Growth hormone treatment completely normalizes adult height and improves body composition in Prader-Willi syndrome: experience from KIGS (Pfizer International Growth Database)

Horm Res. 2008;70(3):182-7. doi: 10.1159/000145019. Epub 2008 Jul 29.

Abstract

Background: Abnormal body composition, with low muscle mass and increased fat mass, as well as short adult stature are common features in Prader-Willi syndrome (PWS), as in growth hormone (GH) deficiency.

Methods: We followed a cohort of 22 genetically verified patients with PWS from the start of GH (Genotropin) treatment at the median age of 6.9 years (4.9-11.3) to near-adult height at 18.1 years (16.4-21.2). The patients were treated with a median GH dose of 0.03 mg/kg/day (0.02-0.03) for a median duration of 10.2 years (6.9-11.5).

Results: All patients reached near-adult height within midparental height median -0.5 SDS (-1.4 to 0.7) and 0.9 SDS (0.1-1.9) for girls and boys, respectively. The body composition improved but did not normalize. Only 7 of the 22 patients were reported to be in puberty. None of the patients were reported to be on sex hormone substitution which might contribute to not reaching a normal body composition. No serious side effects were reported when the caloric intake was controlled to maintain an appropriate body weight.

Conclusion: GH treatment in children with Prader-Wili syndrome normalizes adult height and improves body composition.

Publication types

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

MeSH terms

  • Absorptiometry, Photon
  • Body Composition / drug effects*
  • Body Height / drug effects*
  • Child
  • Child, Preschool
  • Databases, Factual
  • Female
  • Human Growth Hormone / therapeutic use*
  • Humans
  • Longitudinal Studies
  • Male
  • Prader-Willi Syndrome / drug therapy*
  • Prader-Willi Syndrome / physiopathology

Substances

  • Human Growth Hormone