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- Title
Outcomes in children treated with growth hormone for Prader-Willi syndrome: data from the ANSWER Program® and NordiNet® International Outcome Study.
- Authors
Angulo, Moris; Abuzzahab, M. Jennifer; Pietropoli, Alberto; Ostrow, Vlady; Kelepouris, Nicky; Tauber, Maithe
- Abstract
Background: Growth hormone (GH) deficiency is common in patients with Prader-Willi syndrome (PWS) and leads to short adult stature. The current study assessed clinical outcomes based on real-world observational data in pediatric patients with PWS who were treated with GH. Methods: Data from patients previously naïve to treatment with GH who began therapy with somatropin were collected from 2006 to 2016 in the observational American Norditropin® Studies: Web-Enabled Research (ANSWER) Program® and NordiNet® International Outcome Study. Variables affecting change from baseline in height standard deviation scores (HSDS; n = 129) and body mass index standard deviation scores (BMI SDS; n = 98) were determined. Results: Patients included in both HSDS and BMI SDS analyses were treated with a mean GH dose of 0.03 mg/kg/d (SD, 0.01 mg/kg/d). Results from the HSDS analysis revealed that baseline age and years on treatment had a significant impact on the change in HSDS. In the BMI SDS analysis, longer GH treatment time led to a greater change in BMI SDS from baseline, and patients with a higher BMI at the start of treatment had a greater decrease in BMI over time. Conclusions: GH is effective in the management of children with PWS. Earlier treatment resulted in a greater gain in height, and a longer treatment period resulted in better outcomes for both height and BMI. Trial registration: This study was registered with ClinicalTrials.gov (NCT01009905) on November 9, 2009.
- Subjects
SOMATOTROPIN; PRADER-Willi syndrome; GROWTH of children; CHILD patients; BODY mass index
- Publication
International Journal of Pediatric Endocrinology, 2020, Vol 2020, Issue 1, pN.PAG
- ISSN
1687-9848
- Publication type
Article
- DOI
10.1186/s13633-020-00090-6