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Twenty Years of GH Treatment in Adults with Prader-Willi Syndrome
Prader-Willi syndrome (PWS) is a rare neurodevelopmental genetic disorder. In adults, the syndrome is characterised by muscular hypotonia, a different body composition with more body fat than muscle mass, hyperphagia, behavioural problems, and cognitive dysfunction. Endocrine deficiencies are common...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8234514/ https://www.ncbi.nlm.nih.gov/pubmed/34204309 http://dx.doi.org/10.3390/jcm10122667 |
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author | Sjöström, Anna Höybye, Charlotte |
author_facet | Sjöström, Anna Höybye, Charlotte |
author_sort | Sjöström, Anna |
collection | PubMed |
description | Prader-Willi syndrome (PWS) is a rare neurodevelopmental genetic disorder. In adults, the syndrome is characterised by muscular hypotonia, a different body composition with more body fat than muscle mass, hyperphagia, behavioural problems, and cognitive dysfunction. Endocrine deficiencies are common, including growth hormone (GH) deficiency. Here, we present data from a cross-sectional study in adults with PWS with a focus on the long-term safety of GH treatment. A total of 22 patients (14 men) were treated with GH for a median of 20 years. Data on body composition, hormones, and metabolic parameters were retrieved from the patients’ medical records. The median age was 27 years. The median GH dose was 0.5 mg/day. Insulin-like growth factor 1 (IGF-I) and blood lipids were normal, while fasting glucose and HbA1c were slightly elevated in three men with diabetes. Fat mass was less than fat free mass in all, though this was less pronounced in women. GH treatment did not negatively affect the metabolic profile, and none developed cardiovascular diseases or cancer. All adults on long-term GH treatment had a normal body composition and our results indicate that treatment was safe. However, PWS is a complex, multisystemic disease and continuous, individual considerations are required during GH treatment, especially in patients with risk factors for adverse effects. |
format | Online Article Text |
id | pubmed-8234514 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-82345142021-06-27 Twenty Years of GH Treatment in Adults with Prader-Willi Syndrome Sjöström, Anna Höybye, Charlotte J Clin Med Article Prader-Willi syndrome (PWS) is a rare neurodevelopmental genetic disorder. In adults, the syndrome is characterised by muscular hypotonia, a different body composition with more body fat than muscle mass, hyperphagia, behavioural problems, and cognitive dysfunction. Endocrine deficiencies are common, including growth hormone (GH) deficiency. Here, we present data from a cross-sectional study in adults with PWS with a focus on the long-term safety of GH treatment. A total of 22 patients (14 men) were treated with GH for a median of 20 years. Data on body composition, hormones, and metabolic parameters were retrieved from the patients’ medical records. The median age was 27 years. The median GH dose was 0.5 mg/day. Insulin-like growth factor 1 (IGF-I) and blood lipids were normal, while fasting glucose and HbA1c were slightly elevated in three men with diabetes. Fat mass was less than fat free mass in all, though this was less pronounced in women. GH treatment did not negatively affect the metabolic profile, and none developed cardiovascular diseases or cancer. All adults on long-term GH treatment had a normal body composition and our results indicate that treatment was safe. However, PWS is a complex, multisystemic disease and continuous, individual considerations are required during GH treatment, especially in patients with risk factors for adverse effects. MDPI 2021-06-17 /pmc/articles/PMC8234514/ /pubmed/34204309 http://dx.doi.org/10.3390/jcm10122667 Text en © 2021 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 Sjöström, Anna Höybye, Charlotte Twenty Years of GH Treatment in Adults with Prader-Willi Syndrome |
title | Twenty Years of GH Treatment in Adults with Prader-Willi Syndrome |
title_full | Twenty Years of GH Treatment in Adults with Prader-Willi Syndrome |
title_fullStr | Twenty Years of GH Treatment in Adults with Prader-Willi Syndrome |
title_full_unstemmed | Twenty Years of GH Treatment in Adults with Prader-Willi Syndrome |
title_short | Twenty Years of GH Treatment in Adults with Prader-Willi Syndrome |
title_sort | twenty years of gh treatment in adults with prader-willi syndrome |
topic | Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8234514/ https://www.ncbi.nlm.nih.gov/pubmed/34204309 http://dx.doi.org/10.3390/jcm10122667 |
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