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随机对照试验的系统审查,采用适应性和传统组序列设计 - - 在心血管临床试验中的应用
Jufen Zhang1,2, Christy Saju3
1School of Medicine, Faculty of Health, Education, Medicine and Social Care, Anglia Ruskin University, Bishop Hall Lane, Chelmsford, CM1 1SQ, U.K.. Jufen.Zhang@aru.ac.uk.
BMC medical research methodology
|September 7, 2023
概括
适应性试验设计在心血管临床试验中比传统的组序列设计更具灵活性. 虽然自适应设计的使用越来越多,但其报告需要改进,以提高清晰度和可发现性.
科学领域:
- 临床试验设计 临床试验设计
- 心血管研究研究心血管研究
- 生物统计学 生物统计学
背景情况:
- 临床试验设计对试验结果和资源分配产生重大影响.
- 传统的组序列设计允许提前停止,但缺乏灵活性.
- 适应性设计通过基于中间分析进行修改,提高了灵活性和效率.
研究的目的:
- 系统地审查和描述心血管研究中的适应性和传统群体顺序试验设计.
- 了解心脏病学文献中这些试验设计的当前报告实践.
主要方法:
- 在控制试验的PubMed,Embase和Cochrane中央注册表 (1980年1月 - 2022年6月) 的系统文献搜索.
- 包括心血管疾病患者随机控制的2/3期试验,使用适应性或组序列设计.
- 关于试验特征和报告的收集数据的描述性统计分析.
主要成果:
- 包括56个试验:43个 (76.8%) 使用传统的组序列设计,13个 (23.2%) 使用适应性设计.
- 适应性设计显示使用率增加,特别是在2015年 (38.6%) 之后. 样本大小的重新估计是常见的 (61.5%).
- 早期停止发生在67%的群体顺序试验和54%的适应性试验中,原因各不相同 (有效性,徒劳性).
结论:
- 适应性试验设计因其灵活性而在心血管临床试验中越来越受欢迎.
- 需要改善心脏病学研究中适应性设计的报告标准.
- 对适应性试验的最佳报告准则进行进一步的研究是有必要的.
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