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.
Muscle Nerve. 2019 Apr;59(4):445-450. doi: 10.1002/mus.26405. Epub 2019 Jan 11.
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.
目前尚无专门用于评估眼咽型肌营养不良（OPMD）患者口咽吞咽困难的患者报告结局（PRO）问卷。为了选择合适的问卷，必须评估现有问卷的内容效度。本研究旨在：（1）确定 OPMD 相关的吞咽困难症状；（2）评估目前用于评估 OPMD 吞咽困难严重程度的 PRO 的内容效度。
对吞咽困难相关症状识别和口咽吞咽困难相关 PRO 进行了两步文献回顾。通过德尔菲调查，使用专家小组对症状进行了验证。通过内容分析记录了 PRO 问卷的内容效度。
共确定了 10 个 PRO 问卷。没有一个问卷涵盖了 OPMD 的整个症状谱，因此缺乏内容效度。
需要开发和验证一种新的 PRO 问卷来评估 OPMD 的吞咽困难，以确定新治疗方法对症状缓解的重要性。肌肉神经 59:445-450, 2019.
