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.
RCT Entities:
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.
Authors: Jennifer Proffitt; Kathryn Osann; Barbara McManus; Virginia E Kimonis; Janalee Heinemann; Merlin G Butler; David A Stevenson; June-Anne Gold Journal: Am J Med Genet A Date: 2018-12-19 Impact factor: 2.802
Authors: Merlin G Butler; Jaehoon Lee; Ann M Manzardo; June-Anne Gold; Jennifer L Miller; Virginia Kimonis; Daniel J Driscoll Journal: Pediatrics Date: 2014-12-08 Impact factor: 7.124