克罗恩病的临床和内镜结果测量的可靠性和响应性
Reena Khanna1,2, Brian G Feagan1,2,3, Guangyong Zou2,3
1Department of Medicine, University of Western Ontario, London, Ontario, Canada.
克罗恩病的试验受益于使用持续内镜测量,以提高可靠性. 结合临床和内镜数据的综合结果在克罗恩病研究中提高了对治疗效果的敏感性.
科学领域:
- 胃肠病学 胃肠病学
- 临床试验设计 临床试验设计
- 生物统计学 生物统计学
背景情况:
- 对于克罗恩病 (CD) 试验的监管指南强调评估症状和粘膜炎症的共同初级终点.
- 描述CD活动评估的性能对于试验设计和解释至关重要.
研究的目的:
- 为了评估常用的疾病活动评估在克罗恩病的操作特征.
- 为了比较内镜指数和临床结果的可靠性和响应性.
- 为了确定克罗恩病临床试验的最佳复合终点.
主要方法:
- 利用了129名参与者在克罗恩病试验中的生物和免疫调节器原始患者研究中的内镜和临床数据.
- 通过使用标准化惯例,获得克罗恩病简单内镜评分 (SES-CD) 和克罗恩病内镜严重性指数 (CDEIS).
- 通过类内相关系数和使用标准化效应大小的响应度评估指数可靠性.
主要成果:
- 持续对SES-CD和CDEIS的评分显示出相当大的评审者之间的可靠性 (ICC > 0.6).
- SES-CD,CDEIS,患者报告的结果-2和克罗恩病活动指数 (CDAI) 显示出类似的治疗反应.
- 综合结局 (CDAI < 150和CDEIS < 6) 对治疗效果最敏感 (28.9%的增加).
结论:
- 对于克罗恩病的内镜指数是更可靠的,当使用作为连续措施.
- 结合内镜和临床数据的复合终点可以提高临床试验中对治疗效果的敏感性.
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