The objective of this study was to evaluate the effects and adverse side-effects of growth hormone (GH) therapy in children with Prader-Willi syndrome (PWS).

Forty-one patients who had been treated with GH for more than two years (24 boys and 17 girls, mean age 7.3±3.3 years during treatment initiation) were enrolled for this study.

After 2 years of GH therapy, the height and weight standard deviation scores (SDS) increased significantly (1.19±1.37 vs. 0.02±1.45, and 1.02±2.42 vs. 1.63±2.22, P <0.002); however the percentage body fat decreased (44.6±9.9% vs. 38.1±10.5%, P <0.001). Further, no change was observed in the thyroid and serum glucose levels, but the total cholesterol level decreased. GH therapy did not impact glucose control in the patients with diabetes. The most common adverse effects of GH therapy were the progression of scoliosis and adenoid hypertrophy.

GH therapy improved the height SDS and body composition in patients with PWS. However, GH should be used with caution in patients with scoliosis and adenoid hypertrophy.