Growth hormone treatment in adults with Prader-Willi syndrome: the Scandinavian study

Endocrine. 2012 Apr;41(2):191-9. doi: 10.1007/s12020-011-9560-4. Epub 2011 Nov 12.


Prader-Willi syndrome (PWS) is characterized by short stature, muscular hypotonia, cognitive dysfunction, and hyperphagia usually leading to severe obesity. Patients with PWS share similarities with growth hormone deficiency (GHD). Few studies have dealt with growth hormone (GH) treatment in PWS adults. The purpose of the Scandinavian study was to evaluate the effects of GH on body composition, lipid and glucose metabolism, physical performance and safety parameters in adults with PWS. Twenty-five women and 21 men with PWS were randomized to treatment with GH or placebo during 1 year followed by 2 years of open labeled GH treatment. At baseline 1/3 had normal BMI, six patients severe GHD, ten impaired glucose tolerance and seven diabetes. At 1 year insulin-like growth factor I (IGF-I) SDS had increased by 1.51 (P < 0.001) and body composition improved in the GH treated group. Visceral fat decreased by 22.9 ml (P = 0.004), abdominal subcutaneous fat by 70.9 ml (P = 0.003) and thigh fat by 21.3 ml (P = 0.013), whereas thigh muscle increased 6.0 ml (P = 0.005). Lean body mass increased 2.25 kg (P = 0.005), and total fat mass decreased 4.20 kg (P < 0.001). The positive effects on body composition were maintained after 2 years of GH treatment. Peak expiratory flow increased by 12% (P < 0.001) at 2 years of GH treatment. Lipid and glucose metabolism were unchanged, however, three patients developed diabetes at 2 years of GH treatment. In conclusion GH treatment had beneficial effects on the abnormal body composition without serious adverse events making it a logic treatment option in adults with PWS.

Publication types

  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Adult
  • Body Composition / drug effects
  • Cohort Studies
  • Diabetes Complications / blood
  • Diabetes Complications / drug therapy
  • Diabetes Complications / metabolism
  • Diabetes Complications / physiopathology
  • Double-Blind Method
  • Female
  • Follow-Up Studies
  • Human Growth Hormone / adverse effects
  • Human Growth Hormone / therapeutic use*
  • Humans
  • Insulin Resistance
  • Insulin-Like Growth Factor I / analysis
  • Male
  • Middle Aged
  • Peak Expiratory Flow Rate / drug effects
  • Prader-Willi Syndrome / blood
  • Prader-Willi Syndrome / drug therapy*
  • Prader-Willi Syndrome / metabolism
  • Prader-Willi Syndrome / physiopathology
  • Recombinant Proteins / adverse effects
  • Recombinant Proteins / therapeutic use
  • Scandinavian and Nordic Countries
  • Severity of Illness Index
  • Young Adult


  • Recombinant Proteins
  • Human Growth Hormone
  • Insulin-Like Growth Factor I