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Long-term growth hormone therapy changes the natural history of body composition and motor function in children with

Aaron L Carrel1, Susan E Myers, Barbara Y Whitman

  • 1Department of Pediatrics, University of Wisconsin, American Family Children's Hospital, 600 Highland Avenue H4-436, Madison, Wisconsin 53792, USA. alcarrel@wisc.edu

The Journal of Clinical Endocrinology and Metabolism
|January 12, 2010
PubMed

Insights

Recombinant human growth hormone (hGH) therapy initiated in infancy significantly improves body composition, motor function, and height in children with Prader-Willi syndrome (PWS). Long-term hGH treatment favorably alters the natural history of PWS, suggesting early intervention is beneficial.

Area of Science:

  • Pediatric Endocrinology
  • Genetics and Genetic Diseases
  • Metabolic Disorders

Background:

  • Prader-Willi syndrome (PWS) is characterized by reduced muscle mass, hypotonia, and growth impairment in children.
  • Previous studies suggest recombinant human growth hormone (hGH) may improve body composition and physical function in PWS, but lack long-term control data.
  • This study assesses the long-term impact of early-life hGH therapy on the natural history of PWS.

Purpose of the Study:

  • To compare height, body composition, and strength in children with PWS receiving long-term hGH therapy versus those untreated.
  • To evaluate the effects of early-onset hGH treatment on the natural progression of PWS.

Main Methods:

  • Forty-eight children with PWS were studied: 21 treated with hGH for 6 years (early initiation) and 27 age-matched controls prior to hGH treatment.
  • Key parameters analyzed included height, percent body fat, lean body mass, carbohydrate/lipid metabolism, and motor strength.
  • Analysis of covariance was used to compare outcomes between the treated and control groups.

Main Results:

  • Children treated with hGH showed significantly lower body fat (36.1% vs. 44.6%), greater height (131 cm vs. 114 cm), and improved motor strength (standing broad jump and sit-ups).
  • hGH therapy also led to improved lipid profiles, with increased HDL cholesterol (58.9 mg/dl vs. 44.9 mg/dl) and decreased LDL cholesterol (100 mg/dl vs. 131 mg/dl).
  • No significant differences were observed in fasting glucose or insulin levels between the groups.

Conclusions:

  • Early initiation of hGH treatment (before 2 years of age) in children with PWS leads to significant improvements in body composition, motor function, height, and lipid profiles.
  • These beneficial effects suggest that long-term hGH therapy favorably modifies the natural history of PWS.
  • The magnitude of these improvements justifies considering hGH initiation during infancy, outweighing potential risks.
Abstract

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