A review of patient reported outcome measures for use in DM1 patients

Introduction: The heterogeneity of symptoms experienced by Myotonic Dystrophy type 1 (DM1) patients means patient reported outcome (PRO) assessments are uniquely suited to address this through questionnaires. Method: A structured literature review of PRO measures used in DM1 populations, comparing psychometric data from this population was undertaken. Results: One health status measure, 3 activities of daily living scales, 3 health-related quality of life (HRQOL) assessments, and 5 sleep and fatigue measures have validity and reliability information from DM1 populations. The Myotonic Dystrophy Health Index (MDHI) and DM1 Activity and Participation scale (DM1-Activ) have the strongest validity and reliability evidence. The DM1-Activ c has been published recently and builds on the DM1-Activ by adding more relevant items. Discussion: The PRO instruments we identified have varying psychometric evidence in DM1 populations; all require further testing to be confident of their ability to make accurate and valid measurements of symptoms, HRQOL, and Activities of Daily Living (ADL) in a DM1 population.

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