Use of odds ratio or relative risk to measure a treatment effect in clinical trials with multiple correlated binary outcomes: data from the NINDS t-PA stroke trial

Use of odds ratio or relative risk to measure a treatment effect in clinical trials with multiple correlated binary outcomes: data from the NINDS t-PA stroke trial
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DOI:
10.1002/sim.841.abs
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发表时间:
2001-07-15
影响因子:
2
通讯作者:
Tilley, BC
Tilley, BC
中科院分区:
医学3区
文献类型:
--
作者:
Lu, M;Tilley, BC

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在临床试验中,当一个单一的结果不足以描述感兴趣的潜在概念时,可能有必要在多个相关结果上比较治疗组。基于Logit链接函数的全局测试提供了用于评估相关二元结果中的共同治疗效果的赔率比的估计。在本文中,我们扩展了广义估计方程(GEE)的使用,以对数连接函数为基础,从相关的二元结果中计算公共相对风险。在全局测试的背景下,我们讨论了LOGIT和LOG链接的等价性和差异性及其估计。我们还推导了基于多个相关的二元结果和类别协变量的两个处理组之间的共同风险差异的计算公式,假设Logit和对数线性链接之间的渐近等价。我们讨论了当不同链接上的模型产生不同结果时,在Logit和LOG链接之间进行选择时使用的统计工具。提供了使用NINDS t-PA卒中试验数据的例子。在对相关的二元结果的研究中,我们得出结论,LOGIT或LOG链接的选择可以基于链接好度的比较,版权所有(C)2001 John Wiley&Sons,Ltd.。
In clinical trials, when a single outcome is not sufficient to describe the underlying concept of interest, it may be necessary to compare treatment groups on multiple correlated outcomes. A global test based on a logit link function provides an estimate of the odds ratio for assessing a common treatment effect among correlated binary outcomes. In this paper we extend the use of generalized estimating equations (GEE) to calculate a common relative risk from correlated binary outcomes based on a log link function. In the context of global tests, we discuss the equivalence and difference between logit and log links and their estimates. We also derive a formula for calculating a common risk difference between two treatment groups based on multiple correlated binary outcomes with categorical covariates, assuming the asymptotic equivalency between the logit and log-linear links. We discuss the statistical tools to be used in choosing between the logit and log links when models on different links yield contrasting results. Examples using data from the NINDS t-PA Stroke Trials are provided. We conclude, in a study of correlated binary outcomes, that the choice of the logit or log link could be based on a comparison of goodness-of-link, Copyright (C) 2001 John Wiley & Sons, Ltd.