Effects on growth and metabolism of growth hormone treatment for 3 years in 36 children with Prader-Willi syndrome

Horm Res Paediatr. 2011 Feb;75(2):123-30. doi: 10.1159/000319709. Epub 2010 Sep 15.


Background/aims: Prader-Willi syndrome (PWS) is a complex genetic disorder whose many manifestations include obesity and short stature. Diabetes, osteoporosis, and scoliosis are common. We evaluated the effects of human growth hormone (hGH).

Methods: A prospective cohort study of 36 children (1-15 years of age) with genetically confirmed PWS who were given hGH (mean dose 0.033 ± 0.006 mg/kg/day) for 36 months. At baseline and once yearly, we evaluated growth, insulin-like growth factor-1 (IGF-1), body composition, bone mineral density (BMD), glucose tolerance, serum lipids, and spinal radiographs.

Results: Height gain over the 3-year period was 1.2 SD score. Lean body mass increased significantly during each treatment year. Total body fat decreased by 5.42 and 1.17% in the 1st and 2nd years, respectively. BMD remained unchanged during therapy. IGF-1 and homeostasis model assessment index of insulin resistance increased, and glucose intolerance was found in 22.7% of patients at baseline and 0% at 3 years. None of the patients had diabetes. Their lipid profile improved. Scoliosis was present in 27.8% of the patients at baseline and 47.2% at 3 years.

Conclusion: GH treatment in children with PWS has multiple beneficial effects on growth and body composition. Tolerance is good, with an improvement in glucose metabolism, although IGF-1 levels and insulin resistance parameters should be monitored closely. The high rate of scoliosis warrants monitoring by a pediatric orthopedic surgeon.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Body Composition / drug effects
  • Bone Density / drug effects
  • Carbohydrate Metabolism / drug effects
  • Child
  • Cohort Studies
  • Female
  • Human Growth Hormone / therapeutic use*
  • Humans
  • Insulin Resistance / physiology
  • Insulin-Like Growth Factor I / metabolism
  • Lipid Metabolism / drug effects
  • Male
  • Prader-Willi Syndrome / drug therapy*
  • Prader-Willi Syndrome / metabolism*
  • Prospective Studies
  • Scoliosis / etiology


  • Human Growth Hormone
  • Insulin-Like Growth Factor I