Shan Guogen
Department of Epidemiology and Biostatistics, School of Public Health, University of Nevada Las Vegas, Las Vegas, NV, 89154, USA.
Contemp Clin Trials Commun. 2021 Jan 23;21:100732. doi: 10.1016/j.conctc.2021.100732. eCollection 2021 Mar.
Restricted mean survival time is an alternative measure of treatment effect to hazard ratio in clinical trials with time-to-event outcome. The current methods have been focused on one-stage designs. In this article, we propose optimal two-stage designs for a single-arm study with the smallest expected sample size. We compare the performance of the new optimal two-stage designs with the existing one-stage design with regards to the expected sample size and the expected total study length. The simulation results indicate that the new two-stage designs can save the expected sample size substantially as compared to the one-stage design. We use a non-small cell lung cancer trial to illustrate the application of the proposed designs. The proposed optimal two-stage designs are recommended for use when time for patient accrual is longer than the pre-specified follow-up time.
在具有事件发生时间结局的临床试验中，受限平均生存时间是一种替代风险比的治疗效果衡量指标。目前的方法主要集中在单阶段设计上。在本文中，我们针对单臂研究提出了具有最小期望样本量的最优两阶段设计。我们在期望样本量和期望总研究时长方面，将新的最优两阶段设计与现有的单阶段设计的性能进行了比较。模拟结果表明，与单阶段设计相比，新的两阶段设计可以大幅节省期望样本量。我们使用一项非小细胞肺癌试验来说明所提出设计的应用。当患者入组时间长于预先指定的随访时间时，建议使用所提出的最优两阶段设计。
