Review of patient-reported outcome measures for use in myotonic dystrophy type 1 patients

Muscle Nerve. 2017 Jul;56(1):86-92. doi: 10.1002/mus.25469. Epub 2017 Mar 1.

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-Activc 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.

Keywords: myotonic dystrophy type 1 (DM1); patient-reported outcome (PRO); psychometric; reliability; validity.

Publication types

  • Review
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Activities of Daily Living / psychology
  • Fatigue / diagnosis
  • Fatigue / etiology
  • Humans
  • Myotonic Dystrophy / complications
  • Myotonic Dystrophy / diagnosis*
  • Myotonic Dystrophy / psychology*
  • Patient Reported Outcome Measures*
  • Psychometrics
  • Quality of Life / psychology*
  • Sleep Wake Disorders / etiology
  • Surveys and Questionnaires