《简要食管吞咽困难问卷》和Eckardt评分的临床意义阈值定义：一项多中心心理测量学研究

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中文摘要

背景： 《简要食管吞咽困难问卷》（BEDQ）和Eckardt评分（ES）是吞咽困难最常用的患者报告结局（PROs）。然而，其以患者为中心的截断值、最小重要差异（MID）和重测信度尚未确立。我们旨在填补这些空白，并评估BEDQ的治疗反应性。
方法： 这项前瞻性多中心研究在西班牙和拉丁美洲的三级中心进行，纳入了因高分辨率测压而被转诊的成人。衍生出三个队列：截断值队列（随后分为衍生队列和验证队列）、重测队列（连续子样本重新填写PROs）和治疗反应性队列（接受贲门失弛缓症治疗的患者）。使用5点Likert患者总体印象严重程度（PGIS）量表评估自我感知的严重程度，并应用PGIS < 2（无/轻度症状（NMSS））和≥ 2。
结果： 截断值队列包括571名患者，分为衍生队列（397）和验证队列（174）。BEDQ截断值< 9和ES < 4最能预测NMSS。验证队列和自举法证实了这些截断值的稳定性。BEDQ的MID为5分，ES为1分，在各队列中一致。BEDQ显示出极好的重测信度（组内相关系数= 0.97），显著高于ES（0.85）。两种PROs在治疗后均显示出统计学显著降低（两者p < 0.001），BEDQ和ES的标准化反应均值分别为2.14和1.98。
结论： BEDQ < 9和ES < 4是识别NMSS患者的临床意义阈值。BEDQ变化超过5分和ES变化超过1分代表临床意义差异。与ES相比，BEDQ表现出更优越的心理测量学特性。

英文摘要

BACKGROUND: The Brief Esophageal Dysphagia Questionnaire (BEDQ) and Eckardt score (ES) are the most used patient-reported outcomes (PROs) for dysphagia. However, their patient-centered cutoffs, minimal important difference (MID), and test-retest reliability have not been established. We aimed to address these gaps and evaluate BEDQ treatment responsiveness.
METHODS: This prospective multicenter study was conducted in tertiary centers in Spain and Latin America and enrolled adults referred for high-resolution manometry. Three cohorts were derived: the cutoff cohort (subsequently subdivided into a derivation and a validation cohort), the test-retest cohort (consecutive subsample re-administered PROs), and the treatment responsiveness cohort (patients treated for achalasia). Self-perceived severity was evaluated using a 5-point Likert Patient Global Impression of Severity (PGIS) scale and applied as PGIS < 2 (No/Mild symptoms (NMSS)) and ≥ 2.
RESULTS: The cutoff cohort included 571 patients, subdivided into derivation (397) and validation (174) cohorts. A BEDQ cutoff of < 9 and ES < 4 optimally predicted NMSS. The validation cohort and bootstrapping confirmed the stability of these cutoffs. MID was 5 points for BEDQ and 1 point for ES, consistent across cohorts. BEDQ showed excellent test-retest reliability (intraclass correlation coefficient = 0.97), significantly higher than ES (0.85). Both PROs showed statistically significant reductions following treatment (p < 0.001 for both), with standardized response means of 2.14 and 1.98 for BEDQ and ES, respectively.
CONCLUSIONS: BEDQ < 9 and ES < 4 are clinically meaningful thresholds for identifying patients with NMSS. Changes exceeding 5 points for BEDQ and 1 point for ES represent clinically meaningful differences. BEDQ demonstrates superior psychometric properties compared to ES.