On Group Sequential Enrichment Design for Basket Trial
On Group Sequential Enrichment Design for Basket Trial
复制标题
篮式试验的分组序贯富集设计
DOI：
10.1080/19466315.2016.1200999
复制
发表时间：
2016
影响因子：
1.8
通讯作者：
R. Beckman
中科院分区：
文献类型：
--
作者：
S. Yuan;A. Chen;Li He;Cong Chen;C. Gause;R. Beckman
摘要癌症正在成为一个由分子亚型定义的“小生境疾病”的集合。这种理解形成了篮子试验的基础，该试验将不同肿瘤适应症中具有相同分子亚型的多个患者队列汇集在一起，从而促进靶向治疗的开发。有效修剪在篮子设计中至关重要，因为它确保了所选适应症的内部一致性，并提高了所选适应症池的成功概率。在这篇文章中，我们考虑修剪不活跃和非常活跃的迹象，并提出了一组序贯富集设计与测试程序，以控制家庭明智的错误率（FWER）在弱意义上，并保持这样的修剪策略篮子试验的权力。我们比较建议的设计与相关的设计，只修剪无反应的子群。申报设计的总误分类率和真正无反应适应症的误分类率较小，而平均功效相似。
ABSTRACT Cancer is becoming a collection of “niche diseases” defined by the molecular subtypes. Such understanding forms the basis of the basket trial, which pools together multiple cohorts of patients with the same molecular subtype across different tumor indications and thus facilitates the development of targeted therapies. Efficient pruning is critical in basket designs as it ensures the internal consistency of the selected indications and improves the probability of success on the selected pool of indications. In this article, we consider pruning both inactive and extremely active indications and propose a group sequential enrichment design with testing procedures to both control the family-wise error rate (FWER) in the weak sense and maintain the power for basket trials with such pruning strategy. We compare the proposed design with a relevant design which prunes only unresponsive subgroups. The total misclassification rates and misclassification rates among truly unresponsive indications are smaller for the proposed design, while the average power is similar.
影响因子：
1.5
作者：
Jenkins, Martin;Stone, Andrew;Jennison, Christopher
通讯作者：
Jennison, Christopher