Inference for the median residual life function in sequential multiple assignment randomized trials.

Inference for the median residual life function in sequential multiple assignment randomized trials.
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序贯多重分配随机试验中的中位剩余寿命函数的推断。

DOI:
10.1002/sim.6042
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发表时间:
2014
影响因子:
2
通讯作者:
Wahed,AbdusS
Wahed,AbdusS
中科院分区:
医学3区
文献类型:
--
作者:
Kidwell,KelleyM;Ko,JinH;Wahed,AbdusS

文献摘要

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在生存分析中,中位剩余寿命通常用作评估治疗有效性的汇总指标;然而,尚不清楚如何使用序贯随机临床试验的数据估计给定动态治疗方案的中位剩余寿命。我们提出了一种方法来估计一个动态的治疗方案特定的中位剩余寿命(MERL)的功能,从序贯多分配随机试验。我们提出的MERL估计,这是基于逆概率加权,以及,两个方差估计的MERL估计。一个方差估计来自Lunceford,Davidian和Tsiatis 2002年基于生存函数的方差估计,另一个使用三明治估计。MERL估计的评价,其两个方差估计进行了比较,通过模拟研究,表明估计和方差估计产生近似无偏的结果在大样本。为了证明我们的方法,估计已被应用到一个顺序随机白血病临床试验的数据。版权所有© 2013约翰威利父子有限公司.
In survival analysis, median residual lifetime is often used as a summary measure to assess treatment effectiveness; it is not clear, however, how such a quantity could be estimated for a given dynamic treatment regimen using data from sequential randomized clinical trials. We propose a method to estimate a dynamic treatment regimen‐specific median residual life (MERL) function from sequential multiple assignment randomized trials. We present the MERL estimator, which is based on inverse probability weighting, as well as, two variance estimates for the MERL estimator. One variance estimate follows from Lunceford, Davidian and Tsiatis' 2002 survival function‐based variance estimate and the other uses the sandwich estimator. The MERL estimator is evaluated, and its two variance estimates are compared through simulation studies, showing that the estimator and both variance estimates produce approximately unbiased results in large samples. To demonstrate our methods, the estimator has been applied to data from a sequentially randomized leukemia clinical trial. Copyright © 2013 John Wiley & Sons, Ltd.