Adaptive two-stage designs for single-arm phase IIA cancer clinical trials
Adaptive two-stage designs for single-arm phase IIA cancer clinical trials
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DOI：
10.1111/j.0006-341x.2004.00193.x
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发表时间：
2004-06-01
期刊：
影响因子：
1.9
通讯作者：
Shih, WJ
中科院分区：
文献类型：
--
作者：
Lin, Y;Shih, WJ
新抗癌疗法的ITA期试验的主要目的是确定该疗法是否对特定类型的肿瘤有足够的希望，以保证其进一步发展。如果真实缓解率低于某个不感兴趣的水平，并且假设检验在特定目标缓解率下具有把握度，则拒绝该治疗进行进一步研究。两阶段设计通常用于这种情况。然而，在许多情况下，调查人员往往表示关注的不确定性，在规划阶段的目标备择假设的研究权力。在这篇文章中，由一个真实的例子的动机，我们提出了一个自适应两阶段设计的策略，将使用的信息在第一阶段的研究，要么拒绝治疗或继续测试，无论是乐观或怀疑的目标响应率，而I型错误率得到控制。我们还引入了新的最优标准，以减少预期的总样本量。
The main purpose of a phase ITA trial of a new anticancer therapy is to determine whether the therapy has sufficient promise against a specific type of tumor to warrant its further development. The therapy will be rejected for further investigation if the true response rate is less than some uninteresting level and the test of hypothesis is powered at a specific target response rate. Two-stage designs are commonly used for this situation. However, in many situations investigators often express concern about uncertainty in targeting the alternative hypothesis to study power at the planning stage. In this article, motivated by a real example, we propose a strategy for adaptive two-stage designs that will use the information at the first stage of the study to either reject the therapy or continue testing with either an optimistic or a skeptic target response rate, while the type I error rate is controlled. We also introduce new optimal criteria to reduce the expected total sample size.