Background: Published case reports and anecdotal experience suggest a positive effect of dexamphetamine, a CNS stimulant on impetus and weight in patients with hypothalamic obesity. Based on these observations, patients presenting to our obesity clinic with hypothalamic obesity are offered off-label treatment with dexamphetamine.
Method: Between 2010 and 2015, patients starting dexamphetamine treatment were enrolled in a prospective observation study. A retrospective chart review was conducted to establish BMI-SDS development prior to treatment initiation. Impetus was rated on a scale from 1 to 5 at baseline and every 3 months. 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 23 single doses. Side effects were recorded in a standardized fashion.
Results: Nine patients (three males) mean age 17.2 years (range: 13.023.8) were included in the study. The primary diagnosis was craniopharyngeoma in 6 patients, ganglioglioma WHO °I in one patient, neonatal meningitis in one patient and astrocytoma in one patient. Time from initial CNS insult to initiation of dexamphetamine treatment was 5.7 years on average (range 4 months to 17.4 years). All patients demonstrated a steady increase in BMI-SDS from the time of initial diagnosis up until the initiation of treatment. Of the nine Patients, two were excluded from the evaluation because of proven non-compliance. Baseline BMI-SDS of the remaining seven patients was +3.1 (1.94.4). After a mean treatment duration of 1.8 years (0.24.1), BMI-SDS decreased on average by 0.5 (01.36) and the mean score for impetus improved from 1.3 to 2.8. No significant side effects were reported.
Conclusion: Dexamphetamine lead to improved impetus and stabilisation or reduction of BMI-SDS in a cohort of seven patients with hypothalamic obesity. Considering the projected increase in BMI-SDS according the natural course of the disease, these findings are promising and warrant further study.
01 - 03 Oct 2015
European Society for Paediatric Endocrinology