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

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 i...

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Autores principales: Grootjen, Lionne N., Trueba-Timmermans, Demi J., Damen, Layla, Mahabier, Eva F., Kerkhof, Gerthe F., Hokken-Koelega, Anita C. S.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9105093/
https://www.ncbi.nlm.nih.gov/pubmed/35566622
http://dx.doi.org/10.3390/jcm11092496
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author Grootjen, Lionne N.
Trueba-Timmermans, Demi J.
Damen, Layla
Mahabier, Eva F.
Kerkhof, Gerthe F.
Hokken-Koelega, Anita C. S.
author_facet Grootjen, Lionne N.
Trueba-Timmermans, Demi J.
Damen, Layla
Mahabier, Eva F.
Kerkhof, Gerthe F.
Hokken-Koelega, Anita C. S.
author_sort Grootjen, Lionne N.
collection PubMed
description 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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spelling pubmed-91050932022-05-14 Long-Term Growth Hormone Treatment of Children with PWS: The Earlier the Start, the Better the Outcomes? Grootjen, Lionne N. Trueba-Timmermans, Demi J. Damen, Layla Mahabier, Eva F. Kerkhof, Gerthe F. Hokken-Koelega, Anita C. S. J Clin Med Article 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. MDPI 2022-04-29 /pmc/articles/PMC9105093/ /pubmed/35566622 http://dx.doi.org/10.3390/jcm11092496 Text en © 2022 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).
spellingShingle Article
Grootjen, Lionne N.
Trueba-Timmermans, Demi J.
Damen, Layla
Mahabier, Eva F.
Kerkhof, Gerthe F.
Hokken-Koelega, Anita C. S.
Long-Term Growth Hormone Treatment of Children with PWS: The Earlier the Start, the Better the Outcomes?
title Long-Term Growth Hormone Treatment of Children with PWS: The Earlier the Start, the Better the Outcomes?
title_full Long-Term Growth Hormone Treatment of Children with PWS: The Earlier the Start, the Better the Outcomes?
title_fullStr Long-Term Growth Hormone Treatment of Children with PWS: The Earlier the Start, the Better the Outcomes?
title_full_unstemmed Long-Term Growth Hormone Treatment of Children with PWS: The Earlier the Start, the Better the Outcomes?
title_short Long-Term Growth Hormone Treatment of Children with PWS: The Earlier the Start, the Better the Outcomes?
title_sort long-term growth hormone treatment of children with pws: the earlier the start, the better the outcomes?
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9105093/
https://www.ncbi.nlm.nih.gov/pubmed/35566622
http://dx.doi.org/10.3390/jcm11092496
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