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


Journal

Muscle & nerve
ISSN: 1097-4598
Titre abrégé: Muscle Nerve
Pays: United States
ID NLM: 7803146

Informations de publication

Date de publication:
04 2019
Historique:
received: 16 10 2017
revised: 11 12 2018
accepted: 15 12 2018
pubmed: 24 12 2018
medline: 6 7 2019
entrez: 22 12 2018
Statut: ppublish

Résumé

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

Identifiants

pubmed: 30575974
doi: 10.1002/mus.26405
doi:

Types de publication

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

Langues

eng

Sous-ensembles de citation

IM

Pagination

445-450

Subventions

Organisme : Bioblast Pharma, LTD
Pays : International
Organisme : Junior 2 FRQS Researcher award
ID : CC#256277
Pays : International
Organisme : Junior 2 FRQS Researcher award
ID : CG#231011
Pays : International
Organisme : Groupe de recherche interdisciplinaire sur les maladies Neuromusculaires-Bioblast
Pays : International

Informations de copyright

© 2018 Wiley Periodicals, Inc.

Auteurs

Claudia Côté (C)

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.

Cynthia Gagnon (C)

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.

Sarah Youssof (S)

Department of Neurology, University of New Mexico, Albuquerque, New Mexico.

Nicolette sKurtz (N)

Department of Neurology, University of New Mexico, Albuquerque, New Mexico.

Bernard Brais (B)

Department of Neurology and Neurosurgery, McGill University, Québec, Canada.

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Classifications MeSH