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Physical effects of growth hormone treatment in children with Prader-Willi syndrome

S E Myers1, A L Carrel, B Y Whitman

  • 1Department of Pediatrics, Saint Louis University, Missouri, USA. myersse@slu.edu

Acta Paediatrica (Oslo, Norway : 1992). Supplement
|January 8, 2000
PubMed

Insights

Growth hormone (GH) therapy significantly improved linear growth, body composition, and muscle strength in children with Prader-Willi syndrome. This treatment reduced physical disabilities, offering substantial benefits for affected individuals.

Area of Science:

  • Pediatrics
  • Endocrinology
  • Genetics

Background:

  • Prader-Willi syndrome (PWS) is a complex genetic disorder characterized by hyperphagia, developmental delay, and distinct physical features.
  • Children with PWS often exhibit impaired growth and reduced muscle mass, contributing to physical disabilities.

Purpose of the Study:

  • To evaluate the efficacy of growth hormone (GH) treatment in improving linear growth, body composition, muscle strength, pulmonary function, and resting energy expenditure in children with PWS.

Main Methods:

  • A randomized controlled trial involving 54 children (aged 4-16 years) with PWS.
  • Participants were randomized to receive GH (3 IU/m²/day) or no intervention after a 6-month observation period.
  • Assessment of linear growth, body composition (fat mass, lean mass), muscle strength, pulmonary function, and resting energy expenditure over 1 year.

Main Results:

  • GH treatment significantly increased height velocity SDS and lean body mass, while decreasing body fat percentage.
  • Improvements were observed in respiratory muscle function and physical strength.
  • No significant change in resting energy expenditure was noted, though respiratory quotients decreased.

Conclusions:

  • Growth hormone therapy is effective in improving key physical parameters in children with Prader-Willi syndrome.
  • GH treatment can mitigate some of the physical disabilities associated with PWS, enhancing quality of life.

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