Eight years of growth hormone treatment in children with Prader-Willi syndrome: maintaining the positive effects

J Clin Endocrinol Metab. 2013 Oct;98(10):4013-22. doi: 10.1210/jc.2013-2012. Epub 2013 Sep 3.


Background: The most important reason for treating children with Prader-Willi syndrome (PWS) with GH is to optimize their body composition.

Objectives: The aim of this ongoing study was to determine whether long-term GH treatment can counteract the clinical course of increasing obesity in PWS by maintaining the improved body composition brought during early treatment.

Setting: This was a multicenter prospective cohort study.

Methods: We have been following 60 prepubertal children for 8 years of continuous GH treatment (1 mg/m(2)/d ≈ 0.035 mg/kg/d) and used the same dual-energy x-ray absorptiometry machine for annual measurements of lean body mass and percent fat.

Results: After a significant increase during the first year of GH treatment (P < .0001), lean body mass remained stable for 7 years at a level above baseline (P < .0001). After a significant decrease in the first year, percent fat SD score (SDS) and body mass index SDS remained stable at a level not significantly higher than at baseline (P = .06, P = .14, resp.). However, body mass index SDSPWS was significantly lower after 8 years of GH treatment than at baseline (P < .0001). After 8 years of treatment, height SDS and head circumference SDS had completely normalized. IGF-1 SDS increased to +2.36 SDS during the first year of treatment (P < .0001) and remained stable since then. GH treatment did not adversely affect glucose homeostasis, serum lipids, blood pressure, and bone maturation.

Conclusion: This 8-year study demonstrates that GH treatment is a potent force for counteracting the clinical course of obesity in children with PWS.

Publication types

  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Absorptiometry, Photon
  • Adolescent
  • Body Composition / drug effects*
  • Body Height / drug effects
  • Bone Density / drug effects
  • Child
  • Child, Preschool
  • Disease Progression
  • Female
  • Human Growth Hormone / pharmacology
  • Human Growth Hormone / therapeutic use*
  • Humans
  • Male
  • Obesity / diagnostic imaging
  • Obesity / drug therapy*
  • Prader-Willi Syndrome / diagnostic imaging
  • Prader-Willi Syndrome / drug therapy*
  • Prospective Studies
  • Treatment Outcome


  • Human Growth Hormone