用于生存终点的生物标志物阈值适应性设计。
Biomarker threshold adaptive designs for survival endpoints.
作者信息
Diao Guoqing, Dong Jun, Zeng Donglin, Ke Chunlei, Rong Alan, Ibrahim Joseph G
机构信息
a Department of Statistics , George Mason University , Fairfax , Virginia , USA.
b Amgen Inc ., Thousand Oaks , California , USA.
出版信息
J Biopharm Stat. 2018;28(6):1038-1054. doi: 10.1080/10543406.2018.1434191. Epub 2018 Feb 13.
Due to the importance of precision medicine, it is essential to identify the right patients for the right treatment. Biomarkers, which have been commonly used in clinical research as well as in clinical practice, can facilitate selection of patients with a good response to the treatment. In this paper, we describe a biomarker threshold adaptive design with survival endpoints. In the first stage, we determine subgroups for one or more biomarkers such that patients in these subgroups benefit the most from the new treatment. The analysis in this stage can be based on historical or pilot studies. In the second stage, we sample subjects from the subgroups determined in the first stage and randomly allocate them to the treatment or control group. Extensive simulation studies are conducted to examine the performance of the proposed design. Application to a real data example is provided for implementation of the first-stage algorithms.
由于精准医学的重要性，确定适合正确治疗方法的合适患者至关重要。生物标志物在临床研究和临床实践中都普遍使用，它有助于选择对治疗有良好反应的患者。在本文中，我们描述了一种具有生存终点的生物标志物阈值自适应设计。在第一阶段，我们确定一种或多种生物标志物的亚组，使得这些亚组中的患者从新治疗中获益最大。此阶段的分析可以基于历史研究或先导研究。在第二阶段，我们从第一阶段确定的亚组中抽取受试者，并将他们随机分配到治疗组或对照组。我们进行了广泛的模拟研究以检验所提出设计的性能。还提供了一个实际数据示例的应用，以实现第一阶段的算法。
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