Effects of 5 years growth hormone treatment in patients with Prader-Willi syndrome.

BACKGROUND: Short-term studies showed favorable effects of growth hormone (GH) treatment in patients with Prader-Willi syndrome (PWS).

AIMS: To evaluate the long-term effects of GH therapy in patients with PWS retrospectively.

PATIENTS AND METHODS: Effects of GH treatment (0.5 IU/kg/w s.c.) for a period of 1 to 5 years were assessed for 37 Japanese patients with PWS aged 3-(9/12) to 21-(3/12) years. Height and weight were expressed as standard deviation scores (SDSs) of Japanese PWS patients. Height velocity, final height, body mass index (BMI) and Rohrer index were also evaluated.

RESULTS: After 1 year of treatment, the mean height velocity improved significantly from 4.32 to 8.69 cm per year (p < 00001). After 5 years of treatment, mean height SDS increased from -0.99 to +0.88 (p = 0.003). Mean final height of treated patients was 158.0 cm in males and 147.7 cm in females. Mean Rohrer index improved from 182 to 164 (p < 0.0001) after 1 year of treatment and stayed stable thereafter.

CONCLUSIONS: Long-term treatment with GH in patients with PWS improved height velocity, height SDS, final height, and the degree of obesity. These data encourage the long-term use of GH in these patients.