Review of patient-reported outcome measures for use in myotonic dystrophy type 1 patients
Authors:
Symonds, T., Randall, J. A., and Campbell, P.
Abstract:
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.
METHODS: 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 (ADL) 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 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.
CONCLUSIONS: 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 ADL in a DM1 population. Muscle Nerve 56: 86-92, 2017.
Congenital malformations, deformations and chromosomal abnormalities and related symptoms Diseases of and symptoms related to the musculoskeletal system and connective tissue
