Retrospective study of the potential benefits and adverse events during growth hormone treatment in children with Prader-Willi syndrome.

Objective: To assess the effectiveness of growth hormone (GH) treatment of children with Prader-Willi syndrome (PWS) in clinical practice.

Study Design: This was a review of 23 patients with PWS (14 males, 9 females) under age 18 years, 10 of whom (5 males, 5 females) had been treated with GH for periods between 0.1 and 5.5 years. All of these patients had a GH level < 5.5 microg/L on 2 GH stimulation tests.

Results: In the 8 patients treated with GH for more than 1 year, median height velocity was 8.6 cm/year (range, 2.0 to 14.5 cm/year) during the first year, greater than that in the no-GH group (5.5 cm/year [range, 0.8 to 7.0 cm/year]) (P < .05). The evolution of body mass index (BMI) was similar in both groups, however (GH group: 3.1 standard deviation score [SDS; range, -2.5 to +6.7] at GH initiation and 3.3 SDS [range, -0.4 to +8.9] at last visit; no-GH group: 3.2 SDS [range, -0.3 to +6.4] at first visit and 2.6 SDS [range, -0.1 to +6.4] at last visit). In 3 patients treated with GH, sequential body composition analysis by dual-energy X-ray absorptiometry revealed no benefit. In both groups, stabilization or diminution of BMI was more often observed in children of highly educated parents. Two of the 10 patients treated with GH developed obstructive sleep apnea (OSA) 1 to 2 months after starting GH, 1 of whom died (reported previously).

Conclusions: GH therapy in children with PWS in the clinical setting did not lead to any discernible improvement in BMI or body composition and appeared to be associated with OSA. Regardless of GH therapy, parental education was associated with better outcome.