Wu Jianrong, Chen Li, Wei Jing, Weiss Heidi, Miller Rachel W, Villano John L
Division of Cancer Biostatistics, University of Kentucky, Lexington, Kentucky.
Markey Cancer Center, University of Kentucky, Lexington, Kentucky.
Pharm Stat. 2019 Mar;18(2):212-222. doi: 10.1002/pst.1916. Epub 2018 Nov 20.
Molecularly targeted, genomic-driven, and immunotherapy-based clinical trials continue to be advanced for the treatment of relapse or refractory cancer patients, where the growth modulation index (GMI) is often considered a primary endpoint of treatment efficacy. However, there little literature is available that considers the trial design with GMI as the primary endpoint. In this article, we derived a sample size formula for the score test under a log-linear model of the GMI. Study designs using the derived sample size formula are illustrated under a bivariate exponential model, the Weibull frailty model, and the generalized treatment effect size. The proposed designs provide sound statistical methods for a single-arm phase II trial with GMI as the primary endpoint.
针对复发或难治性癌症患者的治疗，分子靶向、基因组驱动和基于免疫疗法的临床试验仍在不断推进，其中生长调节指数（GMI）通常被视为治疗疗效的主要终点。然而，很少有文献考虑以GMI作为主要终点的试验设计。在本文中，我们推导了在GMI的对数线性模型下得分检验的样本量公式。在双变量指数模型、威布尔脆弱模型和广义治疗效应量下，说明了使用推导的样本量公式的研究设计。所提出的设计为以GMI作为主要终点的单臂II期试验提供了合理的统计方法。
