Methylphenidate improves weight control in childhood brain tumor survivors with hypothalamic obesity

Pediatr Blood Cancer. 2020 Jul;67(7):e28379. doi: 10.1002/pbc.28379. Epub 2020 May 8.


Background: Hypothalamic obesity causes unrelenting weight gain for childhood brain tumor survivors. No single therapy has proven effective for treatment. We aimed to evaluate effectiveness of long-term methylphenidate therapy on body mass index (BMI) change in children with hypothalamic obesity.

Methods: A retrospective analysis included children with a history of brain tumor and hypothalamic obesity receiving methylphenidate (10-60 mg/day) for hypothalamic obesity. Subjects were evaluated for BMI trajectory before and after methylphenidate start. Given that z-scores can be skewed in severely obese children, we calculated BMI as a percent of the BMI at the 95th percentile for the child's age and gender (BMI% 95th).

Results: Twelve patients with hypothalamic obesity completed methylphenidate therapy for at least 6 months (median 3.1 years, range 1.0-5.8 years). All subjects had a suprasellar tumor (nine [75%] with craniopharyngioma) and pituitary dysfunction. Pretreatment median BMI percent of the 95th percentile was 125.6% (interquartile range [IQR] 25-75: 115.3-138.3%) with BMI z-score of 2.4 (IQR 25-75: 2.1-2.6). Following methylphenidate treatment, there was a 69.9% reduction in the median slope of BMI change. Eleven of 12 patients (92%) had a reduction in the slope of their BMI change on methylphenidate treatment. Postmethylphenidate median BMI percent of the 95th percentile decrease to 115.2% (IQR 25-75: 103.6-121.2%) with median BMI z-score of 2.1 (IQR 25-75: 1.8-2.2). Mild side effects were noted in six patients.

Conclusions: Methylphenidate use reduced and sustained BMI change in children with hypothalamic obesity. Stimulant therapy is an effective first-line agent for treatment of hypothalamic obesity.

Keywords: CNS tumors; childhood obesity; endocrinology; pharmacologic therapy.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Brain Neoplasms / complications*
  • Brain Neoplasms / therapy
  • Cancer Survivors / statistics & numerical data*
  • Central Nervous System Stimulants / therapeutic use*
  • Child
  • Child, Preschool
  • Female
  • Follow-Up Studies
  • Humans
  • Hypothalamic Diseases / diagnosis
  • Hypothalamic Diseases / drug therapy*
  • Hypothalamic Diseases / etiology
  • Male
  • Methylphenidate / therapeutic use*
  • Obesity / diagnosis
  • Obesity / drug therapy*
  • Obesity / etiology
  • Prognosis
  • Retrospective Studies
  • Weight Loss / drug effects*


  • Central Nervous System Stimulants
  • Methylphenidate