Background: Published case reports and anecdotal experience suggest a positive effect of dexamphetamine on impetus and weight in patients with hypothalamic obesity.
Objective and hypotheses: We aimed to observe these effects in our patients who are offered off-label treatment with dexamphetamine.
Method: Between 2010 and 2013, patients starting dexamphetamine treatment were enrolled in a prospective observation study. BMISDS was determined and impetus was rated on a scale from 1 to 5 at baseline and every 3 months. A retrospective chart review was conducted to establish BMISDS development prior to treatment initiation. Dexamphetamine administration was initiated at a single dose of 5 mg/day, and titrated to effect up to a dose of 20 mg/day in two to three single doses. Side effects were recorded in a standardized fashion.
Results: Six patients, two males, four females, age 17.5 years on average (range: 14.523.8) were included in the study. The primary diagnosis was craniopharyngeoma in five patients, and neonatal meningitis in one patient. Time from initial CNS insult to initiation of dexamphetamine treatment was 6.6 years on average (range 2.717.4 years). All patients demonstrated a steady increase in BMISDS from the time of initial diagnosis up until the initiation of treatment. Baseline BMISDS was +3.1 (1.94.1). After an average treatment duration of 1.6 years (0.23.0), all patients experienced a sustained increase in impetus and a stabilization or reduction in BMISDS by 0.3 (00.8). No significant side effects were reported.
Conclusion: Dexamphetamine lead to improved impetus and stabilization or reduction of BMISDS in a cohort of six patients with hypothalamic obesity. Considering the projected increase in BMISDS according the natural course of the disease, these findings are promising and warrant further study.
20 - 22 Sep 2014
European Society for Paediatric Endocrinology