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

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

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.

Keywords: Body height; Body mass index; Growth disorders; Human growth hormone; Registries.

Conflict of interest statement

MA has nothing to disclose. MJA has received research support from AstraZeneca. She also serves as a research investigator for Novo Nordisk, Ascendis, Soleno, Levo, Millendo, and Rhythm; as a speaker for Novo Nordisk and Rhythm; and as an advisory board member for Ascendis and Rhythm. AP, VO, and NK are employees of Novo Nordisk. MT is an advisory board member for Novo Nordisk, Pfizer, Ipsen, and Merck Serono, a consultant for Alizé Pharma, a recipient of grants from Novo Nordisk, Pfizer, Ipsen, and Sandoz, a research investigator for Novo Nordisk and Alizé Pharma, and a speaker for Novo Nordisk, Pfizer, Ipsen, and Merck Serono.

Figures

Fig. 1
Fig. 1
Model-based mean estimates for change from baseline over time in HSDS in patients with the diagnosis of PWS in both the ANSWER Program® and NordiNet® IOS. Abbreviations: HSDS, height standard deviation score; PWS, Prader-Willi syndrome
Fig. 2
Fig. 2
Model-based mean estimates for change from baseline over time in BMI SDS in patients with the diagnosis of PWS in both the ANSWER Program® and NordiNet® IOS. Abbreviations: BMI, body mass index; PWS, Prader-Willi syndrome; SDS, standard deviation score

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