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International Journal of Pediatric Endocrinology

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Open Access 01-12-2020 | Research

Outcomes in children treated with growth hormone for Prader-Willi syndrome: data from the ANSWER Program® and NordiNet® International Outcome Study

Authors: Moris Angulo, M. Jennifer Abuzzahab, Alberto Pietropoli, Vlady Ostrow, Nicky Kelepouris, Maithe Tauber

Published in: International Journal of Pediatric Endocrinology | Issue 1/2020

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.

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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: Moris Angulo
M. Jennifer Abuzzahab
Alberto Pietropoli
Vlady Ostrow
Nicky Kelepouris
Maithe Tauber
Publication date: 01-12-2020
Publisher: BioMed Central
Published in: International Journal of Pediatric Endocrinology / Issue 1/2020
Electronic ISSN: 1687-9856
DOI: https://doi.org/10.1186/s13633-020-00090-6

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Outcomes in children treated with growth hormone for Prader-Willi syndrome: data from the ANSWER Program® and NordiNet® International Outcome Study

Abstract

Background

Methods

Results

Conclusions

Trial registration

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Abstract

Background

Methods

Results

Conclusions

Trial registration

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