一项回顾性分析：用于评估克罗恩病活动度的患者报告结局指标的发展

A retrospective analysis: the development of patient reported outcome measures for the assessment of Crohn's disease activity.

作者信息

Khanna R, Zou G, D'Haens G, Feagan B G, Sandborn W J, Vandervoort M K, Rolleri R L, Bortey E, Paterson C, Forbes W P, Levesque B G

机构信息

Department of Medicine, University of Western Ontario, London, ON, Canada; Robarts Clinical Trials Inc., University of Western Ontario, London, ON, Canada.

出版信息

Aliment Pharmacol Ther. 2015 Jan;41(1):77-86. doi: 10.1111/apt.13001. Epub 2014 Oct 27.

DOI:10.1111/apt.13001

PMID:25348809

Abstract

BACKGROUND

The Crohn's Disease Activity Index (CDAI) is a measure of disease activity based on symptoms, signs and a laboratory test. The US Food and Drug Administration has indicated that patient reported outcomes (PROs) should be the primary outcome in randomised controlled trials for Crohn's disease (CD).

AIM

As no validated PRO exists for CD, to investigate whether CDAI diary card items could be modified for this purpose.

METHODS

Data from a trial of rifaximin-extended intestinal release were used to identify cut-points for stool frequency, pain and general well-being using receiver operating characteristic curves with CDAI <150 as criterion. The operating properties of 2- and 3-item PRO were evaluated using data from a trial of methotrexate in CD. Regression analysis determined PRO2 and PRO3 scores that correspond to CDAI-defined thresholds of 150, 220 and 450 and changes of 50, 70 and 100 points.

RESULTS

Optimum cut-points for CDAI remission were mean daily stool frequency ≤1.5, abdominal pain ≤1, and general well-being score of ≤1 (areas under the ROC curve 0.79, 0.91 and 0.89, respectively). The effect estimates were similar using 2- and 3-item PROs or CDAI. PRO2 and PRO3 values corresponding to CDAI scores of 150, 220 and 450 points were 8, 14, 34 and 13, 22, 53. The corresponding values for CDAI changes of 50, 70 and 100, were 2, 5, 8 and 5, 9, 14. Responsiveness to change was similar for both PROs.

CONCLUSION

Patient reported outcomes derived from CDAI diary items may be appropriate for use in clinical trials for CD.

摘要

背景

克罗恩病活动指数（CDAI）是一种基于症状、体征和实验室检查的疾病活动度测量方法。美国食品药品监督管理局指出，患者报告结局（PROs）应作为克罗恩病（CD）随机对照试验的主要结局。

目的

由于目前尚无经过验证的CD专用PRO，本研究旨在探讨能否对CDAI日记卡项目进行修改以用于此目的。

方法

使用利福昔明长效肠释制剂试验的数据，以CDAI<150为标准，通过绘制受试者工作特征曲线来确定粪便频率、疼痛和总体健康状况的切点。使用CD患者甲氨蝶呤试验的数据评估2项和3项PRO的操作特性。回归分析确定了与CDAI定义的150、220和450阈值以及50、70和100分变化相对应的PRO2和PRO3分数。

结果

CDAI缓解的最佳切点为每日平均粪便频率≤1.5、腹痛≤1和总体健康状况评分≤1（ROC曲线下面积分别为0.79、0.91和0.89）。使用2项和3项PRO或CDAI时，效应估计相似。与CDAI评分为150、220和450分相对应的PRO2和PRO3值分别为8、14、34和13、22、53。与CDAI变化50、70和100相对应的值分别为2、5、8和5、9、14。两种PRO对变化的反应性相似。