Berger Vance W, Stefanescu Catalina, Zhou Yan Yan
University of Maryland Baltimore County and National Cancer Institute, Executive Plaza North, Suite 3131, Bethesda, MD 20892-7354, USA.
Biom J. 2006 Dec;48(6):992-1007. doi: 10.1002/bimj.200610277.
We consider the problem of testing for independence against the consistent superiority of one treatment over another when the response variable is binary and is compared across two treatments in each of several strata. Specifically, we consider the randomized clinical trial setting. A number of issues arise in this context. First, should tables be combined if there are small or zero margins? Second, should one assume a common odds ratio across strata? Third, if the odds ratios differ across strata, then how does the standard test (based on a common odds ratio) perform? Fourth, are there other analyzes that are more appropriate for handling a situation in which the odds ratios may differ across strata? In addressing these issues we find that the frequently used Cochran-Mantel-Haenszel test may have a poor power profile, despite being optimal when the odds ratios are common. We develop novel tests that are analogous to the Smirnov, modified Smirnov, convex hull, and adaptive tests that have been proposed for ordered categorical data.
当反应变量为二元变量且在几个分层中的每一层内对两种治疗方法进行比较时，我们考虑针对一种治疗方法相对于另一种治疗方法的持续优越性检验独立性的问题。具体而言，我们考虑随机临床试验设置。在此背景下会出现一些问题。首先，如果存在小的或为零的边缘频数，表格是否应该合并？其次，是否应该假设各分层的优势比相同？第三，如果各分层的优势比不同，那么标准检验（基于共同的优势比）的表现如何？第四，是否有其他分析方法更适合处理各分层优势比可能不同的情况？在解决这些问题时，我们发现尽管常用的 Cochr an-Mantel-Haenszel 检验在优势比相同时是最优的，但它的功效可能较差。我们开发了类似于针对有序分类数据提出的 Smirnov 检验、修正 Smirnov 检验、凸包检验和自适应检验的新型检验方法。
