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Long-term effects of recombinant human growth hormone therapy in children with Prader-Willi syndrome
Peter M Wolfgram1, Aaron L Carrel, David B Allen
1Department of Pediatrics, University of Wisconsin School of Medicine & Public Health, Madison, Wisconsin 53792-4108, USA.
Insights
Recombinant human growth hormone (hGH) therapy offers significant benefits for children with Prader-Willi syndrome (PWS), improving growth and metabolic health. Recent evidence suggests these advantages outweigh potential risks, supporting its use in PWS management.
Area of Science:
- Pediatric Endocrinology
- Genetics
- Metabolic Disorders
Background:
- Prader-Willi syndrome (PWS) is a complex genetic disorder affecting growth and metabolism.
- Recombinant human growth hormone (hGH) is used to manage PWS symptoms.
- Assessing the benefit-risk profile of hGH in PWS is crucial.
Purpose of the Study:
- To review recent evidence on the efficacy and safety of hGH in children with PWS.
- To conduct a benefit-risk analysis of hGH therapy for PWS.
- To inform clinical decision-making regarding hGH treatment in PWS.
Main Methods:
- Systematic review of recent studies on hGH therapy in pediatric PWS patients.
- Analysis of reported outcomes including linear growth, body composition, and metabolic parameters.
- Evaluation of adverse events, including sleep-disordered breathing and sudden death.
Main Results:
- hGH therapy consistently improves stature, body composition, and metabolic markers in children with PWS.
- Preliminary data suggest potential cognitive development benefits with hGH treatment.
- Concerns regarding scoliosis progression and sudden death are not substantiated by recent findings; sleep-related cardiovascular function may improve.
Conclusions:
- Recent evidence reinforces the benefits of hGH therapy in PWS, potentially including cognitive aspects.
- hGH therapy can reduce morbidity and enhance function in children with PWS.
- The benefits of hGH treatment for PWS appear to outweigh the associated risks.
Purpose Of Review:
Recombinant human growth hormone (hGH) therapy in children with Prader-Willi syndrome (PWS) improves linear growth, body composition, physical strength and agility, and other metabolic parameters. These benefits must be weighed against potential adverse effects, including rare occurrences of sudden death. This review summarizes recent evidence important to a benefit-risk analysis of hGH use in children with PWS.
Recent Findings:
Studies consistently show that hGH improves stature, body composition, fat percentage and distribution, and other metabolic markers in children with PWS. Preliminary reports of improved cognitive development during hGH have also emerged. Scoliosis progression is influenced by growth rate, but frequency of occurrence and severity are not increased by hGH exposure. PWS genotype does not appear to affect response to hGH. Concerns about hGH-associated sudden death persist, but recent studies show either absence of change in sleep-disordered breathing or improved sleep cardiovascular function during hGH therapy.
Summary:
Recent studies confirm and expand reported benefits of hGH therapy in children with PWS, including a possible salutary role in cognitive development. These findings support previous assertions that hGH can reduce morbidity and improve function in children with PWS, and suggest that potential risks of such treatment are favorably balanced by its benefits.
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