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Outcomes in children treated with growth hormone for Prader-Willi syndrome: data from the ANSWER Program® and
Moris Angulo1, M Jennifer Abuzzahab2, Alberto Pietropoli3
1NYU Winthrop Hospital, Mineola, NY, USA.
Insights
Growth hormone (GH) therapy improves height and body mass index (BMI) in children with Prader-Willi syndrome (PWS). Early and extended GH treatment leads to better outcomes for PWS patients.
Area of Science:
- Pediatric Endocrinology
- Metabolic Disorders
- Genetics and Rare Diseases
Background:
- Growth hormone (GH) deficiency is a common complication in Prader-Willi syndrome (PWS), often resulting in short adult stature.
- Real-world data is crucial for understanding the clinical outcomes of GH treatment in pediatric PWS patients.
Purpose of the Study:
- To assess the clinical effectiveness of somatropin (GH) in pediatric patients with PWS.
- To identify factors influencing changes in height standard deviation scores (HSDS) and body mass index standard deviation scores (BMI SDS) during GH therapy.
Main Methods:
- Observational data from the ANSWER Program and NordiNet International Outcome Study (2006-2016) were analyzed.
- Included patients were treatment-naïve to GH before initiating somatropin therapy.
- Statistical analysis determined variables affecting changes in HSDS (n=129) and BMI SDS (n=98).
Main Results:
- Mean GH dose was 0.03 mg/kg/d.
- Baseline age and duration of GH treatment significantly impacted HSDS changes.
- Longer GH treatment duration correlated with greater BMI SDS changes, and higher baseline BMI led to greater BMI reduction over time.
Conclusions:
- Somatropin (GH) is effective for managing PWS in children.
- Earlier initiation of GH therapy is associated with greater height gains.
- Extended GH treatment duration improves both height and BMI outcomes in pediatric PWS patients.
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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