人类健康的顺序多重分配随机试验 (SMARTs) 的设计特征:对2009年至2024年间的研究进行范围审查
Nikki L B Freeman1,2, Sydney E Browder3, Bryce Rowland4,5
1Department of Biostatistics and Bioinformatics, Duke University, Durham, North Carolina, USA nikki.freeman@duke.edu.
BMJ open
|December 30, 2025
概括
连续多重分配随机试验 (SMART) 的报告显示了变化,许多研究未充分利用其开发动态治疗方案 (DTR) 的潜力. 需要标准化指导方针来改善SMART报告和影响.
科学领域:
- 临床试验方法论 临床试验方法论
- 生物统计学 生物统计学
- 医疗保健服务研究 医疗服务研究
背景情况:
- 顺序多重分配随机试验 (SMART) 是适应性试验设计,可实现个性化治疗策略.
- 智能化治疗方案 (SMART) 有助于为顺序性临床决策制定动态治疗方案 (DTR).
- 之前的审查发现SMART报告不一致,需要进行全面评估.
研究的目的:
- 描述人类健康研究中顺序多重分配随机试验 (SMART) 的报告实践.
- 发现SMART设计,实施和分析报告中的不一致性.
- 为制定标准化的SMART特定报告准则提供信息.
主要方法:
- 进行了对2009年1月至2024年2月期间发表的SMART协议和初级分析论文的范围审查.
- 系统地搜索多个数据库,使用双重审核人员选和协商一致的意见分歧.
- 提取了关于DTR研究特征,设计元素和分析方法的数据,以描述性的方式综合发现.
主要成果:
- 103篇论文符合纳入标准,主要来自美国的行为和心理健康领域.
- 干预描述和重新随机化标准一直在报告中.
- 盲目化和随机化细节等操作特征的记录不一致;只有46.7%的初级分析评估了嵌入式DTR,没有一个探索了定制的DTR.
结论:
- 尽管采用越来越多的SMART,但在SMART中仍然存在显著的报告变化.
- 大多数初级分析并没有充分利用SMART在DTR发展方面的潜力.
- 标准化报告准则对于提高SMARTs的科学和临床影响至关重要.
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