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The requirement for a disease-specific patient-reported outcome measure of dysphagia in oculopharyngeal muscular dystrophy |
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| Authors: | Claudia Côté RD PHD Cynthia Gagnon OT PhD Sarah Youssof MD MS Nicolette sKurtz BA Bernard Brais MD PhD |
| Affiliation: | 1. Centre de recherche Charles-Le-Moyne-Saguenay-Lac-St-Jean sur les innovations en santé (CR-CSIS), Faculté de médecine et des sciences de la santé, Université de Sherbrooke, Québec, Canada;2. Centre de recherche Charles-Le-Moyne-Saguenay-Lac-St-Jean sur les innovations en santé (CR-CSIS), Faculté de médecine et des sciences de la santé, Université de Sherbrooke, Québec, Canada Groupe de recherche interdisciplinaire sur les maladies neuromusculaires (GRIMN), CIUSSS du Saguenay-Lac-St-Jean, Québec, Canada;3. Department of Neurology, University of New Mexico, Albuquerque, New Mexico;4. Department of Neurology and Neurosurgery, McGill University, Québec, Canada |
| 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 |