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Time for a general approval of growth hormone treatment in adults with Prader-Willi syndrome

Charlotte Höybye1, Anthony J Holland2, Daniel J Driscoll3

  • 1Department of Endocrinology, Karolinska University Hospital and Department of Molecular Medicine and Surgery, Karolinska Institute, Stockholm, Sweden.

Orphanet Journal of Rare Diseases
|February 9, 2021
PubMed

Insights

Growth hormone (GH) treatment benefits children with Prader-Willi syndrome (PWS). This commentary advocates for international approval of GH therapy for PWS adults, emphasizing its positive effects on body composition and quality of life.

Area of Science:

  • Endocrinology
  • Genetics
  • Neurodevelopmental Disorders

Background:

  • Prader-Willi syndrome (PWS) is a complex neurodevelopmental disorder with characteristic features including hypotonia, obesity risk, and hormonal deficiencies.
  • Growth hormone (GH) deficiency is common in PWS due to hypothalamic dysfunction, impacting growth and body composition.
  • GH treatment is established for children with PWS, improving height, psychomotor development, and body composition.

Discussion:

  • In adults with PWS, GH treatment maintains normal body structure and metabolism.
  • Studies confirm GH's positive impact on body composition, fitness, cardiovascular risk markers, behavior, and quality of life in adults with PWS.
  • Current international approval for GH treatment in PWS primarily focuses on children, with limited access for adults.

Key Insights:

  • GH treatment offers significant benefits for adults with Prader-Willi syndrome, mirroring positive outcomes seen in children.
  • There is an uneven global application of GH treatment for adult PWS patients.
  • Genetically confirmed PWS diagnosis should be the sole criterion for approving GH treatment in adults.

Outlook:

  • Advocating for international approval of GH treatment for all individuals with genetically confirmed PWS, including adults.
  • Expanding access to GH therapy for adults with PWS to improve metabolic health and overall well-being.
  • Promoting standardized guidelines for GH treatment in PWS across pediatric and adult populations globally.

Keywords:
AdultsGrowth hormoneGrowth hormone treatmentPrader-willi syndrome

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