Division of Gastroenterology & Hepatology, Departments of Medicine and Community Health Sciences, Cumming School of Medicine, University of Calgary, Calgary, Alberta, Canada; Alimentiv Inc, London, Ontario, Canada.
Department of Gastroenterology & Hepatology, Academic Medical Center, Amsterdam, The Netherlands.
Gastrointest Endosc. 2022 Jun;95(6):1126-1137.e2. doi: 10.1016/j.gie.2022.01.014. Epub 2022 Feb 1.
Endoscopic outcomes have become important measures of eosinophilic esophagitis (EoE) disease activity, including as an endpoint in randomized controlled trials (RCTs). We evaluated the operating properties of endoscopic measures for use in EoE RCTs.
Modified Research and Development/University of California Los Angeles appropriateness methods and a panel of 15 international EoE experts identified endoscopic items and definitions with face validity that were used in a 2-round voting process to define simplified (all items graded as absent or present) and expanded versions (additional grades for edema, furrows, and/or exudates) of the EoE Endoscopic Reference Score (EREFS). Inter- and intrarater reliability of these instruments (expressed as intraclass correlation coefficients [ICC]) were evaluated using paired endoscopy video assessments of 2 blinded central readers in patients before and after 8 weeks of proton pump inhibitors, swallowed topical corticosteroids, or dietary elimination. Responsiveness was measured using the standardized effect size (SES).
The appropriateness of 41 statements relevant to EoE endoscopic activity (endoscopic items, item definitions and grading, and other considerations relevant for endoscopy) was considered. The original and expanded EREFS demonstrated moderate-to-substantial inter-rater reliability (ICCs of .472-.736 and .469-.763, respectively) and moderate-to-almost perfect intrarater reliability (ICCs of .580-.828 and .581-.828, respectively). Strictures were least reliably assessed (ICC, .072-.385). The original EREFS was highly responsive (SES, 1.126 [95% confidence interval {CI}, .757-1.534]), although both expanded versions of EREFS, scored based on worst affected area, were numerically most responsive to treatment (expanded furrows: SES, 1.229 [95% CI, .858-1.643]; all items expanded: SES, 1.252 [95% CI, .880-1.667]). The EREFS and its modifications were not more reliably scored by segment and also not more responsive when proximal and distal EREFSs were summed.
EREFS and its modifications were reliable and responsive, and the original or expanded versions of the EREFS may be preferred in RCTs. Disease activity scored based on the worst affected area optimizes reliability and responsiveness.
内镜结果已成为嗜酸性食管炎（EoE）疾病活动的重要衡量标准，包括作为随机对照试验（RCT）的终点。我们评估了内镜测量方法在 EoE RCT 中的应用性能。
改良的研究与开发/加利福尼亚大学洛杉矶分校适宜性方法和一个由 15 名国际 EoE 专家组成的小组，使用两轮投票过程对面部有效性进行了评估，确定了简化版（所有项目均评为存在或不存在）和扩展版（水肿、皱襞和/或渗出物的其他等级）的 EoE 内镜参考评分（EREFS）。使用 2 位盲法中央读者对质子泵抑制剂、口服局部皮质类固醇或饮食消除治疗 8 周前后的患者进行内镜视频配对评估，评估这些工具的内部和内部观察者可靠性（表示为组内相关系数[ICC]）。使用标准化效应量（SES）测量反应性。
考虑了 41 项与 EoE 内镜活动相关的适宜性陈述（内镜项目、项目定义和分级以及与内镜相关的其他考虑因素）。原始和扩展的 EREFS 显示出中度至高度的观察者间可靠性（ICC 分别为.472-.736 和.469-.763）和中度至近乎完美的观察者内可靠性（ICC 分别为.580-.828 和.581-.828）。狭窄的评估最不可靠（ICC，.072-.385）。原始的 EREFS 反应性很高（SES，1.126 [95%置信区间{CI}，.757-1.534]），尽管基于最受影响区域评分的两种扩展版本的 EREFS 在治疗方面反应性更高（扩展皱襞：SES，1.229 [95%CI，.858-1.643]；所有项目扩展：SES，1.252 [95%CI，.880-1.667]）。EREFS 及其修改版在分段时评分不可靠，当近端和远端 EREFS 相加时，反应性也不可靠。
EREFS 及其修改版可靠且反应灵敏，原始或扩展版本的 EREFS 可能更适合 RCT。基于最受影响区域评分的疾病活动可优化可靠性和反应性。
