Efficacy and Safety of Long-Term Continuous Growth Hormone Treatment in Children with Prader-Willi Syndrome

R.F.A. de Lind van Wijngaarden, E.P.C. Siemensma, D.A.M. Festen, B.J. Otten, E.G.A.H. van Mil, J. Rotteveel, R.J.H. Odink, G.C.B. van Bindels-de Heus, M. Leeuwen, D.A.P.J. Haring, G. Bocca, E.C.A.M. Houdijk, J.J.G. Hoorweg-Nijman, R.C.F.M. Vreuls, P.E. Jira, A.S.P. Trotsenburg, B. Bakker, E. J. Schroor, J.W. Pilon, J.M. WitS.L.S. Drop, A.C.S. Hokken-Koelega, G. C. B. Karen Bindels-de Heus, Danny A. J. P. Haring

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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/m(2).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. BMISDSPWS significantly 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 (1 mg/m(2).d) improves body composition by decreasing fat% SDS and stabilizing LBMSDS and head circumference SDS and normalizes height SDS without adverse effects. Thus, long-term continuous GH treatment is an effective and safe therapy for children with PWS. (J Clin Endocrinol Metab 94: 4205-4215, 2009)
Original languageUndefined/Unknown
Pages (from-to)4205-4215
JournalJournal of clinical endocrinology and metabolism
Issue number11
Publication statusPublished - 2009

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