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Optimal, minimax and admissible two-stage design for phase II oncology clinical trials

机译:II期肿瘤临床试验的最佳,最小和可允许的两级设计

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摘要

Abstract Background The article aims to compare the efficiency of minimax, optimal and admissible criteria in Simon’s and Fleming’s two-stage design. Methods Three parameter settings (p 1-p 0 = 0.25–0.05, 0.30–0.10, 0.50–0.30) are designed to compare the maximum sample size, the critical values and the expected sample size for minimax, optimal and admissible designs. Type I & II error constraints (α, β) vary across (0.10, 0.10), (0.05, 0.20) and (0.05, 0.10), respectively. Results In both Simon’s and Fleming’s two-stage designs, the maximum sample size of admissible design is smaller than optimal design but larger than minimax design. Meanwhile, the expected samples size of admissible design is smaller than minimax design but larger than optimal design. Mostly, the maximum sample size and expected sample size in Fleming’s designs are considerably smaller than that of Simon’s designs. Conclusions Whenever (p 0, p 1) is pre-specified, it is better to explore in the range of probability q, based on relative importance between maximum sample size and expected sample size, and determine which design to choose. When q is unknown, optimal design may be more favorable for drugs with limited efficacy. Contrarily, minimax design is recommended if treatment demonstrates impressive efficacy.
机译:摘要背景文章旨在比较Simon和Fleming的两级设计中最低限度,最佳和可允许标准的效率。方法三个参数设置(P 1-P 0 = 0.25-0.05,0.30-0.10,0.50-0.30)旨在比较Minimax,最佳和可允许设计的最大样本大小,临界值和预期的样本量。 I&II误差约束(α,β)分别变化(0.10,0.10),(0.05,0.20)和(0.05,0.10)。导致西蒙和弗莱明的两级设计,可允许设计的最大样本尺寸小于最佳设计,但大于Minimax设计。同时,预期的样品可允许设计的尺寸小于Minimax设计,但大于最佳设计。大多数情况下,弗莱明设计中的最大样本大小和预期的样本量远远小于西蒙设计的样本量。结论每当(P 0,P 1)预先指定时,最好在概率Q范围内探讨,基于最大样本大小和预期样本大小之间的相对重要性,并确定要选择的设计。当Q未知时,最佳的设计可能更有利于有限有效的药物。相反,建议使用Minimax设计,如果治疗表明令人印象深刻的疗效。

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