基于扩展长格式数据结构的多态固化模型拟合通用方法

Yilin Jiang, Harm van Tinteren, Marta Fiocco
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引用次数: 0

摘要

多状态治愈模型是一种统计框架,用于分析和描述个体在不同状态之间的时间转换,同时考虑到通过初始治疗治愈的可能性。这种模型在儿科肿瘤学中特别有用,因为有一部分患者通过治疗达到了治愈,因此他们永远不会经历某些事件。我们的研究开发了一种基于扩展长数据格式的通用算法,这种数据格式是长数据格式的扩展,在这种格式中,一个转变可以分成两行,每行分配一个权重,反映其治愈状态的后验概率。多态治愈模型适用于当前的多态模型和混合治愈模型框架。提出的算法使用了期望最大化(EM)算法和加权似然表示法,因此很容易用标准软件包实现。以欧洲血液和骨髓移植学会(EBMT)的数据为例,对该算法进行了应用。估计参数的标准误差通过非参数引导程序获得,同时还介绍了计算观测对数似然的二次衍生矩阵的方法。
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A general approach to fitting multistate cure models based on an extended-long-format data structure
A multistate cure model is a statistical framework used to analyze and represent the transitions that individuals undergo between different states over time, taking into account the possibility of being cured by initial treatment. This model is particularly useful in pediatric oncology where a fraction of the patient population achieves cure through treatment and therefore they will never experience some events. Our study develops a generalized algorithm based on the extended long data format, an extension of long data format where a transition can be split up to two rows each with a weight assigned reflecting the posterior probability of its cure status. The multistate cure model is fit on top of the current framework of multistate model and mixture cure model. The proposed algorithm makes use of the Expectation-Maximization (EM) algorithm and weighted likelihood representation such that it is easy to implement with standard package. As an example, the proposed algorithm is applied on data from the European Society for Blood and Marrow Transplantation (EBMT). Standard errors of the estimated parameters are obtained via a non-parametric bootstrap procedure, while the method involving the calculation of the second-derivative matrix of the observed log-likelihood is also presented.
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