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Three years of growth hormone treatment in young adults with Prader-Willi syndrome: sustained positive effects on body composition.

ABSTRACT: BACKGROUND: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 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. METHODS:Open-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. RESULTS:Estimated 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. CONCLUSIONS:Three 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 REGISTRATION:EudraCT, EudraCT number 2011-001313-14. Registered 17 October 2012.

SUBMITTER: Damen L

PROVIDER: S-EPMC7313113 | biostudies-literature | 2020 Jun

REPOSITORIES: biostudies-literature

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Three years of growth hormone treatment in young adults with Prader-Willi syndrome: sustained positive effects on body composition.

Damen Layla L Donze Stephany H SH Kuppens Renske J RJ Bakker Nienke E NE de Graaff Laura C G LCG van der Velden Janielle A E M JAEM Hokken-Koelega Anita C S ACS

Orphanet journal of rare diseases 20200624 1

<h4>Background</h4>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 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/m<sup>2</sup>/day.<h4>Methods</h4>Open-label, p ...[more]

PMID: 32580778

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Project description:BackgroundPrader-Willi syndrome (PWS) is a rare complex genetic disorder and is characterized by short stature, muscular hypotonia, abnormal body composition, psychomotor retardation, and hyperphagia. Recombinant human growth hormone (rhGH) treatment improves the symptoms in children with PWS, and early treatment results in more favorable outcomes. However, systematic studies in infants and toddlers under 2 years of age are lacking. This multicenter, randomized, active-controlled, parallel-group, open-label, Phase III study aimed to evaluate the safety of rhGH (Eutropin, LG Chem, Ltd.) and its efficacy on growth, body composition, and motor and cognitive development in infants and toddlers with PWS compared with a comparator treatment (Genotropin, Pfizer, Inc.). Eligible Korean infants or toddlers with PWS were randomly assigned to receive Eutropin or comparator (both 0.24 mg/kg/week, 6 times/week) for 1 year. Height standard deviation score (SDS), body composition, and motor and cognitive development were measured.ResultsThirty-four subjects (less than 24 months old) were randomized into either the Eutropin (N = 17) group or the comparator (N = 17) group. After 52 weeks of rhGH treatment, height SDS and lean body mass increased significantly from baseline in both groups: the mean height SDS change (SD) was 0.75 (0.59) in the Eutropin group and 0.95 (0.66) in the comparator group, and the mean lean body mass change (SD) was 2377.79 (536.25) g in the Eutropin group and 2607.10 (641.36) g in the comparator group. In addition, percent body fat decreased significantly: the mean (SD) change from baseline was - 8.12% (9.86%) in the Eutropin group and - 7.48% (10.26%) in the comparator group. Motor and cognitive developments were also improved in both groups after the 1-year treatment. The incidence of adverse events was similar between the groups.ConclusionsrhGH treatment for 52 weeks in infants and toddlers with PWS improved growth, body composition, and motor and cognitive development, and efficacy and safety outcomes of Eutropin were comparable to those of Genotropin. Hence, Eutropin is expected to provide safe and clinically meaningful improvements in pediatric patients with PWS.Trial registrationThe study was registered at ClinicalTrials.gov (identifier: NCT02204163) on July 30, 2014. URL: https://clinicaltrials.gov/ct2/show/NCT02204163?term=NCT02204163&rank=1.

Dataset Information

Three years of growth hormone treatment in young adults with Prader-Willi syndrome: sustained positive effects on body composition.

Publications

Three years of growth hormone treatment in young adults with Prader-Willi syndrome: sustained positive effects on body composition.

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