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Growth Hormone Treatment for Prader-Willi Syndrome

Maïthé Tauber1, Gwenaelle Diene2, Catherine Molinas3

  • 1Centre de référence du syndrome de Prader-Willi, Hôpital des Enfants, CHU Toulouse, France, 2Axe Pédiatrique du CIC 9302/INSERM. Hôpital des Enfants, Toulouse, France, 3INSERM U1043, Centre de Physiopathologie de Toulouse Purpan, UPS, France,

Pediatric Endocrinology Reviews : PER
|November 1, 2018
PubMed

Insights

Recombinant human growth hormone (rhGH) therapy has transformed Prader-Willi syndrome (PWS) care, improving growth and body composition. Future focus must address co-morbidities not affected by rhGH for comprehensive patient well-being.

Area of Science:

  • Pediatric endocrinology
  • Metabolic disorders
  • Genetics

Background:

  • Prader-Willi syndrome (PWS) is a complex genetic disorder affecting multiple body systems.
  • Recombinant human growth hormone (rhGH) is a key therapeutic agent for PWS, particularly for short stature.
  • The European Marketing Authorization for rhGH in PWS children marked a significant advancement in metabolic and body composition management.

Purpose of the Study:

  • To review the impact of rhGH on children and young adults with PWS.
  • To highlight the role of rhGH in improving linear growth, body composition, and socialization.
  • To identify areas for future research and clinical focus, including co-morbidities and care transition.

Main Methods:

  • Literature review of rhGH treatment in PWS.
  • Analysis of clinical outcomes related to growth, body composition, and socialization.
  • Discussion of PWS pathophysiology and co-morbidities.

Main Results:

  • rhGH treatment has significantly improved linear growth, body composition, and socialization in PWS patients.
  • rhGH has become a cornerstone of PWS management, facilitating multidisciplinary care.
  • The pathophysiology of associated hormonal deficiencies, including GH, is increasingly understood.

Conclusions:

  • rhGH therapy has revolutionized PWS care, offering substantial benefits for growth and metabolic parameters.
  • Addressing co-morbidities like feeding and behavioral issues is crucial for enhancing patient quality of life.
  • Improving the transition of care from adolescence to adulthood remains a significant challenge.

Keywords:
Growth HormonePrader-Willi Syndrome

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