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Myotonic dystrophy patient preferences in patient-reported outcome measures

Chad Heatwole¹, Nicholas Johnson², Jeanne Dekdebrun¹

¹Department of Neurology, University of Rochester Medical Center, Box 673, 601 Elmwood Avenue Rochester, New York, 14642, USA.

|January 13, 2018

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, 2018.

Keywords:

Myotonic Dystrophy Health Index clinical trial myotonic dystrophy type-1 patient relevant patient-reported outcome measure

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