Growth hormone treatment improves body composition in adults with Prader-Willi syndrome

Clin Endocrinol (Oxf). 2003 May;58(5):653-61. doi: 10.1046/j.1365-2265.2003.01769.x.


Objective: Low growth hormone (GH) secretion and hypogonadism are common in patients with Prader-Willi syndrome (PWS). In this study we present the effects of GH treatment on body composition and metabolism in adults with PWS.

Patients and measurements: Nineteen patients with clinical PWS were recruited, 13 had PWS genotype. They were randomised to treatment with placebo or GH (Genotropin, Pharmacia Corporation, Sweden) 0.8 IU (0.2 mg) daily for 1 month and then 1.6 IU (0.5 mg) daily for 5 months. Thereafter patients received open label treatment so that all had 12 months of active GH treatment. Doses were individually titrated to keep serum IGF-I within the normal range for age. Body composition using dual energy X-ray absorptiometry (DXA), metabolic and endocrinological parameters, including oral glucose tolerance test (OGTT), were studied every 6 months. Seventeen patients, nine men and eight women, 17-32 years of age, with a mean body mass index (BMI) of 35 +/- 3.2 kg/m2 completed the study.

Results: Compared to placebo, GH treatment increased IGF-I (P < 0.01) levels and decreased body fat (P = 0.04). When all patients recieved GH treatment a mean reduction in body fat of 2.5% (P < 0.01) concomitant with a mean increase in lean body mass of 2.2 kg (P < 0.05) was seen. Significant changes in body composition were only seen in the patients with the PWS genotype. Lipid profiles were normal in most patients before treatment and did not change. OGTT was impaired in five patients at 12 months, but two of these patients increased in fat mass. Insulin levels were unchanged. According to homeostasis model assessment (HOMA), insulin resistance did not change. Side-effects attributed to water retention occurred in three patients, one of whom had to be given increased diuretic therapy.

Conclusion: This study shows beneficial effects of GH treatment on body composition in adult PWS patients without significant side-effects. Consequently, further studies are encouraged.

Publication types

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

MeSH terms

  • Adipose Tissue / drug effects
  • Adolescent
  • Adult
  • Body Composition / drug effects*
  • Body Mass Index
  • Drug Administration Schedule
  • Female
  • Genotype
  • Glucose Tolerance Test
  • Homeostasis / physiology
  • Human Growth Hormone / adverse effects
  • Human Growth Hormone / therapeutic use*
  • Humans
  • Insulin Resistance / physiology
  • Insulin-Like Growth Factor I / analysis
  • Lipids / analysis
  • Male
  • Methylation
  • Prader-Willi Syndrome / drug therapy*
  • Prader-Willi Syndrome / physiopathology
  • Weight Gain / drug effects


  • Lipids
  • Human Growth Hormone
  • Insulin-Like Growth Factor I