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半参数估计受限平均生存时间作为限制时间的函数。

Semiparametric estimation of restricted mean survival time as a function of restriction time.

机构信息

School of Statistics, University of International Business and Economics, Beijing, China.

出版信息

Stat Med. 2023 Dec 20;42(29):5389-5404. doi: 10.1002/sim.9918. Epub 2023 Sep 22.

Abstract

The restricted mean survival time (RMST) is an appealing measurement in clinical or epidemiological studies with censored survival outcome and receives a lot of attention in the past decades. It provides a useful alternative to the Cox model for evaluating the covariate effect on survival time. The covariate effect on RMST usually varies with the restriction time. However, existing methods cannot address this problem properly. In this article, we propose a semiparametric framework that directly models RMST as a function of the restriction time. Our proposed model adopts a widely-used proportional form, enabling the estimation of RMST predictions across an interval using a unified model. Furthermore, the covariate effect for multiple restriction time points can be derived simultaneously. We develop estimators based on estimating equations theories and establish the asymptotic properties of the proposed estimators. The finite sample properties of the estimators are evaluated through extensive simulation studies. We further illustrate the application of our proposed method through the analysis of two real data examples. Supplementary Material are available online.

摘要

限制平均生存时间(RMST)是一种在有删失生存结果的临床或流行病学研究中很有吸引力的测量方法,在过去几十年中受到了广泛关注。它为评估生存时间上的协变量效应提供了一种有用的替代 Cox 模型的方法。协变量对 RMST 的影响通常随限制时间而变化。然而,现有的方法不能很好地解决这个问题。在本文中,我们提出了一个半参数框架,直接将 RMST 建模为限制时间的函数。我们提出的模型采用了广泛使用的比例形式,能够使用统一的模型在一个区间内估计 RMST 预测值。此外,还可以同时推导出多个限制时间点的协变量效应。我们基于估计方程理论开发了估计量,并建立了所提出的估计量的渐近性质。通过广泛的模拟研究评估了估计量的有限样本性质。我们通过对两个真实数据示例的分析进一步说明了我们提出的方法的应用。补充材料可在线获取。

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