Background: Children with Prader-Willi syndrome (PWS) have abnormal body composition and impaired growth. Short-term GH treatment has beneficial effects. Objectives: The aim of the study was to investigate effects of long-term continuous GH treatment on body composition, growth, bone maturation, and safety parameters. Setting: We conducted a multicenter prospective trial. Design: Fifty-five children with a mean ± SD age of 5.9 ± 3.2 yr were followed during 4 yr of continuous GH treatment (1mg/m2·d). Data were annually obtained in one center: fat percentage (fat%) and lean body mass (LBM) by dual-energy x-ray absorptiometry, height, weight, head circumference, bone age, blood pressure, and fasting IGF-I, IGF binding protein-3, glucose, insulin, glycosylated hemoglobin, total cholesterol, high-density lipoprotein, and low-density lipoprotein. SD scores (SDS) were calculated according to Dutch and PWS reference values (SDS and SDSPWS). Results: Fat%SDS was significantly lower after 4 yr of GH treatment (P < 0.0001). LBMSDS significantly increased during the first year (P = 0.02) but returned to baseline values the second year and remained unchanged thereafter. Mean ± SD height normalized from -2.27 ± 1.2 SDS to -0.24 ± 1.2 SDS (P < 0.0001). Head circumference SDS increased from -0.79 ± 1.0 at start to 0.07 ± 1.1 SDS after 4 yr. BMISDSPWSsignificantly decreased. Mean ± SD IGF-I and the IGF-I/IGF binding protein-3 ratio significantly increased to 2.08 ± 1.1 and 2.32 ± 0.9 SDS, respectively. GH treatment had no adverse effects on bone maturation, blood pressure, glucose homeostasis, and serum lipids. Conclusions: Our study in children with PWS shows that 4 yr of continuous GH treatment (1mg/m2·d) improves body composition by decreasing fat% SDS and stabilizing LBMSDS and head circumference SDS and normalizes heightSDS without adverse effects. Thus, long-term continuous GH treatment is an effective and safe therapy for children with PWS. Copyright

Additional Metadata
Persistent URL,
Journal Journal of Clinical Endocrinology and Metabolism
Note Free full text at PubMed
de Lind van Wijngaarden, R.F.A, Siemensma, E.P.C, Festen, D.A.M, Otten, B.J, van Mil, E.G.A.H, Rotteveel, J, … Hokken-Koelega, A.C.S. (2009). Efficacy and safety of long-term continuous growth hormone treatment in children with Prader-Willi syndrome. Journal of Clinical Endocrinology and Metabolism, 94(11), 4205–4215. doi:10.1210/jc.2009-0454