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/m2 · d) and a control group for 1 and 2 yr, respectively. Pubertal children were randomized to receive somatropin 1.0 or 1.5 mg/m2 · 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°) 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.

Additional Metadata
Keywords Prader Willi syndrome, adolescent, article, catch up growth, child, clinical trial, controlled clinical trial, controlled study, disease severity, drug effect, dual energy X ray absorptiometry, female, human, human growth hormone, infant, lean body weight, major clinical study, male, multicenter study, outcome assessment, preschool child, priority journal, randomized controlled trial, school child, scoliosis, somatomedin C
Persistent URL dx.doi.org/10.1210/jc.2008-1844, hdl.handle.net/1765/16430
Citation
de Lind van Wijngaarden, R.F.A., Klerk, L.W.L., Festen, D.A.M., Duivenvoorden, H.J., Otten, B.J., & Hokken-Koelega, A.C.S.. (2009). Randomized controlled trial to investigate the effects of growth hormone treatment on scoliosis in children with prader-willi syndrome. Journal of Clinical Endocrinology and Metabolism, 94(4), 1274–1280. doi:10.1210/jc.2008-1844