Journal
JOURNAL OF CLINICAL MEDICINE
Volume 11, Issue 9, Pages -Publisher
MDPI
DOI: 10.3390/jcm11092496
Keywords
Prader-Willi syndrome; children; body composition; cognition; growth hormone
Categories
Funding
- Pfizer
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This study compared the long-term effects of growth hormone (GH) treatment in young children with Prader-Willi syndrome (PWS) to untreated controls and assessed whether starting GH in the first year of life is optimal and safe. The results showed that GH-treated children had better body composition and growth compared to untreated controls. Starting GH treatment in the first year of life was found to be both optimal and safe.
Long-term effects of growth hormone (GH) treatment in young children with Prader-Willi syndrome (PWS) have never been compared with untreated age-matched controls with PWS, and it is unclear if starting GH in the first year of life is safe and more effective than starting GH in early childhood. We investigated the effects of long-term GH on body composition, anthropometrics and cognition in young children with PWS compared to untreated controls and assessed whether starting GH in the first year of life is optimal and safe. An open-label, prospective study was performed, comparing GH-treated children with untreated controls, and comparing children who started GH in the first year of life (subgroup A) with children who started between 2-5 years (subgroup C). A total of 82 GH-treated children with PWS and 22 age-matched controls with PWS were included. The main outcome measures were body composition, anthropometrics, IQ, and safety parameters. After 8 years, GH-treated children had significantly better body composition and were taller than age-matched controls. Subgroup A had a lower FM% trajectory during treatment than subgroup C and showed a greater and longer-term increase in the LBM index. After 8 years, subgroup A had a lower trunk/peripheral fat ratio (p = 0.043) and higher IQ (p = 0.043). No adverse effects of starting GH in the first year were found. Children with PWS who received long-term GH had a better body composition and growth than untreated age-matched controls and starting GH in the first year of life was optimal and safe.
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