An exact method for analysis following a two-stage phase II cancer clinical trial
An exact method for analysis following a two-stage phase II cancer clinical trial
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DOI：
10.1002/sim.3837
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发表时间：
2010-12-30
影响因子：
2
通讯作者：
Whitehead, John
中科院分区：
文献类型：
--
作者：
Jovic, Gordana;Whitehead, John
本文介绍了一种精确的方法，用于分析使用两阶段设计进行的II期癌症临床试验，其中可能允许提前停止无效或有效。该方法提供了与研究治疗相关的应答概率的点和区间估计值，以及用于检验其是否超过某个标准空值的p值。由于伦理和效率的原因，两阶段设计常用于肿瘤学II期试验，但在分析结果时很少考虑到这一设计特征。在任何两阶段设计或多阶段设计之后，分析方法应考虑以前进行的中期分析，否则结果将有偏倚。在本文中，一个近似的方法的基础上的对数比值比参数化将通过计算每种方法的精确覆盖概率与精确的方法进行比较。版权所有（C）2010约翰威利父子有限公司
This paper presents an exact method for the analysis of a phase II cancer clinical trial conducted using a two-stage design in which early stopping may be allowed for either futility or efficacy. The method provides a point and interval estimate of the response probability associated with the treatment under investigation and a p-value for testing whether this exceeds some standard null value. Two-stage designs are often used in phase II trials in oncology for reasons of ethics and efficiency, but this design feature is seldom taken into account when the results are analyzed. After any two-stage design or multi-stage design, the method for analysis should take into account the previous interim analyses performed, otherwise the results will be biased. In this paper, an approximate approach based on a log-odds ratio parameterisation will be compared with an exact method through the calculation of the precise coverage probabilities of each approach. Copyright (C) 2010 John Wiley & Sons, Ltd.