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存在治愈比例情况下区间删失数据的半参数变换模型

Semiparametric transformation models for interval-censored data in the presence of a cure fraction.

作者信息

Chen Chyong-Mei, Shen Pao-Sheng, Huang Wei-Lun

机构信息

Institute of Public Health, School of Medicine, National Yang-Ming University, Taipei, Taiwan.

Department of Statistics, Tunghai University, Taiwan.

出版信息

Biom J. 2019 Jan;61(1):203-215. doi: 10.1002/bimj.201700304. Epub 2018 Nov 25.

Abstract

Mixed case interval-censored data arise when the event of interest is known only to occur within an interval induced by a sequence of random examination times. Such data are commonly encountered in disease research with longitudinal follow-up. Furthermore, the medical treatment has progressed over the last decade with an increasing proportion of patients being cured for many types of diseases. Thus, interest has grown in cure models for survival data which hypothesize a certain proportion of subjects in the population are not expected to experience the events of interest. In this article, we consider a two-component mixture cure model for regression analysis of mixed case interval-censored data. The first component is a logistic regression model that describes the cure rate, and the second component is a semiparametric transformation model that describes the distribution of event time for the uncured subjects. We propose semiparametric maximum likelihood estimation for the considered model. We develop an EM type algorithm for obtaining the semiparametric maximum likelihood estimators (SPMLE) of regression parameters and establish their consistency, efficiency, and asymptotic normality. Extensive simulation studies indicate that the SPMLE performs satisfactorily in a wide variety of settings. The proposed method is illustrated by the analysis of the hypobaric decompression sickness data from National Aeronautics and Space Administration.

摘要

当感兴趣的事件仅在由一系列随机检查时间所诱导的区间内发生时,就会出现混合大小写区间删失数据。这种数据在具有纵向随访的疾病研究中很常见。此外,在过去十年中,医学治疗取得了进展,许多类型疾病的治愈患者比例不断增加。因此,对于生存数据的治愈模型的兴趣日益增长,这些模型假设总体中有一定比例的受试者预计不会经历感兴趣的事件。在本文中,我们考虑用于混合大小写区间删失数据回归分析的双组分混合治愈模型。第一个组分是描述治愈率的逻辑回归模型,第二个组分是描述未治愈受试者事件时间分布的半参数变换模型。我们为所考虑的模型提出半参数极大似然估计。我们开发一种EM型算法来获得回归参数的半参数极大似然估计量(SPMLE),并建立它们的一致性、有效性和渐近正态性。大量的模拟研究表明,SPMLE在各种情况下都表现令人满意。通过对美国国家航空航天局的低压减压病数据进行分析来说明所提出的方法。

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