients surviving cranial tumors or irradiation.
Limits
The study sample size was very small (n = 18 total across both arms), treatment duration was limited to 6 months, and physical activity was assessed via subjective parent report rather than objective tracking. Long-term durability and safety beyond 6 months were not evaluated.
Cited by
- supports In pediatric patients with hypothalamic obesity, administering octreotide suppressed insulin release, resulting in weight loss, spontaneous physical activity, and quality-of-life improvements that correlated directly with the degree of insulin suppression.