Related Experiment Videos
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
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.
Abstract:
A randomized, controlled study of 54 children (age, 4-16 years) with Prader-Willi syndrome was conducted to assess the potential beneficial effects of growth hormone (GH) treatment. After observation for 6 months, the children were randomized to receive GH at a dose of 3 IU/m2/day (1 mg/m2/day) (n = 35) or no intervention (n = 19). The effects of GH treatment on linear growth, body composition, muscle strength, pulmonary function and resting energy expenditure were assessed. The levels of GH secreted in response to clonidine stimulation were universally low, and mean (+/- SD) insulin-like growth factor I SDS was -1.2 +/- 0.8 pretreatment. In children treated for 1 year, mean height velocity SDS significantly increased from -1.0 +/- 2.5 to 4.6 +/- 2.9 (p < 0.0001), mean percentage body fat decreased from 46.3 +/- 8.4% to 38.4 +/- 10.7% (p < 0.001), mean lean body mass increased from 20.5 +/- 6.3 kg to 25.6 +/- 4.3 kg (p < 0.01) and respiratory muscle function and physical strength improved. Mean respiratory quotients significantly decreased from 0.81 to 0.77 (p < 0.001); however, resting energy expenditure did not change. Therefore, GH therapy appears to reduce some of the physical disabilities experienced by children with Prader-Willi syndrome.