Myotonic Dystrophy patient preferences in patient-reported outcome measures


In preparation for clinical trials in myotonic dystrophy type-1 (DM1), it is important to develop and identify patient-reported outcome measures with good measurement properties. This paper describes 52 DM1 patients enrolled in two clinical studies who completed the Myotonic Dystrophy Health Index (MDHI), SF-36v2, Individualized Neuromuscular Quality of Life questionnaire (NQoL), 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. The authors conclude that the MDHI correlates well with physical function and is viewed favorably by participants in DM1 clinical studies.

Heatwole C, Johnson N, Dekdebrun J, et al. Myotonic dystrophy patient preferences in patient-reported outcome measures. Muscle Nerve. 2018 Jan 12. doi: 10.1002/mus.26066. [Epub ahead of print]