Long-Term Growth Hormone Treatment of Children with PWS: The Earlier the Start, the Better the Outcomes?

Abstract

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.

The Effects of 5 Years of Growth Hormone Treatment on Growth and Body Composition in Patients with Temple Syndrome

“…BMI SDS was within the normal range at start and did not change significantly during the treatment. Taking into account that FM% was >2 SDS at start of treatment, this further establishes the importance of more extensive body composition analysis in patients with TS14 because BMI is an unreliable measure of adiposity as previously evidenced in patients with PWS [23].…”

New developments and therapies in pediatric endocrinology

“…Treatment for PWS has seen progression too. The advantage of early GH treatment, starting before the age of 1 year, has become more clear [44]. Early diagnosis therefore is paramount and the feasibility of screening is being assessed [45].…”

Atypical 15q11.2-q13 Deletions and the Prader-Willi Phenotype

“…As most of our patients were treated with GH, this may have influenced the presence and severity of PWS symptoms. In particular, patients who started GH at a very young age might have a milder phenotype [ 47 ]. Patients with an atypical deletion that may be missed by a conventional PWS methylation test could have a delayed diagnosis.…”