Introduction
Hypothalamic obesity (HO) in children may have devastating consequences. Lifestyle interventions are mostly insufficient and no effective drug treatment is available. Amphetamines are known for their stimulant effect on resting energy expenditure (REE) and suppressing appetite. We present our experiences of dextroamphetamine treatment in children with HO.

Methods
A retrospective cohort evaluation was performed of patients treated with dextroamphetamine at two endocrine pediatric clinics. Off-label use of dextroamphetamine was initiated in patients with progressive, therapy-resistant acquired, genetic, or congenital HO. Anthropometrics, REE, (hyperphagic) behavior, and side effects were assessed at start and during treatment.

Results
Nineteen patients with a mean age of 12.3 years ± 4.0 were treated. ΔBMI SDS could be evaluated in 17 patients, mean treatment duration was 26.5 months ± 15.0. Fourteen patients (n=10 with acquired HO, n=3 with genetic HO, n=1 with congenital HO) responded by weight loss or BMI stabilization (mean ΔBMI SDS of -0.6 ± 0.8, after mean months 22.4 ± 10.5). In three patients, BMI SDS increased (mean ΔBMI SDS of +0.5 ± 0.1, mean months 29.7 ± 22.6). In the 11 responders of whom REE was measured before and during treatment, mean REE increased with +164 kcal/day (+8.9% of predicted). Thirteen patients (68.4%) reported improvement of hyperphagia, energy level, or behavior. Two patients developed hypertension during treatment. Twelve children continued treatment at last moment of follow-up.

Conclusion

In children with HO, adding treatment with dextroamphetamine to supportive lifestyle intervention, may lower or stabilize BMI SDS, improve REE, reduce hyperphagia, and improve activity level. In acquired HO, these effects seem to be more pronounced. Future studies are needed to support these results.