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

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Abstract

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. 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. 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). 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.
Original languageEnglish
Pages (from-to)879-884
Number of pages6
JournalJournal of Pediatric Endocrinology and Metabolism
Volume32
Issue number8
DOIs
Publication statusPublished - 27 Aug 2019

Keywords

  • body composition
  • growth hormone
  • Prader-Willi syndrome
  • BODY-COMPOSITION
  • CHILDREN
  • SCOLIOSIS
  • TODDLERS

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