Long-term effects of growth hormone therapy on patients with Prader–Willi syndrome
Article first published online: 7 JUN 2010
© 2010 Eli Lilly and Company/Journal Compilation © 2010 Foundation Acta Pædiatrica
Volume 99, Issue 11, pages 1712–1718, November 2010
How to Cite
Sipilä, I., Sintonen, H., Hietanen, H., Apajasalo, M., Alanne, S., Viita, A.-M. and Leinonen, E. (2010), Long-term effects of growth hormone therapy on patients with Prader–Willi syndrome. Acta Paediatrica, 99: 1712–1718. doi: 10.1111/j.1651-2227.2010.01904.x
- Issue published online: 7 JUN 2010
- Article first published online: 7 JUN 2010
- Received 23 December 2009; revised 19 May 2010; accepted 26 May 2010.
- Adult height;
- Prader–Willi syndrome;
- Recombinant human growth hormone
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.