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Current status of growth hormone therapy in Prader-Willi syndrome

Charlotte Höybye1

  • 1a Department of Endocrinology, Metabolism and Diabetology, Karolinska University Hospital, Stockholm, Sweden charlotte.hoybye@karolinska.se.

Expert Review of Endocrinology & Metabolism
|February 10, 2019
PubMed

Insights

Growth hormone (GH) treatment improves physical health and quality of life in Prader-Willi syndrome (PWS) patients. Careful monitoring is essential, but benefits often outweigh risks for this complex genetic disorder.

Area of Science:

  • Endocrinology
  • Genetics
  • Pediatrics

Background:

  • Prader-Willi syndrome (PWS) is a complex genetic disorder.
  • PWS is characterized by hypotonia, hypogonadism, short stature, hyperphagia, obesity, cognitive, and behavioral issues.
  • Abnormal body composition with excess fat and insufficient lean mass is typical, alongside impaired growth hormone (GH) secretion.

Purpose of the Study:

  • To evaluate the efficacy and safety of GH treatment in children and adults with PWS.
  • To highlight the benefits of GH therapy on growth, body composition, and overall well-being.

Main Methods:

  • Review of existing literature on GH treatment in PWS patients.
  • Analysis of outcomes including height, body composition, psychomotor functioning, physical activity, and quality of life.

Main Results:

  • GH treatment in children with PWS improves height, head size, body composition, and psychomotor functioning.
  • In adults with PWS, GH treatment enhances body composition, physical activity, and quality of life.
  • Restricted diet and exercise remain crucial alongside GH therapy.

Conclusions:

  • GH treatment is beneficial for individuals with genetically confirmed PWS, regardless of cognitive disabilities or scoliosis.
  • Careful monitoring of glucose metabolism and respiration is necessary due to potential adverse effects.
  • GH therapy should be continued when benefits exceed the risks.

Keywords:
PWSgrowth hormonegrowth hormone deficiencygrowth hormone treatmentobesity

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