Diazoxide in Children With Obesity After Hypothalamic-Pituitary Lesions: A Randomized, Placebo-Controlled Trial

The objective was to evaluate the safety and efficacy of diazoxide (DZX) for decreasing obesity with hyperinsulinemia in patients treated for hypothalamic-pituitary lesions during childhood.

This was a double-blind, placebo-controlled trial in parallel groups using a centralized randomization process (PEDIAC).

This was a single-center study.

Among the 40 patients included, 35 fulfilled the study requirements.

Interventions included six-month treatment with DZX (4 mg/kg/d) or placebo.

The primary outcome was relative weight change at 2 months. Secondary outcomes were changes in absolute weight, plasma insulin concentrations, glucose peak after oral glucose tolerance test (OGTT), and glycosylated hemoglobin after 2 months.

Eighteen participants were randomized to the DZX group; three withdrew their consent or were excluded after the occurrence of diabetes mellitus at days 10, 10, and 35, respectively; and two dropped out because of protocol non-compliance at day 10. No statistically significant differences in baseline characteristics were observed among the 13 DZX patients and the 17 placebo patients. The relative weight changes at 2 months in the DZX and placebo groups were -0.9 and -0.5%, respectively (P = nonsignificant). No statistically significant differences were observed between the groups concerning the change in absolute weight or glycosylated hemoglobin after 2 months, but the plasma glucose concentrations (basal and after OGTT) were significantly greater in the patients receiving DZX treatment vs those receiving the placebo, whereas the plasma increases in insulin after OGTT were lower.

The 2-month treatment with DZX was not associated with a significant change in weight compared with placebo; however, it was associated with the occurrence of diabetes mellitus in three of 18 patients.