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Patient. 2019 Feb 4. doi: 10.1007/s40271-019-00357-y. [Epub ahead of print]

Health-Related Quality of Life in Patients with Adult-Onset Myotonic Dystrophy Type 1: A Systematic Review.

Author information

1
Institute of Environmental Medicine, Karolinska Institutet, Nobels väg 13, 17177, Stockholm, Sweden. erik.landfeldt@ki.se.
2
ICON plc, Stockholm, Sweden. erik.landfeldt@ki.se.
3
Saco, Stockholm, Sweden.
4
Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, England.
5
Department of Neurology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, Nijmegen, The Netherlands.
6
Department of Neuropediatrics and Muscle Disorders, Medical Centre, University of Freiburg, Faculty of Medicine, Freiburg, Germany.
7
Children's Hospital of Eastern Ontario Research Institute, University of Ottawa, Ottawa, Canada.
8
Division of Neurology, Department of Medicine, The Ottawa Hospital, Ottawa, ON, Canada.

Abstract

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.

PMID:
30714084
DOI:
10.1007/s40271-019-00357-y

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