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Health-Related Quality of Life in Patients with Adult-Onset Myotonic Dystrophy Type 1: A Systematic Review
BACKGROUND: Adult-onset myotonic dystrophy type 1 (DM1) is a chronic, multisystem disorder that leads to disability and premature death. OBJECTIVES: The objective of our study was to conduct a systematic literature review of the health-related quality of life (HRQoL) of patients with DM1. METHODS: W...
Autores principales: | , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Springer International Publishing
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6598969/ https://www.ncbi.nlm.nih.gov/pubmed/30714084 http://dx.doi.org/10.1007/s40271-019-00357-y |
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author | Landfeldt, Erik Edström, Josefin Jimenez-Moreno, Cecilia van Engelen, Baziel G. M. Kirschner, Janbernd Lochmüller, Hanns |
author_facet | Landfeldt, Erik Edström, Josefin Jimenez-Moreno, Cecilia van Engelen, Baziel G. M. Kirschner, Janbernd Lochmüller, Hanns |
author_sort | Landfeldt, Erik |
collection | PubMed |
description | BACKGROUND: Adult-onset myotonic dystrophy type 1 (DM1) is a chronic, multisystem disorder that leads to disability and premature death. OBJECTIVES: The objective of our study was to conduct a systematic literature review of the health-related quality of life (HRQoL) of patients with DM1. METHODS: We searched Embase, Web of Science, and PubMed for English language full-text articles reporting results from studies of HRQoL in patients with adult-onset DM1 published between 1 January 2000 and 21 February 2018. We excluded reviews, editorials, and studies reporting results for a sample with fewer than five patients (to allow for meaningful inference). RESULTS: The search identified 266 unique publications. Of these, 231 were excluded following title and abstract screening and 16 after full-text review, leaving 19 articles for data synthesis. We found 15 articles measuring the HRQoL of patients with adult-onset DM1 using the 36-Item Short Form Health Survey (SF-36), six using the Individualized Neuromuscular Quality of Life Questionnaire (INQoL), and one using Cantril’s Ladder. Available evidence shows that patient HRQoL is impaired in DM1, mainly due to compromised physical health, but also reveals that substantial heterogeneity exists in estimates across studies. CONCLUSIONS: HRQoL in adult-onset DM1 has been extensively studied using the SF-36 and the INQoL, but current estimates are inconclusive, and little is known of the impact of the disease as measured using other instruments. Our data synthesis should help characterize the patient burden of DM1 and inform future studies of HRQoL in this indication. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (10.1007/s40271-019-00357-y) contains supplementary material, which is available to authorized users. |
format | Online Article Text |
id | pubmed-6598969 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2019 |
publisher | Springer International Publishing |
record_format | MEDLINE/PubMed |
spelling | pubmed-65989692019-07-19 Health-Related Quality of Life in Patients with Adult-Onset Myotonic Dystrophy Type 1: A Systematic Review Landfeldt, Erik Edström, Josefin Jimenez-Moreno, Cecilia van Engelen, Baziel G. M. Kirschner, Janbernd Lochmüller, Hanns Patient Systematic Review BACKGROUND: Adult-onset myotonic dystrophy type 1 (DM1) is a chronic, multisystem disorder that leads to disability and premature death. OBJECTIVES: The objective of our study was to conduct a systematic literature review of the health-related quality of life (HRQoL) of patients with DM1. METHODS: We searched Embase, Web of Science, and PubMed for English language full-text articles reporting results from studies of HRQoL in patients with adult-onset DM1 published between 1 January 2000 and 21 February 2018. We excluded reviews, editorials, and studies reporting results for a sample with fewer than five patients (to allow for meaningful inference). RESULTS: The search identified 266 unique publications. Of these, 231 were excluded following title and abstract screening and 16 after full-text review, leaving 19 articles for data synthesis. We found 15 articles measuring the HRQoL of patients with adult-onset DM1 using the 36-Item Short Form Health Survey (SF-36), six using the Individualized Neuromuscular Quality of Life Questionnaire (INQoL), and one using Cantril’s Ladder. Available evidence shows that patient HRQoL is impaired in DM1, mainly due to compromised physical health, but also reveals that substantial heterogeneity exists in estimates across studies. CONCLUSIONS: HRQoL in adult-onset DM1 has been extensively studied using the SF-36 and the INQoL, but current estimates are inconclusive, and little is known of the impact of the disease as measured using other instruments. Our data synthesis should help characterize the patient burden of DM1 and inform future studies of HRQoL in this indication. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (10.1007/s40271-019-00357-y) contains supplementary material, which is available to authorized users. Springer International Publishing 2019-02-04 2019 /pmc/articles/PMC6598969/ /pubmed/30714084 http://dx.doi.org/10.1007/s40271-019-00357-y Text en © The Author(s) 2019 Open Access This article is distributed under the terms of the Creative Commons Attribution-NonCommercial 4.0 International License (http://creativecommons.org/licenses/by-nc/4.0/), which permits any noncommercial use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. |
spellingShingle | Systematic Review Landfeldt, Erik Edström, Josefin Jimenez-Moreno, Cecilia van Engelen, Baziel G. M. Kirschner, Janbernd Lochmüller, Hanns Health-Related Quality of Life in Patients with Adult-Onset Myotonic Dystrophy Type 1: A Systematic Review |
title | Health-Related Quality of Life in Patients with Adult-Onset Myotonic Dystrophy Type 1: A Systematic Review |
title_full | Health-Related Quality of Life in Patients with Adult-Onset Myotonic Dystrophy Type 1: A Systematic Review |
title_fullStr | Health-Related Quality of Life in Patients with Adult-Onset Myotonic Dystrophy Type 1: A Systematic Review |
title_full_unstemmed | Health-Related Quality of Life in Patients with Adult-Onset Myotonic Dystrophy Type 1: A Systematic Review |
title_short | Health-Related Quality of Life in Patients with Adult-Onset Myotonic Dystrophy Type 1: A Systematic Review |
title_sort | health-related quality of life in patients with adult-onset myotonic dystrophy type 1: a systematic review |
topic | Systematic Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6598969/ https://www.ncbi.nlm.nih.gov/pubmed/30714084 http://dx.doi.org/10.1007/s40271-019-00357-y |
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