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

Dutch Growth Research Foundation (R.F.A.d.L.v.W., E.P.C.S., D.A.M.F., A.C.S.H.-K.), 3016 AH Rotterdam, The Netherlands; Departments of Pediatric Endocrinology, Erasmus University Medical Center Rotterdam/Sophia Children’s Hospital (R.F.A.d.L.v.W., E.P.C.S., D.A.M.F., S.L.S.D., A.C.S.H.-K.), 3015 GJ Rotterdam, The Netherlands; Radboud University Nijmegen Medical Center (B.J.O.), 6500 HB Nijmegen, The Netherlands; VU University Medical Center (J.R.), 1081 HV Amsterdam, The Netherlands; University Medical Center Groningen/Beatrix Children’s Hospital (G.B.), 9713 GZ Groningen, The Netherlands; Haga Hospitals/Juliana Children’s Hospital (E.C.A.M.H.), 2566 MJ The Hague, The Netherlands; Amsterdam University Medical Center (A.S.P.v.T.), 1105 AZ Amsterdam; Leiden University Medical Center (B.B., J.M.W.), 2333 ZA Leiden, The Netherlands; Department of Pediatrics, Hieronymus Bosch Medical Center (E.G.A.H.v.M., P.E.J.), 5211 NL ‘s-Hertogenbosch, The Netherlands; St. Catharina Hospital (R.J.H.O.), 5623 EJ Eindhoven, The Netherlands; Erasmus University Medical Center Rotterdam/Sophia Children’s Hospital (G.C.B.B.-d.H.), 3015 GJ Rotterdam, The Netherlands; St. Jansdal Hospital (M.v.L.), 3844 DG Harderwijk, The Netherlands; Diaconessen Hospital (D.A.J.P.H.), 2334 CK Leiden, The Netherlands; St. Antonius Hospital (J.J.G.H.-N.), 3527 CE Utrecht, The Netherlands; Medical Center Twente (R.C.F.M.V.), 7511 JX Enschede, The Netherlands; Reinier de Graaf Hospital (B.B.), 2625 AD Delft, The Netherlands; Isala Hospitals (E.J.S.), 8025 AB Zwolle, The Netherlands; and IJsselmeer Hospitals (J.W.P.), 8233 AA Lelystad, The Netherlands

Address all correspondence and requests for reprints to: Roderick de Lind van Wijngaarden, Clinical Research Fellow, Dutch Growth Research Foundation, Erasmus University Medical Center/Sophia Children’s Hospital, Westzeedijk 106, 3016 AH Rotterdam, The Netherlands. E-mail: r.delindvanwijngaarden{at}erasmusmc.nl.

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 (1 mg/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 SDS_PWS).

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. BMISDS_PWS 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/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.