Analysis of the functional capacity outcome measures for myotonic dystrophy
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- dc.contributor.author Jimenez-Moreno, Aura Cecilia
- dc.contributor.author Nikolenko, Nikoletta
- dc.contributor.author Kierkegaard, Marie
- dc.contributor.author Blain, Alasdair P.
- dc.contributor.author Newman, Jane
- dc.contributor.author Massey, Charlotte
- dc.contributor.author Moat, Dionne
- dc.contributor.author Sodhi, Jas
- dc.contributor.author Atalaia, Antonio
- dc.contributor.author Gorman, Grainne S.
- dc.contributor.author Turner, Chris
- dc.contributor.author Lochmüller, Hanns
- dc.date.accessioned 2019-10-23T08:06:53Z
- dc.date.available 2019-10-23T08:06:53Z
- dc.date.issued 2019
- dc.description.abstract Objectives: Defining clinically relevant outcome measures for myotonic dystrophy type 1 (DM1) that can be valid and feasible for different phenotypes has proven problematic. The Outcome Measures for Myotonic Dystrophy (OMMYD) group proposed a battery of functional outcomes: 6‐minute walk test, 30 seconds sit and stand test, timed 10 m walk test, timed 10 m walk/run test, and nine‐hole peg test. This, however, required a large‐scale investigation. Methods: A cohort of 213 patients enrolled in the natural history study, PhenoDM1, was analyzed in cross‐sectional analysis and subsequently 98 patients were followed for longitudinal analysis. We aimed to assess: (1) feasibility and best practice; (2) intra‐session reliability; (3) validity; and (4) behavior over time, of these tests. Results: OMMYD outcomes proved feasible as 96% of the participants completed at least one trial for all tests and more than half (n = 113) performed all three trials of each test. Body mass index and disease severity associate with functional capacity. There was a significant difference between the first and second trials of each test. There was a moderate to strong correlation between these functional outcomes and muscle strength, disease severity and patient‐reported outcomes. All outcomes after 1 year detected a change in functional capacity except the nine‐hole peg test. Conclusions: These tests can be used as a battery of outcomes or independently based on the shown overlapping psychometric features and strong cross‐correlations. Due to the large and heterogeneous sample of this study, these results can serve as reference values for future studies
- dc.format.mimetype application/pdf
- dc.identifier.citation Jimenez-Moreno AC, Nikolenko N, Kierkegaard M, Blain AP, Newman J, Massey C, Moat D, Sodhi J, Atalaia A, Gorman GS, Turner C, Lochmüller H. Analysis of the functional capacity outcome measures for myotonic dystrophy. Ann Clin Transl Neurol. 2019; 6(8):1487-1497. DOI 10.1002/acn3.50845
- dc.identifier.doi http://dx.doi.org/10.1002/acn3.50845
- dc.identifier.issn 2328-9503
- dc.identifier.uri http://hdl.handle.net/10230/42489
- dc.language.iso eng
- dc.publisher Wiley
- dc.relation.ispartof Ann Clin Transl Neurol. 2019; 6(8):1487-1497
- dc.rights © 2019 The Authors. Annals of Clinical and Translational Neurology published by Wiley Periodicals, Inc on behalf of American Neurological Association. This is an open access article under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0/), which permits use, distribution and reproduction in any medium, provided the original work is properly cited.
- dc.rights.accessRights info:eu-repo/semantics/openAccess
- dc.rights.uri http://creativecommons.org/licenses/by/4.0/
- dc.title Analysis of the functional capacity outcome measures for myotonic dystrophy
- dc.type info:eu-repo/semantics/article
- dc.type.version info:eu-repo/semantics/publishedVersion