title: Review of patient-reported outcome measures for use in myotonic dystrophy type 1 patients.
abstract: 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.
  
  
  
  A structured literature review of PRO measures used in DM1 populations, comparing psychometric data from this population was undertaken.
  
  
  
  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-Activchas been published recently and builds on the DM1-Activ by adding more relevant items.
  
  
  
  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.
authors.affiliation: Clinical Outcomes Solutions, Folkestone, Kent, United Kingdom.
authors.forename: Tara
authors.initials: T
authors.lastname: Symonds
authors.affiliation: Clinical Outcomes Solutions, Folkestone, Kent, United Kingdom.
authors.forename: Jason A
authors.initials: JA
authors.lastname: Randall
authors.affiliation: Clinical Outcomes Solutions, Tucson, Arizona, USA.
authors.forename: Patrick
authors.initials: P
authors.lastname: Campbell
mesh_terms: D000203:Activities of Daily Living / Q000523:psychology; D005221:Fatigue / Q000175:diagnosis / Q000209:etiology; D006801:Humans; D009223:Myotonic Dystrophy / Q000150:complications / Q000175:diagnosis* / Q000523:psychology*; D000071066:Patient Reported Outcome Measures; D011594:Psychometrics; D011788:Quality of Life / Q000523:psychology*; D012893:Sleep Wake Disorders / Q000209:etiology; D011795:Surveys and Questionnaires
keywords: myotonic dystrophy type 1 (DM1); patient-reported outcome (PRO); psychometric; reliability; validity