Abstract
BackgroundIn children with Prader-Willi syndrome (PWS), the benefits of growth hormone treatment are well established. Several one-year studies have shown that growth hormone is also beneficial for adults with PWS, improving body composition. However, little is known about the longer-term effects.This study investigated the effects on body composition in adult patients with PWS during 3 years of growth hormone therapy in a dose of 0.33 mg/m2/day.MethodsOpen-label, prospective study in 43 young adults with PWS with a median (IQR) age of 19.0 (17.5 to 20.7) years. Fat mass percentage SDS and lean body mass SDS were measured annually by DXA.ResultsEstimated mean (95% CI) fat mass percentage SDS decreased during the three-year study from 2.1 (1.9 to 2.3) SDS at start to 1.9 (1.8 to 2.1) SDS, p = 0.012, while lean body mass SDS remained stable at − 2.1 (− 2.4 to − 1.8) SDS at start to − 1.9 (− 2.3 to − 1.6) after 3 years, p = 0.15. Fasting glucose and insulin remained similar during the three-year study, glucose being 4.6 (4.4 to 4.8) mmol/l at start and 4.6 (4.5 to 4.7) mmol/l after 3 years of growth hormone, p = 0.93 and insulin being 59.5 (42.2 to 81.5) pmol/l and 55.0 (42.4 to 69.2) pmol/l, resp., p = 0.54. There were no growth hormone-related adverse events during the study.ConclusionsThree years of growth hormone treatment in young adults with PWS maintains the positive effects on body composition attained during childhood. Thus, adults with PWS benefit from longer-term growth hormone treatment.Trial registrationEudraCT, EudraCT number 2011-001313-14. Registered 17 October 2012.
Highlights
In children with Prader-Willi syndrome (PWS), the benefits of growth hormone treatment are well established
We report the effects of 3 years of continuous growth hormone (GH) treatment with a stable GH dose on body composition in adults with PWS, who were treated with GH during childhood
The Young Adult PWS (YAP) Study was started in 2011 to evaluate the longer-term effects and safety of GH treatment in young adults with PWS who were treated with GH during childhood
Summary
In children with Prader-Willi syndrome (PWS), the benefits of growth hormone treatment are well established. Several one-year studies have shown that growth hormone is beneficial for adults with PWS, improving body composition. This study investigated the effects on body composition in adult patients with PWS during 3 years of growth hormone therapy in a dose of 0.33 mg/m2/day. Prader-Willi syndrome (PWS) is a rare genetic disorder caused by the lack of expression of the imprinted genes on the Prader-Willi region of the paternally inherited chromosome 15. This is mostly caused by a paternal deletion or maternal uniparental disomy (mUPD) and in some cases by an imprinting center defect (ICD) or paternal chromosomal translocation [1, 2]. GH treatment has substantially changed the phenotype of children with PWS [5, 13]
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