Adaptive designs for single-arm phase II trials in oncology
Adaptive designs for single-arm phase II trials in oncology
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DOI：
10.1002/pst.541
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发表时间：
2012-05-01
影响因子：
1.5
通讯作者：
Kieser, Meinhard
中科院分区：
文献类型：
--
作者：
Englert, Stefan;Kieser, Meinhard
肿瘤临床II期试验是为了确定一种新的抗癌治疗方法是否有足够的前景值得进一步研究。两级设计通常用于这种情况，以便尽早终止。到目前为止，文献中提出的设计有一个共同的缺点，即两个阶段的样本量必须在方案中规定，并且必须在试验过程中严格遵守。因此，允许更高程度的灵活性的设计是可取的。在本文中，我们提出了一种新的自适应方法，允许在控制I型错误率的同时，利用中期分析的结果或外部信息任意修改第二阶段的样本量。如果在试验期间不改变样本量，所提出的设计与Chang等人（Biometrics 1987; 43:865874）提出的最佳两阶段设计的特征非常相似。然而，新的设计允许使用中期信息来规划第二阶段，从而满足肿瘤临床II期试验的实际要求。版权所有：John Wiley & Sons， Ltd。
Clinical phase II trials in oncology are conducted to determine whether the activity of a new anticancer treatment is promising enough to merit further investigation. Two-stage designs are commonly used for this situation to allow for early termination. Designs proposed in the literature so far have the common drawback that the sample sizes for the two stages have to be specified in the protocol and have to be adhered to strictly during the course of the trial. As a consequence, designs that allow a higher extent of flexibility are desirable. In this article, we propose a new adaptive method that allows an arbitrary modification of the sample size of the second stage using the results of the interim analysis or external information while controlling the type I error rate. If the sample size is not changed during the trial, the proposed design shows very similar characteristics to the optimal two-stage design proposed by Chang et al. (Biometrics 1987; 43:865874). However, the new design allows the use of mid-course information for the planning of the second stage, thus meeting practical requirements when performing clinical phase II trials in oncology. Copyright (c) 2012 John Wiley & Sons, Ltd.