在炎症性肠道疾病中对已批准的生物制剂和小分子药物进行随机对照试验的脆弱性指数分析
Xiaoyin Bai1, Ziqi Wan2, Yi Li3
1Department of Gastroenterology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing, China.
对于克罗恩病 (CD) 和性结肠炎 (UC) 中的生物制剂和小分子临床试验的稳定性使用脆弱性指数 (FI) 进行了评估. 关键试验显示出比非关键试验更大的弹性,表明治疗疗效数据的潜在漏洞.
科学领域:
- 胃肠病学和肝病学
- 临床试验方法论 临床试验方法论
- 药理学 药理学是指药理学的学科.
背景情况:
- 生物制剂和小分子越来越多地用于克罗恩病 (CD) 和性结肠炎 (UC).
- 评估这些治疗方法的临床试验的稳定性尚未被全面评估.
- 评估试验稳定性对于可靠的治疗疗效数据至关重要.
研究的目的:
- 评估随机对照试验 (RCT) 对生物药物和CD和UC中小分子的稳定性.
- 为了比较关键试验与非关键试验的稳定性.
- 确定影响试验稳健性的因素.
主要方法:
- 在2022年12月1日之前,对已批准的生物/小分子CD/UC进行RCT的系统搜索.
- 包括双臂,1: 1设计RCT.
- 每个试验的脆弱性指数 (FI) 和脆弱性系数 (FQ) 的计算.
主要成果:
- 包括28个RCT (9个关键,19个非关键).
- 关键试验中FI的中位数比非关键试验略高 (8比3).
- 样本大小和事件数量与FI显著相关;研究p值也与FI相关.
结论:
- 对CD和UC治疗的试验结果容易受到小变化的影响,如FI和FQ所示.
- 有助于获得监管批准的关键试验表现出比非关键试验更大的弹性.
- 对试验设计和报告进行进一步调查是有必要的,以确保治疗的可靠性.
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