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Keywords:

  • Adult height;
  • Growth;
  • Prader–Willi syndrome;
  • Recombinant human growth hormone

Abstract

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.