Khanna Reena, Zou GuangYong, Stitt Larry, Feagan Brian G, Sandborn William J, Rutgeerts Paul, McDonald John W D, Dubcenco Elena, Fogel Ronald, Panaccione Remo, Jairath Vipul, Nelson Sigrid, Shackelton Lisa M, Huang Bidan, Zhou Qian, Robinson Anne M, Levesque Barrett G, D'Haens Geert
Robarts Clinical Trials, University of Western Ontario, London, Ontario, Canada.
Division of Gastroenterology, Department of Medicine, University of Western Ontario, London, Ontario, Canada.
Am J Gastroenterol. 2017 Oct;112(10):1584-1592. doi: 10.1038/ajg.2016.580. Epub 2017 Jan 10.
The Crohn's Disease Endoscopic Index of Severity (CDEIS) and the Simple Endoscopic Score for Crohn's Disease (SES-CD) are commonly used to assess Crohn's disease (CD) activity; however neither instrument is fully validated. We evaluated the responsiveness to change of the SES-CD and CDEIS using data from a trial of adalimumab, a drug therapy of known efficacy.
Paired video recordings (N=112) of colonoscopies (baseline and week 8-12) obtained from patients with CD who participated in a trial of adalimumab therapy were reviewed in random order, in duplicate, by four central readers (56 pairs of videos by 2 groups of readers). Responsiveness of the SES-CD and the CDEIS was evaluated by comparing correlations between the observed and pre-specified predictions of change scores for these endoscopic indices with a global endoscopic evaluation of severity (GELS), a patient reported outcome (PRO2), and the Crohn's disease activity index (CDAI), and by calculation of the standardized effect size, and Guyatt's Responsiveness statistic (GRS) using 2 definitions of change; (1) treatment assignment and (2) an absolute change in total PRO2 of 50. The potential application of effect size estimates was demonstrated by calculating hypothetical sample sizes for comparing two independent groups. The impact of removing stenosis as an index item and adjusting for the number of segments observed was also assessed.
Changes in both endoscopic instruments and the GELS were highly correlated. The SES-CD displayed numerically higher effect sizes for both definitions of change. The standardized effect size and GRS estimates (95% confidence interval) for the SES-CD based on treatment assignment were 0.84 (0.53, 1.15) and 0.79 (0.48, 1.09). Corresponding values for the CDEIS were 0.72 (0.42, 1.02) and 0.75 (0.45, 1.06). The standardized effect size and GRS estimates for the SES-CD based on an absolute change in total PRO2 of 50 points or greater were 0.76 (0.49, 1.02) and 0.93 (0.64, 1.21). Corresponding values for CDEIS were 0.70 (0.44, 0.97), 0.83 (0.55, 1.10). Removal of stenosis as an index item and adjusting for observed segments did not improve responsiveness estimates.
Although both the SES-CD and CDEIS are valid measures of endoscopic disease activity that are moderately responsive to changes in endoscopic disease activity, the SES-CD displayed numerically greater responsiveness in this data set.
克罗恩病内镜严重程度指数（CDEIS）和克罗恩病简易内镜评分（SES-CD）常用于评估克罗恩病（CD）活动度；然而，这两种工具均未得到充分验证。我们使用已知疗效的药物疗法阿达木单抗试验的数据，评估了SES-CD和CDEIS对变化的反应性。
从参与阿达木单抗治疗试验的CD患者中获取的结肠镜检查配对视频记录（N = 112）（基线和第8 - 12周），由四位中心阅片者以随机顺序进行重复审核（两组阅片者共56对视频）。通过比较这些内镜指数观察到的变化分数与预先指定的预测分数之间的相关性，以及与整体内镜严重程度评估（GELS）、患者报告结局（PRO2）和克罗恩病活动指数（CDAI）之间的相关性，来评估SES-CD和CDEIS的反应性，并通过计算标准化效应量和使用两种变化定义的盖亚特反应性统计量（GRS）进行评估；（1）治疗分配，（2）总PRO2绝对变化50。通过计算比较两个独立组的假设样本量，展示了效应量估计的潜在应用。还评估了去除狭窄作为一个指数项目以及对观察节段数量进行调整的影响。
两种内镜工具的变化与GELS均高度相关。对于两种变化定义，SES-CD的效应量在数值上更高。基于治疗分配的SES-CD的标准化效应量和GRS估计值（95%置信区间）分别为0.84（0.53，1.15）和0.79（0.48，1.09）。CDEIS的相应值分别为0.72（0.42，1.02）和0.75（0.45，1.06）。基于总PRO2绝对变化50分或更高的SES-CD的标准化效应量和GRS估计值分别为0.76（0.49，1.02）和0.93（0.64，1.21）。CDEIS的相应值分别为0.70（0.44，0.97）、0.83（0.55，1.10）。去除狭窄作为一个指数项目以及对观察节段进行调整并未改善反应性估计值。
虽然SES-CD和CDEIS都是内镜疾病活动度的有效测量指标，对内镜疾病活动度的变化有中度反应，但在该数据集中，SES-CD在数值上显示出更大的反应性。
