Thall P F, Estey E H
Department of Biomathematics, M.D. Anderson Cancer Center, University of Texas, Houston 77030.
Stat Med. 1993 Jul 15;12(13):1197-211. doi: 10.1002/sim.4780121303.
We address the problem of selecting a treatment for phase II evaluation when several candidate treatments emerging from phase I testing are available. A pre-phase II Bayesian selection design which randomizes patients among treatments is proposed. The patient group in the trial has prognosis more favourable than that of phase I patients but less favourable than the target group of the subsequent phase II trial. The patient response rate distribution in each treatment arm is continually updated during the trial for comparison with early termination cutoffs, and the best final treatment must satisfy a minimal posterior efficacy criterion. The primary aim is to replace the usual informal treatment selection process with a fair comparison formally based on a combination of prior opinion and clinical data.
当有几种来自I期试验的候选治疗方法可供选择时,我们着手解决选择用于II期评估的治疗方法这一问题。我们提出了一种II期前的贝叶斯选择设计,该设计在不同治疗方法之间对患者进行随机分组。试验中的患者组预后比I期患者更有利,但比后续II期试验的目标组更不利。在试验过程中,每个治疗组的患者反应率分布会不断更新,以便与早期终止临界值进行比较,并且最佳的最终治疗方法必须满足最小的后验疗效标准。主要目的是用一种基于先验观点和临床数据相结合的正式公平比较,来取代通常的非正式治疗选择过程。