Responsiveness of different disease activity indices in moderate-to-severe ulcerative colitis.

IF 12.8 Q1 MEDICINE, RESEARCH & EXPERIMENTAL Med Pub Date : 2024-09-26 DOI:10.1016/j.medj.2024.09.001

Virginia Solitano, Remo Panaccione, Bruce E Sands, Zhongya Wang, Malcolm Hogan, Guangyong Zou, Laurent Peyrin-Biroulet, Silvio Danese, Linda J Cornfield, Brian G Feagan, Siddharth Singh, Vipul Jairath, Christopher Ma

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Abstract

Background: 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.

Methods: 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.

Findings: 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]).

Conclusions: 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.

Funding: There is no funding for this study.

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中度至重度溃疡性结肠炎患者对不同疾病活动指数的反应。

背景：目前，临床、内镜、组织学和综合工具被用于测量溃疡性结肠炎（UC）患者的疾病活动性。我们比较了梅奥诊所评分（MCS）、改良MCS（mMS；不包括医生总体评估）、部分MCS（pMS；不含内镜子评分的MCS）、罗巴特组织病理学指数（RHI）和UC-100评分对中度至重度活动性UC患者接受乌司替库单抗治疗后病情变化的反应性：对 UNIFI 诱导试验 3 期（ClinicalTrials.gov：NCT02407236）进行了事后分析。中度至重度活动性 UC 患者被随机分配接受乌司替尼或安慰剂治疗。治疗分配是评估反应性的标准，反应性是用接受治疗的参与者比接受安慰剂的参与者得分变化更大的概率（称为获胜概率（WinP））来量化的，并使用非参数方法进行估算：结果：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。在梅奥评分的组成项目中，内镜子评分（WinP 0.76 [0.69-0.82]）和大便次数子评分（WinP 0.74 [0.69-0.79]）的反应性最强。炎症性肠病问卷（IBDQ）生活质量问卷也有反应（WinP 0.78 [0.72-0.82]）：结论：UC 疾病活动指数的反应性相似。结论：UC 疾病活动指数具有相似的反应性，根据治疗环境、评估时间点和测量可行性的不同，可采用不同的评分来证明反应性。这些结果支持使用mMS作为综合主要终点，同时纳入患者报告和内镜结果测量。UC-100评分可能更适用于常规纳入组织学评估的情况：本研究无资金支持。

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来源期刊

Med MEDICINE, RESEARCH & EXPERIMENTAL-

CiteScore

17.70

自引率

0.60%

发文量

102

期刊介绍： Med is a flagship medical journal published monthly by Cell Press, the global publisher of trusted and authoritative science journals including Cell, Cancer Cell, and Cell Reports Medicine. Our mission is to advance clinical research and practice by providing a communication forum for the publication of clinical trial results, innovative observations from longitudinal cohorts, and pioneering discoveries about disease mechanisms. The journal also encourages thought-leadership discussions among biomedical researchers, physicians, and other health scientists and stakeholders. Our goal is to improve health worldwide sustainably and ethically. Med publishes rigorously vetted original research and cutting-edge review and perspective articles on critical health issues globally and regionally. Our research section covers clinical case reports, first-in-human studies, large-scale clinical trials, population-based studies, as well as translational research work with the potential to change the course of medical research and improve clinical practice.