Randomized controlled trial to investigate the effects of growth hormone treatment on scoliosis in children with Prader-Willi syndrome.

Author(s): de Lind van Wijngaarden RF, de Klerk LW, Festen DA, Duivenvoorden HJ, Otten BJ, Hokken-Koelega AC

Affiliation(s): Dutch Growth Research Foundation, Erasmus University Medical Center/Sophia Children's Hospital, Rotterdam, The Netherlands. r.delindvanwijngaarden@erasmusmc.nl

Publication date & source: 2009-04, J Clin Endocrinol Metab., 94(4):1274-80. Epub 2009 Jan 21.

Publication type: Multicenter Study; Randomized Controlled Trial

CONTEXT: The prevalence of scoliosis in children with Prader-Willi syndrome (PWS) is 30-80%, depending on age. Although reports about effects of GH treatment on scoliosis in children with PWS are limited, scoliosis is generally considered a contraindication for GH treatment. OBJECTIVE: The aim was to study the effects of GH treatment on the onset of scoliosis and curve progression in children with PWS. DESIGN: We conducted a multicenter, randomized, controlled GH study in infants and prepubertal and pubertal children. Infants and prepubertal children were randomized into a GH-treated group (1.0 mg/m(2) . d) and a control group for 1 and 2 yr, respectively. Pubertal children were randomized to receive somatropin 1.0 or 1.5 mg/m(2) . d. Yearly, x-rays of the spine were taken, and height, weight, truncal lean body mass (with dual energy x-ray absorptiometry), and IGF-I were measured. PATIENTS: A total of 91 children with PWS (median age, 4.7 yr; interquartile range, 2.1-7.4) participated in the study. MAIN OUTCOME MEASURES: We measured the onset of scoliosis (Cobb >10 degrees ) and scoliotic curve progression. RESULTS: GH-treated children had similar onset of scoliosis and curve progression as randomized controls (P = 0.27-0.79 and P = 0.18-0.98, respectively). GH treatment, IGF-I sd score (SDS), and catch-up growth had no adverse effect on the onset of scoliosis or curve progression, even after adjustment for confounders. Height SDS, truncal lean body mass, and IGF-I SDS were significantly higher in GH-treated children than in randomized controls. At baseline, a higher IGF-I SDS was associated with a lower severity of scoliosis. CONCLUSIONS: Scoliosis should no longer be considered a contraindication for GH treatment in children with PWS.