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

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

Cross-cultural adaptation of the SWAL-QOL and the Sydney Swallow Questionnaire (SSQ) into French-Canadian and preliminary assessment for their use in an oculopharyngeal muscular dystrophy (OPMD) population

“…No difference was observed in the DSB scores of the two groups of participants. One hypothesis is that the frequency of speci c swallowingrelated symptoms may not accurately re ect the severity of dysphagia in OPMD, because of the slowly progressive nature of the disease and the patient's ability to compensate over time for the swallowing problems with changes in diet or the use of other coping strategies [34,11]. Coping strategies, which are not addressed by the SWAL-QOL, are important aspects of the construct of dysphagia in OPMD [35].…”

“…In the eld of dysphagia, there are a number of PRO questionnaires differing by their evaluation purpose, psychometric properties and target populations [9,10]. However, these questionnaires are not speci cally designed to assess oropharyngeal dysphagia in OPMD and do not cover the entire symptom spectrum [11]. Two questionnaires, the Swallowing Quality of Life instrument (SWAL-QOL) and the Sydney Swallow Questionnaire (SSQ), were found to have at least some evidence of content validity for OPMD [11] and therefore were chosen to be pretested as two potential outcome measures for use in clinical trials.…”

“…Its usefulness appears complementary to the SWAL-QOL since it addresses swallowing symptoms that are not covered by the latter, such as di culty with different types of food and liquid (hard, soft and dry foods/thin and thick liquids). These symptoms were considered important for dysphagia assessment in patients with OPMD, following expert review [11]. The reliability and validity of the SSQ were documented in participants with oropharyngeal dysphagia of various etiologies, but not speci cally in people with OPMD.…”

Dysphagia in adult myopathies

“…A panel of experts reviewed the most frequent symptoms and assessed their effect on patients' life [44] . Of the list of 24 reported symptoms, swallowing difficulty mainly to solid food, a sense of throat blockade and coughing and choking during eating were considered to be the most problematic.…”

French-Canadian Cross-Cultural Adaptation of the SWAL-QOL and the SSQ and A Preliminary Psychometric Assessment for Their Use in an Oculopharyngeal Muscular Dystrophy (OPMD) Population

“…In the eld of dysphagia, there are a number of PRO questionnaires differing by their evaluation purpose, psychometric properties and target populations [9,10]. However, these questionnaires are not speci cally designed to assess oropharyngeal dysphagia in OPMD and do not cover the entire symptom spectrum [11]. Two questionnaires, the Swallowing Quality of Life instrument (SWAL-QOL) and the Sydney Swallow Questionnaire (SSQ), were found to have at least some evidence of content validity for OPMD [11] and therefore were chosen to be pretested as two potential outcome measures for use in clinical trials.…”