眼咽型肌营养不良症吞咽困难非仪器化临床结局评估的验证：一项结构化的、COSMIN指导的评估

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中文摘要

背景： 支持在眼咽型肌营养不良症（OPMD）患者中使用非仪器化吞咽困难结局测量的证据仍然有限。
方法： 这项COSMIN指导的验证研究评估了OPMD成人患者的患者报告和基于表现的吞咽困难测量工具。参与者完成了Dysphagiameter-OPMD、悉尼吞咽问卷（SSQ）和进食评估工具-10（EAT-10），以及基于表现的吞咽评估，包括作为对照的80 mL饮水试验、80 mL增稠果汁试验和10.5 g奶酪试验。评估了结构效度、内部一致性、评定者内和评定者间信度以及测量误差。
结果： 所有PROM和PerfOM的结构效度均得到支持，≥75%的预设假设得到证实。信度总体为良好至优秀，大多数PROM的ICC≥0.80，所有perfOM的ICC>0.90，尽管饮水试验的估计值受到极端观察值和较长完成时间代表性有限的影响。增稠果汁试验显示出小的系统性评定者内偏倚，并且与水试验相比提供的附加价值有限，而奶酪试验的一致性估计对极端观察值敏感。
结论： 总体而言，研究结果提供了支持OPMD特异性PROM和部分基于表现的测量工具效度和信度的证据，同时强调了它们在吞咽结局评估中的互补作用。

英文摘要

Evidence supporting the use of non-instrumental dysphagia outcome measures in people living with oculopharyngeal muscular dystrophy (OPMD) remains limited. This COSMIN-guided validation study evaluated patient-reported and performance-based dysphagia measures in adults with OPMD. Participants completed the Dysphagiameter-OPMD, the Sydney Swallow Questionnaire (SSQ), and the Eating Assessment Tool-10 (EAT-10), along with performance-based swallowing assessments including the 80-mL water drinking test, used as the comparator, 80-mL thickened juice test, and 10.5-g cheese test. Construct validity, internal consistency, intra- and inter-rater reliability, and measurement error were evaluated. Construct validity was supported for all PROMs and PerfOMs, with ≥ 75% of predefined hypotheses confirmed. Reliability was generally good to excellent, with ICC ≥ 0.80 for most PROMs and > 0.90 for all perfOMs, although estimates for the drinking tests were influenced by extreme observations and the limited representation of longer completion times. The thickened juice test showed a small systematic intra-rater bias and provided limited added value over the water test, while agreement estimates for the cheese test were sensitive to extreme observations. Overall, findings provide evidence supporting the validity and the reliability of OPMD-specific PROMs and selected performance-based measures, while highlighting their complementary roles in swallowing outcome assessment.