The requirement for a disease-specific patient-reported outcome measure of dysphagia in oculopharyngeal muscular dystrophy

Muscle Nerve. 2019 Apr;59(4):445-450. doi: 10.1002/mus.26405. Epub 2019 Jan 11.

Abstract

Introduction: There is no patient-reported outcome (PRO) questionnaire specifically designed to assess oropharyngeal dysphagia in oculopharyngeal muscular dystrophy (OPMD). To select a suitable questionnaire, content validity of the existing questionnaires must be assessed. This study sought (1) to identify dysphagia-related symptoms in OPMD and (2) to assess content validity of currently available PRO for the assessment of dysphagia severity in OPMD.

Methods: A two-step literature review was conducted of dysphagia-related symptom identification and oropharyngeal dysphagia-related PRO. Symptoms were validated with an expert panel by using a Delphi survey. Content validity of PRO questionnaires was documented through content analysis.

Results: Ten PRO questionnaires were identified. None of the questionnaires cover the entire symptom spectrum in OPMD and thus lack content validity.

Discussion: The development and validation of a new PRO questionnaire to assess dysphagia in OPMD is required to establish the importance of symptomatic relief from new treatments. Muscle Nerve 59:445-450, 2019.

Keywords: content validity; dysphagia symptoms; oculopharyngeal muscular dystrophy; oropharyngeal dysphagia; patient-reported outcome.

Publication types

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

MeSH terms

  • Aged
  • Deglutition Disorders / etiology*
  • Deglutition Disorders / physiopathology
  • Delphi Technique
  • Disability Evaluation
  • Female
  • Humans
  • Male
  • Middle Aged
  • Muscular Dystrophy, Oculopharyngeal / complications*
  • Muscular Dystrophy, Oculopharyngeal / physiopathology
  • Patient Reported Outcome Measures
  • Reproducibility of Results
  • Self Report*
  • Surveys and Questionnaires