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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...

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Autores principales: Sjöström, Anna, Höybye, Charlotte
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
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.
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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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