Dextroamphetamine Treatment in Children With Hypothalamic Obesity
Open accessAbstract
Introduction: Hypothalamic obesity (HO) in children has severe health consequences. Lifestyle interventions are mostly insufficient and currently no drug treatment is approved for children with HO. Amphetamines are known for their stimulant side-effect on resting energy expenditure (REE) and suppressing of appetite. Earlier case series have shown positive effects of amphetamines on weight in children with acquired HO. We present our experiences with dextroamphetamine treatment in the, up to now, largest cohort of children with HO. Methods: A retrospective cohort evaluation was performed of children with HO treated with dextroamphetamine at two academic endocrine pediatric clinics. Off-label use of dextroamphetamine was initiated in patients with progressive, therapy-resistant acquired or congenital HO. Anthropometrics, REE, self-reported (hyperphagic) behavior and energy level, and side effects were assessed at start and during treatment. Results: Nineteen patients with a mean age of 12.3 ± 4.0 years had been treated with dextroamphetamine. In two patients, ΔBMI SDS could not be evaluated due to short treatment duration or the simultaneous start of extensive lifestyle treatment. Mean treatment duration of the 17 evaluated patients was 23.7 ± 12.7 months. Fourteen patients ( n = 10 with acquired HO, n = 4 with congenital HO) responded by BMI decline or BMI stabilization (mean ΔBMI SDS of -0.6 ± 0.8, after a mean period of 22.4 ± 10.5 months). In three patients, BMI SDS increased (mean ΔBMI SDS of +0.5 ± 0.1, after a mean period of 29.7 ± 22.6 months). In 11 responders, measured REE divided by predicted REE increased with +8.9%. Thirteen patients (68.4%) reported decreased hyperphagia, improvement of energy level and/or behavior during treatment. Two patients developed hypertension during treatment, which resulted in dosage adjustment or discontinuation of treatment. Twelve children continued treatment at last moment of follow-up. Conclusion: In addition to supportive lifestyle interventions, dextroamphetamine treatment may improve BMI in children with HO. Furthermore, dextroamphetamines have the potential to decrease hyperphagia and improve resting energy expenditure, behavior, and energy level. In patients with acquired HO, these effects seem to be more pronounced when compared to patients with congenital HO. Future studies are needed to support these results.
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In a retrospective study, dextroamphetamine demonstrated favorable benefits with respect to weight loss (or attenuation of weight gain) while also achieving decreases in hyperphagia and improved energy.
“In a retrospective study, dextroamphetamine (50) has demonstrated favorable benefits with respect to weight loss (or attenuation of weight gain) while also achieving decreases in hyperphagia and improved energy (50).”
From Approach to the patient with acquired hypothalamic syndrome (2026) · cites this paper as [50]
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- Authors
- van Schaik, J., Welling, M. S., de Groot, C. J., van Eck, J. P., Juriaans, A., Burghard, M., Oude Ophuis, S. B. J., Bakker, B., Tissing, W. J. E., Schouten-van Meeteren, A. Y. N., van den Akker, E. L. T., & van Santen, H. M.
- Affiliations
- Erasmus MC (NL)
- University Medical Center Utrecht (NL)
- University Medical Center Groningen (NL)
- Venue
- Frontiers in Endocrinology
- Publisher
- Frontiers Media SA
- Published
- 2022-03-09
- Volume
- 13
- Pages
- 845937-845937
- ISSN
- 1664-2392 (ISSN-L)
- PMC
- PMC8959487
- OpenAlex
- W4225966467
- Cited by
- 30
- References
- 41
- Citations by year
- 2026: 9 · 2025: 9 · 2024: 3 · 2023: 7 · 2022: 2
- FWCI
- 2.77
- Type
- journal article
- Language
- en
- Open access
- gold
- doi.org
- Licence
- Locations
- Frontiers in Endocrinology (publishedVersion, cc-by)
- PubMed Central (publishedVersion, cc-by)
- PubMed (publishedVersion)
- EUR Research Repository (Erasmus University Rotterdam) (publishedVersion, cc-by)
- University of Groningen research database (University of Groningen / Centre for Information Technology) (publishedVersion, cc-by)
- Data Archiving and Networked Services (DANS) (submittedVersion, other-oa)
- EUR Research Repository (Erasmus University Rotterdam) (submittedVersion, cc-by)
- DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
- Pure Amsterdam UMC (submittedVersion, cc-by)
- University of Groningen research database (University of Groningen / Centre for Information Technology) (submittedVersion, cc-by)
- Topics
- Life Sciences › Neuroscience › Endocrine and Autonomic Systems › Regulation of Appetite and Obesity
- Health Sciences › Medicine › Pharmacology › Pharmacology and Obesity Treatment
- Health Sciences › Medicine › Reproductive Medicine › Hypothalamic control of reproductive hormones
- Keywords
- hypothalamic obesity, d-amphetamine, childhood obesity, hyperphagia, resting energy expenditure, BMI reduction, hypertension, stimulant side effects
- MeSH terms
- Adolescent, Child, Dextroamphetamine, Energy Metabolism, Humans, Hypothalamic Diseases, Obesity, Retrospective Studies
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