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, Rotterdam, The Netherlands; Erasmus University Medical Center / Sophia Children's Hospital, Department of Pediatric Endocrinology, Rotterdam, The Netherlands; Erasmus University Medical Center / Sophia Children's Hospital, Department of Pediatric Orthopedic Surgery, Rotterdam, The Netherlands; The Netherlands Institute for Health Sciences, Erasmus University Rotterdam, Rotterdam, The Netherlands; University Medical Center St. Radboud, Department of Pediatric Endocrinology, Nijmegen, The Netherlands.

Publication date & source: 2009-01-21, J Clin Endocrinol Metab., [Epub ahead of print]

Publication type:

CONTEXT: The prevalence of scoliosis in children with Prader-Willi syndrome (PWS) is 30-80%, depending on age. Although reports about effects of growth hormone (GH) treatment on scoliosis in children with PWS are limited, scoliosis is generally considered a contraindication for GH treatment. OBJECTIVE: To study the effects of GH treatment on the onset of scoliosis and curve progression in children with PWS. DESIGN: A multi-center, randomized controlled GH study in infants, prepubertal and pubertal children. Infants and prepubertal children were randomized into a GH-treated group (1.0 mg/m(2).day) and a control group for one and two years, respectively. Pubertal children were randomized to receive somatropin 1.0 or 1.5 mg/m(2).day. Yearly, X-rays of the spine were taken and height, weight, truncal lean body mass (trunkLBM with DXA), and IGF-I were measured. PATIENTS: 91 children with PWS, median (iqr) age 4.7 (2.1-7.4) years. MAIN OUTCOME MEASURES: 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 SDS, and catch-up growth had no adverse effect on the onset of scoliosis or curve progression, also not after adjustment for confounders. HeightSDS, trunkLBM 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.