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Published on: January 7, 2016
Growth hormone improves mobility and body composition in infants and toddlers with Prader-Willi syndrome
Aaron L Carrel1, Victoria Moerchen, Susan E Myers
1Department of Pediatrics, University of Wisconsin Children's Hospital, Madison, USA. alcarrel@wisc.edu
Insights
Growth hormone (GH) therapy in infants and toddlers with Prader-Willi syndrome (PWS) improves body composition and motor skills. Early GH treatment may mitigate body composition changes and enhance motor development in PWS patients.
Area of Science:
- Pediatrics
- Endocrinology
- Genetics
Background:
- Prader-Willi syndrome (PWS) is a complex genetic disorder affecting multiple body systems.
- Infants and toddlers with PWS often experience challenges with growth, body composition, and motor development.
Purpose of the Study:
- To evaluate the impact of growth hormone (GH) treatment on body composition and motor development in young children with PWS.
- To assess if early initiation of GH therapy influences outcomes in PWS.
Main Methods:
- A randomized controlled trial involving 29 infants and toddlers (4-37 months) with PWS.
- Participants were assigned to either GH treatment (1mg/m²/day) or observation for 12 months.
- Body composition (DXA), energy expenditure (deuterium dilution), and motor skills (TIME) were assessed.
Main Results:
- GH treatment significantly decreased body fat percentage and increased lean body mass and height velocity Z scores.
- Subjects receiving GH showed improved mobility skill acquisition, particularly when treatment began before 18 months of age.
- These improvements were statistically significant compared to the control group.
Conclusions:
- Twelve months of GH treatment significantly enhances body composition in infants and toddlers with PWS.
- Initiating GH therapy before 18 months of age accelerates motor development, specifically mobility skills.
- Early GH intervention shows potential to mitigate PWS-related body composition deterioration and promote motor development.
Objectives:
To determine the effect of growth hormone (GH) on body composition and motor development in infants and toddlers with Prader-Willi syndrome (PWS).
Study Design:
Twenty-nine subjects with PWS (4-37 months of age) were randomized to GH treatment (1mg/m 2 /day) or observation for 12 months. Percent body fat, lean body mass, and bone mineral density were measured by dual x-ray absorptiometry; energy expenditure was measured by deuterium dilution; and motor constructs of mobility (M) and stability (S) were assessed using the Toddler Infant Motor Evaluation (TIME).
Results:
GH-treated subjects, compared with controls, demonstrated decreased percent body fat (mean, 22.6% +/- 8.9% vs 28.5% +/- 7.9%; P < .001), increased lean body mass (mean, 9.82 +/- 1.9 kg vs 6.3 +/- 1.9 kg; P < .001), and increased height velocity Z scores (mean, 5. 0 +/- 1.8 vs 1.4 +/- 1.0; P < .001). Patients who began GH before 18 months of age showed higher mobility skill acquisition compared with controls within the same age range (mean increase in raw score, 284 +/- 105 vs 206 +/- 63; P < .05).
Conclusions:
GH treatment of infants and toddlers with PWS for 12 months significantly improves body composition and when begun before 18 months of age increases mobility skill acquisition. These results suggest that GH therapy instituted early in life may lessen deterioration of body composition in PWS while also accelerating motor development.
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