Abstract
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
Highlights
Prader-Willi syndrome (PWS) is a rare complex genetic disorder and is characterized by short stature, muscular hypotonia, abnormal body composition, psychomotor retardation, and hyperphagia
PWS is characterized by muscular hypotonia, failure to thrive in infancy, short stature, psychomotor retardation, and hyperphagia resulting in severe obesity, as well as hypothalamic dysfunction, which may become apparent in later childhood [4,5,6]
Patients The following patients were included in this study: 1) prepubertal pediatric patients with PWS confirmed using methylation polymerase chain reaction (PCR) genetic testing; 2) pediatric patients naïve to Recombinant human growth hormone (rhGH) treatments or previously treated with rhGH for less than 6 months; 3) pediatric patients without other causes for growth retardation except for PWS; 4) pediatric patients who were not being administered any drug that may have an effect on the secretion and actions of growth hormone (GH), anticonvulsants, or cyclosporin at screening, and who had not been administered any of these drugs within 6 months prior to screening
Summary
Prader-Willi syndrome (PWS) is a rare complex genetic disorder and is characterized by short stature, muscular hypotonia, abnormal body composition, psychomotor retardation, and hyperphagia. 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.). Patients with PWS have a peculiar body composition with a high body fat mass percentage and a low lean body mass (LBM) This has been observed even in underweight infants with PWS [7, 8]. Recombinant human GH (rhGH) treatment for improvement of the symptoms in children with PWS has been available and regularized since rhGH preparations were approved for use in children with PWS by the US FDA in 2000 and the EMA in 2001 [11]
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