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


METHODS
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.


METHODS
A total of 91 children with PWS (median age, 4.7 yr; interquartile range, 2.1-7.4) participated in the study.


METHODS
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.
de Lind van Wijngaarden
Roderick F A
RF
Dutch Growth Research Foundation, Erasmus University Medical Center/Sophia Children's Hospital, Rotterdam, The Netherlands. r.delindvanwijngaarden@erasmusmc.nl
de Klerk
Luuk W L
LW
Festen
Dederieke A M
DA
Duivenvoorden
Hugo J
HJ
Otten
Barto J
BJ
Hokken-Koelega
Anita C S
AC
D000293:Adolescent; D015992:Body Mass Index; D002648:Child; D002675:Child, Preschool; D018450:Disease Progression; D005260:Female; D019382:Human Growth Hormone / Q000627:therapeutic use*; D006801:Humans; D007223:Infant; D007334:Insulin-Like Growth Factor I / Q000378:metabolism; D008297:Male; D011218:Prader-Willi Syndrome / Q000150:complications*; D011627:Puberty; D011859:Radiography; D012600:Scoliosis / Q000188:drug therapy* / Q000209:etiology / Q000503:physiopathology; D013131:Spine / Q000000981:diagnostic imaging
D019382:Human Growth Hormone; D007334:Insulin-Like Growth Factor I