Abstract
BackgroundGrowth 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.MethodsData 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.ResultsPatients 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.ConclusionsGH 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 registrationThis study was registered with ClinicalTrials.gov (NCT01009905) on November 9, 2009.
Highlights
Growth hormone (GH) deficiency is common in patients with Prader-Willi syndrome (PWS) and leads to short adult stature
Based on availability of follow-up data to perform the current analyses, a total of 129 patients were included in the height standard deviation scores (HSDS) analysis and 98 in the Body mass index (BMI) Standard deviation score (SDS) analysis
The findings demonstrated that patients in both the American Norditropin® Studies: Web-Enabled Research (ANSWER) Program® and NordiNet® International Outcome Study (IOS) are expected to experience similar increases in HSDS over time
Summary
Growth hormone (GH) deficiency is common in patients with Prader-Willi syndrome (PWS) and leads to short adult stature. Prader-Willi syndrome (PWS) is a rare multisystemic genetic disorder that arises from the lack of expression of paternally inherited genes known to be imprinted and located in the 15q11-q13 region. Children with PWS who have GH deficiency do not experience height gain acceleration typical of puberty, leading to short adult stature Approved by the US Food and Drug Administration (FDA) for the treatment of PWS in 2000 [3], rhGH demonstrated efficacy in increasing short-term growth and adult height in patients with a range of growth disorders [10,11,12,13,14,15]. The most recent clinical guidelines for the use of rhGH for PWS recommend that infants and children begin with a GH dose of 0.5 mg/m2/d, incrementally titrating every 3 to 6 months based on clinical response toward a dose of 1.0 mg/m2 [2]
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