Safety and effectiveness of growth hormone therapy in infants with Prader-Willi syndrome younger than 2 years: a prospective study

J Pediatr Endocrinol Metab. 2019 Aug 27;32(8):879-884. doi: 10.1515/jpem-2018-0539.


Background There is little evidence of the effects of early treatment with growth hormone (GH) in infants with Prader-Willi syndrome (PWS). A prospective study was conducted to assess the safety of GH therapy in infants younger than 2 years of age with PWS. Methods A total of 14 patients with PWS started treatment with GH under the age of 2 years and were followed over a 2-year period. A deletion of chromosome 15 was present in nine infants (64.3%) and maternal uniparental disomy 15 in five infants (35.7%). The median age at start of GH treatment was 9.6 months (interquartile range [IQR] 9.0-18.3 months). Changes in height standard deviation score (SDS), body mass index (BMI) SDS and subcapsular and tricipital skinfolds in the follow-up period were evaluated with a mixed-model regression analysis using the Package R. Results There were no fatal adverse events. A significant decrease (p < 0.001) in tricipital and subcapsular skinfold thickness, with an upward trend of height SDS and a downward trend of BMI SDS, was observed. Infants who started GH before 15 months of age started walking at a median of 18.0 [17.0-19.5] months vs. 36.6 [36.3-37.8] months for those who began treatment with GH after 15 months of age (p = 0.024). Conclusions GH treatment in infants with PWS less than 2 years of age is safe and improved body composition. Infants who received GH before the age of 15 months started to walk earlier.

Keywords: Prader-Willi syndrome; body composition; growth hormone.

Publication types

  • Clinical Trial

MeSH terms

  • Body Composition*
  • Body Height*
  • Body Mass Index*
  • Child Development / drug effects
  • Cognition / drug effects
  • Female
  • Follow-Up Studies
  • Human Growth Hormone / administration & dosage*
  • Humans
  • Infant
  • Male
  • Motor Activity / drug effects
  • Prader-Willi Syndrome / drug therapy*
  • Prognosis
  • Prospective Studies
  • Safety


  • Human Growth Hormone