Estimation of survival distributions of treatment policies in two-stage randomization designs in clinical trials
Estimation of survival distributions of treatment policies in two-stage randomization designs in clinical trials
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DOI：
10.1111/j.0006-341x.2002.00048.x
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发表时间：
2002-03-01
期刊：
影响因子：
1.9
通讯作者：
Tsiatis, AA
中科院分区：
文献类型：
--
作者：
Lunceford, JK;Davidian, M;Tsiatis, AA
一些临床试验遵循这样的设计，即患者在入组时随机接受主要治疗，然后根据疾病缓解情况再随机接受维持治疗。理想情况下，分析将允许不同的治疗策略，即，如果预先指定，则将主要治疗和维持治疗组合进行比较。标准做法是对初次和后续头端治疗进行单独分析，这并不直接解决该问题。我们提出了一致的估计生存分布和平均限制生存调整为每个治疗政策，在这样的两个阶段的研究，并获得大样本的性质。白血病临床试验数据集和通过模拟的方法证明。
Some clinical trials follow a design where patients are randomized to a primary therapy at entry followed by another randomization to maintenance therapy contingent upon disease remission. Ideally, analysis would allow different treatment policies, i.e., combinations of primary and maintenance therapy if specified up-front, to be compared. Standard practice is to conduct separate analyses for the primary and follow-tip treatments, which does not address this issue directly. We propose consistent estimators for the survival distribution and mean restricted survival tune for each treatment policy in such two-stage studies and derive large-sample properties. The methods are demonstrated on a leukemia clinical trial data set and through simulation.