Background: Prader-Willi syndrome (PWS) children have impaired growth, and abnormal body composition. Previous 1-year controlled studies showed improvement of height and body composition during GH-treatment. Objective: To evaluate growth, body composition and body proportions during GH-treatment in a large group of PWS children. Design/patients: We performed a randomized controlled GH trial in 91 prepubertal PWS children (42 infants, 49 children, aged 3-14 years). After stratification for age, infants were randomized to GH-treatment (GH-group; 1 mg/m2/day; n = 20), or no treatment (control group; n = 22) for 1 year. In the second year all infants were treated with GH. After stratification for BMI, children > 3 years of age were randomized to GH-treatment (GH-group; 1 mg/m2/day; n = 27) or no treatment (control group; n = 22) for 2 years. Anthropometric parameters were assessed once in every 3 months. Body composition was measured by Dual Energy X-ray Absorptiometry. Results: Median (interquartile range, iqr) height SDS increased during 2 years of GH in infants from -2.3 (-2.8 to -0.7) to -0.4 (-1.1-0.0) and in prepubertal children from -2.0 (-3.1 to -1.7) to -0.6 (-1.1 to -0.1). In non-GH-treated children height SDS did not increase. Head circumference completely normalized during 1 and 2 years of GH in infants and children, respectively. Body fat percentage and body proportions improved in GH-treated children, but did not completely normalize. Lean body mass SDS improved compared to the control group. Serum IGF-I increased to levels above the normal range in most GH-treated children. Conclusions: Our randomized study shows that GH-treatment in PWS children significantly improves height, BMI, head circumference, body composition and body proportions. PWS children are highly sensitive to GH, suggesting that monitoring of serum IGF-I is indicated.

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Persistent URL dx.doi.org/10.1111/j.1365-2265.2008.03228.x, hdl.handle.net/1765/29688
Citation
Festen, D.A.M, de Lind van Wijngaarden, R.F.A, van Eekelen, M, Otten, B.J, Wit, J.M, Duivenvoorden, H.J, & Hokken-Koelega, A.C.S. (2008). Randomized controlled GH trial: Effects on anthropometry, body composition and body proportions in a large group of children with Prader-Willi syndrome. Clinical Endocrinology, 69(3), 443–451. doi:10.1111/j.1365-2265.2008.03228.x