Double inverse-weighted estimation of cumulative treatment effects under nonproportional hazards and dependent censoring.

Double inverse-weighted estimation of cumulative treatment effects under nonproportional hazards and dependent censoring.
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DOI:
10.1111/j.1541-0420.2010.01449.x
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发表时间:
2011-03
期刊:
影响因子:
1.9
通讯作者:
Wei G
Wei G
中科院分区:
数学3区
文献类型:
--
作者:
Schaubel DE;Wei G

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在时间到事件数据的医学研究中,非比例风险和依赖审查是估计治疗效果时非常常见的问题。处理随时间变化的治疗效果的传统方法是对随时间变化的治疗效果进行参数化建模。这种方法的局限性包括难以验证指定功能形式的正确性,以及在存在随时间变化的治疗效果的情况下,研究人员通常对累积而不是瞬时治疗效果感兴趣。在许多应用中,滤波时间并非与事件时间无关。因此,我们提出了在存在非比例危险和依赖审查的情况下估计累积治疗效果的方法。提出了累积危险度比、相对危险度和限制平均寿命差异三个指标。对于每个测量,我们提出了一个双反加权估计器,首先使用处理加权逆概率(IPTW)来平衡处理特异性协变量分布,然后使用审查加权逆概率(IPCW)来克服依赖审查。所提出的估计量是一致的和渐近正态的。通过仿真研究了它们的有限样本性质。所提出的方法用于比较不同种族的肾脏等待名单死亡率。
In medical studies of time to event data, non-proportional hazards and dependent censoring are very common issues when estimating the treatment effect. A traditional method for dealing with time-dependent treatment effects is to model the time-dependence parametrically. Limitations of this approach include the difficulty to verify the correctness of the specified functional form and the fact that, in the presence of a treatment effect that varies over time, investigators are usually interested in the cumulative as opposed to instantaneous treatment effect. In many applications, censoring time is not independent of event time. Therefore, we propose methods for estimating the cumulative treatment effect in the presence of non-proportional hazards and dependent censoring. Three measures are proposed, including the ratio of cumulative hazards, relative risk and difference in restricted mean lifetime. For each measure, we propose a double-inverse-weighted estimator, constructed by first using inverse probability of treatment weighting (IPTW) to balance the treatment-specific covariate distributions, then using inverse probability of censoring weighting (IPCW) to overcome the dependent censoring. The proposed estimators are shown to be consistent and asymptotically normal. We study their finite-sample properties through simulation. The proposed methods are used to compare kidney wait list mortality by race.
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