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比例平均剩余寿命模型下联合发病和现患队列数据的分析。

Analysis of combined incident and prevalent cohort data under a proportional mean residual life model.

机构信息

Department of Biostatistics and Epidemiology, University of Massachusetts, Amherst, Massachusetts.

Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, Texas.

出版信息

Stat Med. 2019 May 30;38(12):2103-2114. doi: 10.1002/sim.8098. Epub 2019 Jan 24.

Abstract

The Nun Study, a longitudinal study to examine risk factors for the progression of dementia, consists of subjects who were already diagnosed with dementia (ie, prevalent cohort) and those who do not have dementia (ie, incident cohort) at study enrollment. When assessing the risk factors' effects on the survival time from dementia diagnosis until death, utilizing data from both cohorts supports more efficient statistical inference because the two cohorts provide valuable complementary information. A major challenge in analyzing the combined cohort data is that the prevalent cases are not representative of the target population. Moreover, the dates of dementia diagnosis are not ascertained for the prevalent cohort in the Nun Study. Hence, the survival time for the prevalent cohort is only partially observed from study enrollment until death or censoring, with the time from dementia diagnosis to study enrollment missing. In this paper, we propose an efficient estimation method that uses both incident and prevalent cohorts under the proportional mean residual life model. By assuming proportionality of the mean residual life time with covariates in the incident cohort, we can utilize the natural relationship between the mean residual life function and the hazard function of the survival time measured from enrollment until death for the prevalent cohort. We evaluate the efficiency gain from using the combined cohort data through simulations and demonstrate that the proposed method is valid and efficient.

摘要

修女研究是一项纵向研究,旨在探讨痴呆进展的风险因素,研究对象包括已经被诊断患有痴呆症的患者(即现患队列)和在研究入组时没有痴呆症的患者(即新发队列)。在评估风险因素对从痴呆症诊断到死亡的生存时间的影响时,利用两个队列的数据可以支持更有效的统计推断,因为这两个队列提供了有价值的互补信息。分析合并队列数据的一个主要挑战是,现患病例不能代表目标人群。此外,修女研究中的现患队列并未确定痴呆症的诊断日期。因此,现患队列的生存时间仅部分观察到从研究入组到死亡或删失,而从痴呆症诊断到研究入组的时间缺失。在本文中,我们提出了一种有效的估计方法,该方法在比例平均剩余寿命模型下同时利用新发和现患队列的数据。通过假设新发队列中平均剩余寿命与协变量的比例,我们可以利用平均剩余寿命函数与从入组到死亡的生存时间的风险函数之间的自然关系,来评估使用合并队列数据的效率增益。我们通过模拟验证了该方法的有效性和效率。

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