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Time for a general approval of growth hormone treatment in adults with Prader–Willi syndrome
Prader-Willi syndrome (PWS) is a complex, multi-system, neurodevelopmental disorder characterised by neonatal muscular hypotonia, short stature, high risk of obesity, hypogonadism, intellectual disabilities, distinct behavioural/psychiatric problems and abnormal body composition with increased body...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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BioMed Central
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7869190/ https://www.ncbi.nlm.nih.gov/pubmed/33557878 http://dx.doi.org/10.1186/s13023-020-01651-x |
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author | Höybye, Charlotte Holland, Anthony J. Driscoll, Daniel J. |
author_facet | Höybye, Charlotte Holland, Anthony J. Driscoll, Daniel J. |
author_sort | Höybye, Charlotte |
collection | PubMed |
description | Prader-Willi syndrome (PWS) is a complex, multi-system, neurodevelopmental disorder characterised by neonatal muscular hypotonia, short stature, high risk of obesity, hypogonadism, intellectual disabilities, distinct behavioural/psychiatric problems and abnormal body composition with increased body fat and a deficit of lean body mass. Growth hormone (GH) deficiency and other hormone deficiencies are common due to hypothalamic dysfunction. In children with PWS GH treatment has been widely demonstrated to improve body composition, normalise height and improve psychomotor development. In adults with PWS, GH’s main effects are to maintain normal body structure and metabolism. The positive effects of GH treatment on body composition, physical fitness and beneficial effects on cardiovascular risk markers, behaviour and quality of life in adults with PWS are also well established from several studies. GH treatment is approved for treatment of children with PWS in many countries, but until recently not as a treatment in young adults in the transition period or for adults in general. In this commentary we want to draw attention to the uneven global use of GH treatment, specifically in adults with PWS, and advocate for GH treatment to be approved internationally, not just for children, but also for adults with PWS and based only on the diagnosis of genetically confirmed PWS. |
format | Online Article Text |
id | pubmed-7869190 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | BioMed Central |
record_format | MEDLINE/PubMed |
spelling | pubmed-78691902021-02-08 Time for a general approval of growth hormone treatment in adults with Prader–Willi syndrome Höybye, Charlotte Holland, Anthony J. Driscoll, Daniel J. Orphanet J Rare Dis Letter to the Editor Prader-Willi syndrome (PWS) is a complex, multi-system, neurodevelopmental disorder characterised by neonatal muscular hypotonia, short stature, high risk of obesity, hypogonadism, intellectual disabilities, distinct behavioural/psychiatric problems and abnormal body composition with increased body fat and a deficit of lean body mass. Growth hormone (GH) deficiency and other hormone deficiencies are common due to hypothalamic dysfunction. In children with PWS GH treatment has been widely demonstrated to improve body composition, normalise height and improve psychomotor development. In adults with PWS, GH’s main effects are to maintain normal body structure and metabolism. The positive effects of GH treatment on body composition, physical fitness and beneficial effects on cardiovascular risk markers, behaviour and quality of life in adults with PWS are also well established from several studies. GH treatment is approved for treatment of children with PWS in many countries, but until recently not as a treatment in young adults in the transition period or for adults in general. In this commentary we want to draw attention to the uneven global use of GH treatment, specifically in adults with PWS, and advocate for GH treatment to be approved internationally, not just for children, but also for adults with PWS and based only on the diagnosis of genetically confirmed PWS. BioMed Central 2021-02-08 /pmc/articles/PMC7869190/ /pubmed/33557878 http://dx.doi.org/10.1186/s13023-020-01651-x Text en © The Author(s) 2021 Open AccessThis article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated in a credit line to the data. |
spellingShingle | Letter to the Editor Höybye, Charlotte Holland, Anthony J. Driscoll, Daniel J. Time for a general approval of growth hormone treatment in adults with Prader–Willi syndrome |
title | Time for a general approval of growth hormone treatment in adults with Prader–Willi syndrome |
title_full | Time for a general approval of growth hormone treatment in adults with Prader–Willi syndrome |
title_fullStr | Time for a general approval of growth hormone treatment in adults with Prader–Willi syndrome |
title_full_unstemmed | Time for a general approval of growth hormone treatment in adults with Prader–Willi syndrome |
title_short | Time for a general approval of growth hormone treatment in adults with Prader–Willi syndrome |
title_sort | time for a general approval of growth hormone treatment in adults with prader–willi syndrome |
topic | Letter to the Editor |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7869190/ https://www.ncbi.nlm.nih.gov/pubmed/33557878 http://dx.doi.org/10.1186/s13023-020-01651-x |
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