Long-term effects of growth hormone therapy on patients with Prader-Willi syndrome.

AIM

To assess the effects of recombinant human growth hormone (rhGH) treatment in children with Prader-Willi syndrome.

DESIGN

A 1-year study and an observational follow-up visit 10 years later.

METHODS

In 20 patients with Prader-Willi syndrome (PWS): clinical assessment, laboratory tests, body composition analysis by dual energy X-ray absorptiometry, sleep polygraphy, health-related quality of life assessed by 16D.

RESULTS

Only two patients had normal growth hormone secretion at baseline. All patients were significantly shorter than their expected heights, but experienced catch-up growth during growth hormone treatment. At follow-up, 13 patients had reached adult heights and were markedly taller than historical controls. The cumulative dose of rhGH over 10 years correlated inversely with the total body fat percentage (p = 0.033). However, patients remained severely obese at 10 years. Sleep polygraphy was abnormal in more than half of the patients. Health-related quality of life of the patients remained substantially below that of normal population.

CONCLUSION

Growth hormone markedly improved adult height in subjects with PWS when compared to historical data. The cumulative dose of growth hormone correlated with reduction in body fat; nevertheless, patients remained severely obese.