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


Dr. Ilkka Sipilä, M.D., Ph.D., Hospital for Children and Adolescents, Paediatrics, University of Helsinki, Hernesaarenkatu 7 B 16b, 00150 Helsinki, Finland. Tel: +358 40 5502 551 | Fax: +358 40 5502 551 | Email:


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.

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