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用于删失生存数据的灵活参数比例风险模型和比例优势模型及其在预后建模和治疗效果估计中的应用

Flexible parametric proportional-hazards and proportional-odds models for censored survival data, with application to prognostic modelling and estimation of treatment effects.

作者信息

Royston Patrick, Parmar Mahesh K B

机构信息

Cancer Division, MRC Clinical Trials Unit, 222 Euston Road, London NW1 2DA, UK.

出版信息

Stat Med. 2002 Aug 15;21(15):2175-97. doi: 10.1002/sim.1203.

Abstract

Modelling of censored survival data is almost always done by Cox proportional-hazards regression. However, use of parametric models for such data may have some advantages. For example, non-proportional hazards, a potential difficulty with Cox models, may sometimes be handled in a simple way, and visualization of the hazard function is much easier. Extensions of the Weibull and log-logistic models are proposed in which natural cubic splines are used to smooth the baseline log cumulative hazard and log cumulative odds of failure functions. Further extensions to allow non-proportional effects of some or all of the covariates are introduced. A hypothesis test of the appropriateness of the scale chosen for covariate effects (such as of treatment) is proposed. The new models are applied to two data sets in cancer. The results throw interesting light on the behaviour of both the hazard function and the hazard ratio over time. The tools described here may be a step towards providing greater insight into the natural history of the disease and into possible underlying causes of clinical events. We illustrate these aspects by using the two examples in cancer.

摘要

删失生存数据的建模几乎总是通过Cox比例风险回归来完成。然而,对此类数据使用参数模型可能具有一些优势。例如,非比例风险这一Cox模型的潜在难题,有时可以用简单的方式处理,并且风险函数的可视化要容易得多。本文提出了Weibull模型和对数逻辑斯蒂模型的扩展形式,其中使用自然三次样条来平滑基线对数累积风险以及对数累积失败概率函数。还引入了进一步的扩展,以允许部分或所有协变量具有非比例效应。本文提出了一个关于为协变量效应(如治疗效应)选择的尺度是否合适的假设检验。新模型应用于两个癌症数据集。结果为风险函数和风险比随时间的变化行为提供了有趣的见解。这里描述的工具可能是朝着更深入了解疾病自然史以及临床事件可能的潜在原因迈出的一步。我们通过癌症中的两个例子来说明这些方面。

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