Growth hormone treatment for Prader-Willi syndrome: A review

Neuropeptides. 2020 Oct:83:102084. doi: 10.1016/j.npep.2020.102084. Epub 2020 Aug 21.

Abstract

The Prader-Willi Syndrome (PWS) is a rare developmental disorder that contributed by multiple genes. Phenotypically, infants with PWS exhibit hypotonia and developmental delay, whilst older children and adults have cognitive impairments, neuropsychiatric symptoms, impaired motor development, neurological anomalies, endocrine dysfunctions like growth hormone (GH) deficiency, and hyperphagia that leads to obesity. Although mechanisms remain elusive, GH treatment has been recommended as the standard treatment for PWS children. In addition to better motor development, improved body composition and linear growth have been well established, but mental flexibility and behavioural problems remained largely untouched. This review will systemically analyze the recent clinical trials of GH treatment on PWS patients. The emphasis is on the mental and behavioural improvements by GH treatment, and a few concerns to initiate GH treatment. This review will finally propose possible future explorations on basic studies that may shed new light on clinical trials of GH treatment on PWS.

Keywords: Body composition; Clinical trials; Growth hormone; Mental flexibility; Prader-Willi syndrome.

Publication types

  • Review

MeSH terms

  • Body Composition / drug effects
  • Child
  • Cognition / drug effects
  • Human Growth Hormone / pharmacology
  • Human Growth Hormone / therapeutic use*
  • Humans
  • Prader-Willi Syndrome / drug therapy*

Substances

  • Human Growth Hormone