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Growth Hormone Treatment in Children With Prader-Willi Syndrome: Three Years of Longitudinal Data in Prepubertal

Nienke E Bakker1,2, Anders Lindberg3, Joseph Heissler4

  • 1Dutch Growth Research Foundation, 3016 AH Rotterdam, The Netherlands.

The Journal of Clinical Endocrinology and Metabolism
|March 22, 2017
PubMed

Insights

Growth hormone (GH) treatment significantly improves height in children with Prader-Willi syndrome (PWS). While BMI increases, it remains below concerning levels, though safety monitoring is crucial for all PWS patients.

Area of Science:

  • Pediatrics
  • Endocrinology
  • Genetics

Background:

  • Prader-Willi syndrome (PWS) is a complex genetic disorder often associated with growth failure and obesity.
  • Growth hormone (GH) deficiency is common in children with PWS, impacting linear growth.
  • Previous studies suggest potential benefits of GH therapy, but large-scale data are needed.

Purpose of the Study:

  • To evaluate the efficacy and safety of GH treatment in a large cohort of children with PWS.
  • To assess the impact of GH on linear growth and body mass index (BMI) in prepubertal and adolescent PWS patients.
  • To analyze long-term outcomes, including adult height and safety events.

Main Methods:

  • A worldwide retrospective cohort study utilizing longitudinal data from the Pfizer International Growth Database (KIGS) from 1987 to 2012.
  • Inclusion of 522 prepubertal children treated with GH for three years and 173 children who reached adult height.
  • Safety analysis encompassed 2332 children, monitoring serious adverse events and deaths.

Main Results:

  • GH treatment significantly improved height standard deviation score (SDS) in prepubertal children (to -0.31) and adolescents before puberty (to -0.22).
  • Adult height SDS averaged -1.19, with a total height gain of 0.95 SDS.
  • Body mass index SDS increased in prepubertal children (to 1.53) but remained below +2 SDS, and 12 deaths were reported in the safety cohort.

Conclusions:

  • GH treatment is effective in improving linear growth in children with PWS.
  • GH therapy helps mitigate the typical increase in obesity seen in PWS, maintaining BMI SDS below +2.
  • Close safety monitoring is essential for all children with PWS, irrespective of GH treatment status.
Abstract

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