Myotonic dystrophy patient preferences in patient‐reported outcome measures. Issue 1 (7th February 2018)

• Record Type: Journal Article
• Authors: Heatwole, Chad; Johnson, Nicholas; Dekdebrun, Jeanne; Dilek, Nuran; Eichinger, Kate; Hilbert, James; Luebbe, Elizabeth; Martens, William; Mcdermott, Michael P.; Thornton, Charles; Moxley, Richard
• Is Part Of: Muscle & nerve. Volume 58:Issue 1(2018)
• Page Start: 49
• Page End: 55
• Publication Date: 2018-02-07
• Subjects: clinical trial -- Myotonic Dystrophy Health Index -- myotonic dystrophy type‐1 -- patient relevant -- patient‐reported outcome measure; Neuromuscular diseases -- Periodicals; Muscles -- Periodicals; Nerves -- Periodicals; 616.74
• DOI: 10.1002/mus.26066
• Languages: English
• ISSNs: 0148-639X

Abstract

ABSTRACT: Introduction : 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