ESPE2021 ePoster Category 2 Fat, metabolism and obesity (59 abstracts)
The effects of dextroamphetamine treatment in children with hypothalamic obesity
J Samaan 1,2 , MS Welling 3,4 , CJ de Groot 3,4 , O Abawi 3,4 , M Burghard 2,5 , L Kleinendorst 4,6 , B van der Voorn 3,4 , MM van Haelst 4,6 , B Oude Ophuis 2,7 , GA Kamp 8 , J Rotteveel 9 , AYN van Schouten-Meeteren 2 , ELT van den Akker 3,4 & HM van Santen 1,2
1Division of Pediatric Endocrinology, Wilhelmina Children’s Hospital, University Medical Center, Utrecht, Netherlands; 2Princess Maxima Center for Pediatric Oncology, Utrecht, Netherlands; 3Division of Pediatric Endocrinology, Erasmus MC Sophia Children’s Hospital, University Medical Center, Rotterdam, Netherlands; 4Obesity Center CGG, Erasmus MC Sophia Children’s Hospital, University Medical Center, Rotterdam, Netherlands; 5Dept. of Exercise Physiology, Child Development & Exercise Center, Wilhelmina Children’s Hospital, University Medical Center, Utrecht, Netherlands; 6Dept. of Clinical Genetics, Amsterdam UMC, Vrije Universiteit Amsterdam/University of Amsterdam, Amsterdam, Netherlands; 7Dept. of Pediatric Psychiatry, Wilhelmina Children’s Hospital, University Medical Center, Utrecht, Netherlands; 8Dept. of Pediatrics, Tergooi Hospital, Blaricum, Netherlands; 9Dept. of Pediatric Endocrinology, VU Medical Center, Amsterdam, Netherlands
Introduction: Children with hypothalamic dysfunction, e.g. due to a genetic or acquired cause such as suprasellar tumours, often suffer from hyperphagia and/or decreased resting energy expenditure (REE). This process induces uncontrollable weight gain, resulting in severe hypothalamic obesity (HO). No effective treatment is available yet for HO. Amphetamines are psychostimulants that are known for their appetite reducing and REE stimulating side effects. Here, we present the effects of dextroamphetamine treatment in children and adolescents with acquired, genetic, or congenital HO.
Methods: A retrospective cohort evaluation was performed at 2 endocrine paediatric clinics. Patients with progressive therapy-resistant HO, either due to an acquired, genetic, or congenital cause, and off-label treatment with dextroamphetamines were included. Treatment was initiated at once or twice daily 5 mg. This could be weekly increased with 5 mg/day to a maximum of 0.5 mg/kg/day, depending on the patient’ wellbeing or side effects. At start and during follow-up, BMI standard deviation scores (BMI SDS), REE (% of predicted), changes in (hyperphagic) behaviour, and side effects were assessed.
Results: Nineteen patients were treated with dextroamphetamines. Their mean age was 12.3 years ± 4.0 and mean treatment duration was 19.5 months ± 12.9. Fourteen children (acquired in n = 10, genetic in n = 3, congenital in n = 1) responded to therapy, either with weight loss (n = 11) or BMI SDS stabilization (n = 3), with a mean ΔBMI SDS of -0.6 ± 0.8. The predicted REE increased with 9.7% ± 10.6 in the responder subgroup (n = 11). Three children (acquired in n = 2, genetic in n = 1) gained weight (mean ΔBMI SDS +0.5 ± 0.1). In two children, the effects of dextroamphetamines could not be evaluated due to early treatment termination because of hypertension or a concurrent intensive supportive lifestyle treatment at a rehabilitation clinic. Thirteen out of 19 children reported improvement of hyperphagia, energy level, and/or behaviour. Reported side effects were hypertension, difficulties falling asleep, and negative behaviour changes. At last moment of follow-up, treatment was continued in ten children with an acquired HO and two children with a genetic HO.
Conclusion: In children and adolescents with progressive therapy-resistant HO, due to an acquired, genetic, or congenital cause, treatment with dextroamphetamine may be beneficial and result in either weight loss or weight stabilization. Moreover, it reduces subjectively reported hyperphagia and improve energy level and/or behaviour. Future studies are needed to support these results.
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