The direct assignment option as a modular design component: an example for the setting of two predefined subgroups.
The direct assignment option as a modular design component: an example for the setting of two predefined subgroups.
复制标题
直接分配选项作为模块化设计组件：两个预定义亚组设置的示例。
DOI：
10.1155/2015/210817
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发表时间：
2015
影响因子：
--
通讯作者：
Mandrekar SJ
中科院分区：
文献类型：
--
作者：
An MW;Lu X;Sargent DJ;Mandrekar SJ
背景先前提出了一种II期设计，可选择直接分配（停止随机化，并根据中期分析将所有患者分配至实验治疗，IA）用于预定义的亚组。在这里，我们通过将直接赋值选项应用于两个预定义子组的设置并检验单独的子组主效应来说明该选项的模块性。方法.我们使用1 IA（DAD-1）检验2亚组直接分配选项设计的功效，以检验单独的亚组主效应，并评估在事后检验中检测相互作用的功效。模拟评估了该设计与2亚组平衡随机化设计（1 IA，BRD-1）相比的统计学特性。考虑了亚组1（0.4/0.2）和亚组2（0.1/0.2，0.4/0.2）中治疗/对照的不同缓解率。结果与2-亚组BRD-1相比，2-亚组DAD-1保留了功效和I型错误率，同时在相互作用的事后检验中表现出合理的功效。结论直接分配选项是一种灵活的设计组件，可纳入更广泛的设计框架，同时保持理想的统计特性、临床吸引力和后勤简单性。
Background. A phase II design with an option for direct assignment (stop randomization and assign all patients to experimental treatment based on interim analysis, IA) for a predefined subgroup was previously proposed. Here, we illustrate the modularity of the direct assignment option by applying it to the setting of two predefined subgroups and testing for separate subgroup main effects. Methods. We power the 2-subgroup direct assignment option design with 1 IA (DAD-1) to test for separate subgroup main effects, with assessment of power to detect an interaction in a post-hoc test. Simulations assessed the statistical properties of this design compared to the 2-subgroup balanced randomized design with 1 IA, BRD-1. Different response rates for treatment/control in subgroup 1 (0.4/0.2) and in subgroup 2 (0.1/0.2, 0.4/0.2) were considered. Results. The 2-subgroup DAD-1 preserves power and type I error rate compared to the 2-subgroup BRD-1, while exhibiting reasonable power in a post-hoc test for interaction. Conclusion. The direct assignment option is a flexible design component that can be incorporated into broader design frameworks, while maintaining desirable statistical properties, clinical appeal, and logistical simplicity.
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DOI：
10.1177/1740774510368574
发表时间：
2010-10
期刊：
Clinical trials (London, England)
影响因子：
--
作者：
Mandrekar SJ;Sargent DJ
通讯作者：
Sargent DJ
影响因子：
45.3
作者：
Freidlin, Boris;Sun, Zhuoxin;Korn, Edward L.
通讯作者：
Korn, Edward L.
影响因子：
45.3
作者：
Lievre, Astrid;Bachet, Jean-Baptiste;Laurent-Puig, Pierre
通讯作者：
Laurent-Puig, Pierre
影响因子：
2.7
作者：
Freidlin, Boris;Korn, Edward L.;Gray, Robert
通讯作者：
Gray, Robert
影响因子：
45.3
作者：
Rubinstein, LV;Korn, EL;Smith, MA
通讯作者：
Smith, MA