Solitano Virginia, Panaccione Remo, Sands Bruce E, Wang Zhongya, Hogan Malcolm, Zou Guangyong, Peyrin-Biroulet Laurent, Danese Silvio, Cornfield Linda J, Feagan Brian G, Singh Siddharth, Jairath Vipul, Ma Christopher
Department of Medicine, Division of Gastroenterology, Western University, London, ON, Canada; Alimentiv, Inc., London, ON, Canada; Division of Gastroenterology and Gastrointestinal Endoscopy, IRCCS Ospedale San Raffaele, Università Vita-Salute San Raffaele, Milan, Italy.
Division of Gastroenterology and Hepatology, Cumming School of Medicine, University of Calgary, Calgary, AB, Canada.
Med. 2025 Feb 14;6(2):100512. doi: 10.1016/j.medj.2024.09.001. Epub 2024 Oct 4.
Clinical, endoscopic, histological, and composite instruments are currently used to measure disease activity in patients with ulcerative colitis (UC). We compared the responsiveness of the Mayo Clinic score (MCS), modified MCS (mMS; excluding physician global assessment), partial MCS (pMS; MCS without endoscopic subscore), Robart's Histopathology Index (RHI), and UC-100 score to change after ustekinumab treatment in patients with moderately to severely active UC.
Post hoc analysis of the phase 3 UNIFI induction trial (ClinicalTrials.gov: NCT02407236) was conducted. Participants with moderately to severely active UC were randomized to receive ustekinumab or placebo. Treatment assignment was the criterion to assess responsiveness, which was quantified using the probability of a treated participant having a larger change in score than a placebo participant, termed the win probability (WinP), and estimated using nonparametric methods.
The UC-100 score demonstrated large responsiveness (WinP 0.72 [95% confidence interval: 0.66-0.78]), and the MCS (0.68 [0.62-0.73]), mMS (0.69 [0.63-0.75]), and pMS (0.65 [0.59-0.71]) demonstrated similar effect sizes. Of the component items of the Mayo score, the endoscopic subscore (WinP 0.76 [0.69-0.82]) and the stool frequency subscore (WinP 0.74 [0.69-0.79]) were the most responsive. The Inflammatory Bowel Disease Questionnaire (IBDQ) quality-of-life questionnaire was also responsive (WinP 0.78 [0.72-0.82]).
UC disease activity indices are similarly responsive. Depending on the treatment setting, time point of evaluation, and feasibility of measurement, different scores may be used to demonstrate response. These results support the use of mMS as a composite primary endpoint, incorporating both patient-reported and endoscopic outcome measures. The UC-100 score may be more appropriate in settings that also routinely incorporate histological evaluation.
There is no funding for this study.
目前，临床、内镜、组织学和综合工具用于测量溃疡性结肠炎（UC）患者的疾病活动度。我们比较了梅奥诊所评分（MCS）、改良MCS（mMS；不包括医生整体评估）、部分MCS（pMS；MCS不包括内镜亚评分）、罗巴特组织病理学指数（RHI）和UC - 100评分在中度至重度活动性UC患者接受乌司奴单抗治疗后的变化反应性。
对3期UNIFI诱导试验（ClinicalTrials.gov：NCT02407236）进行事后分析。中度至重度活动性UC患者被随机分配接受乌司奴单抗或安慰剂。治疗分配是评估反应性的标准，使用治疗参与者得分变化大于安慰剂参与者的概率（称为获胜概率[WinP]）进行量化，并采用非参数方法进行估计。
UC - 100评分显示出较大的反应性（WinP 0.72[95%置信区间：0.66 - 0.78]），MCS（0.68[0.62 - 0.73]）、mMS（0.69[0.63 - 0.75]）和pMS（0.65[0.59 - 0.71]）显示出相似的效应大小。在梅奥评分的各个组成项目中，内镜亚评分（WinP 0.76[0.69 - 0.82]）和大便频率亚评分（WinP 0.74[0.69 - 0.79]）反应性最强。炎症性肠病问卷（IBDQ）生活质量问卷也具有反应性（WinP 0.78[0.72 - 0.82]）。
UC疾病活动指数的反应性相似。根据治疗背景、评估时间点和测量的可行性，可使用不同的评分来证明反应情况。这些结果支持使用mMS作为综合主要终点，纳入患者报告和内镜结果测量。在常规纳入组织学评估的情况下，UC - 100评分可能更合适。
本研究无资金支持。
