Exact group-sequential designs for clinical trials with randomized play-the-winner allocation.
Exact group-sequential designs for clinical trials with randomized play-the-winner allocation.
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具有随机胜者分配的临床试验的精确组序贯设计。
DOI：
10.1002/sim.998
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发表时间：
2002
影响因子：
2
通讯作者：
Rosenberger,WilliamF
中科院分区：
文献类型：
--
作者：
Stallard,Nigel;Rosenberger,WilliamF
序贯设计和适应性治疗分配的使用可有效减少临床试验中接受劣效治疗的患者数量。在大样本情况下，当检验统计量的渐近正态性可以利用时，标准序贯设计可以与适应性分配相结合。在小样本中，如果假设正态性，则可能不满足计划错误率约束。我们通过考虑特定自适应分配方案下检验统计量的精确分布来构造具有指定性质的序贯停止规则来解决这个问题，随机化赢家规则。使用这种方法，与传统的平等分配试验相比，试验与自适应分配需要更大的总样本量，以达到一个给定的权力。更有趣的是，根据真实成功率，自适应分配设计中分配至劣效治疗的预期患者数量也可能更大。版权所有© 2002年约翰威利父子有限公司。
The use of both sequential designs and adaptive treatment allocation are effective in reducing the number of patients receiving an inferior treatment in a clinical trial. In large samples, when the asymptotic normality of test statistics can be utilized, a standard sequential design can be combined with adaptive allocation. In small samples the planned error rate constraints may not be satisfied if normality is assumed. We address this problem by constructing sequential stopping rules with specified properties by consideration of the exact distribution of test statistics under a particular adaptive allocation scheme, the randomized play‐the‐winner rule. Using this approach, compared to traditional equal allocation trials, trials with adaptive allocation are shown to require a larger total sample size to achieve a given power. More interestingly, the expected number patients allocated to the inferior treatment may also be larger for the adaptive allocation designs depending on the true success rates. Copyright © 2002 John Wiley & Sons, Ltd.