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Myotonic dystrophy patient preferences in patient‐reported outcome measures
Author(s) -
Heatwole Chad,
Johnson Nicholas,
Dekdebrun Jeanne,
Dilek Nuran,
Eichinger Kate,
Hilbert James,
Luebbe Elizabeth,
Martens William,
Mcdermott Michael P.,
Thornton Charles,
Moxley Richard
Publication year - 2018
Publication title -
muscle and nerve
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.025
H-Index - 145
eISSN - 1097-4598
pISSN - 0148-639X
DOI - 10.1002/mus.26066
Subject(s) - myotonic dystrophy , muscular dystrophy , medicine , physical therapy , quality of life (healthcare) , physical medicine and rehabilitation , patient reported outcome , clinical significance , psychology , gerontology , nursing
: When preparing for clinical trials in myotonic dystrophy type‐1 (DM1), it is important that researchers develop and identify patient‐reported outcome measures with good measurement properties. Methods : Fifty‐two DM1 patients enrolled in 2 clinical studies completed the Myotonic Dystrophy Health Index (MDHI), 36‐Item Short Form Health Survey (version 2; SF‐36v2), Individualized Neuromuscular Quality of Life questionnaire (INQoL), and a questionnaire comparing the relevance, usability, overall preference, and perceived responsiveness of each measure. The associations between instrument scores and physical function, genetic test results, and employment status were examined. Results : The MDHI was preferred over the INQoL in 13 of 13 areas and was preferred over the SF‐36v2 in 9 of 13 areas. The MDHI was the only score that was associated with participant employment status, CTG repeat length, and the 3 measurements of clinical function. Discussion : The MDHI correlates well with physical function and is viewed favorably by participants in DM1 clinical studies. Muscle Nerve 58 : 49–55, 2018