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具有连续结果的临床试验的响应自适应随机化

Response-adaptive randomization for clinical trials with continuous outcomes.

作者信息

Zhang Lanju, Rosenberger William F

机构信息

Department of Mathematics and Statistics, University of Maryland, Baltimore County, 1000 Hilltop Circle, Baltimore, Maryland 21250, USA.

出版信息

Biometrics. 2006 Jun;62(2):562-9. doi: 10.1111/j.1541-0420.2005.00496.x.

Abstract

We provide an explicit asymptotic method to evaluate the performance of different response-adaptive randomization procedures in clinical trials with continuous outcomes. We use this method to investigate four different response-adaptive randomization procedures. Their performance, especially in power and treatment assignment skewing to the better treatment, is thoroughly evaluated theoretically. These results are then verified by simulation. Our analysis concludes that the doubly adaptive biased coin design procedure targeting optimal allocation is the best one for practical use. We also consider the effect of delay in responses and nonstandard responses, for example, Cauchy distributed response. We illustrate our procedure by redesigning a real clinical trial.

摘要

我们提供了一种显式渐近方法,用于评估具有连续结果的临床试验中不同响应自适应随机化程序的性能。我们使用该方法研究了四种不同的响应自适应随机化程序。从理论上全面评估了它们的性能,特别是在检验效能和治疗分配偏向于更优治疗方面的性能。然后通过模拟对这些结果进行了验证。我们的分析得出结论,针对最优分配的双重自适应偏倚硬币设计程序是实际应用中最好的程序。我们还考虑了响应延迟和非标准响应(例如柯西分布响应)的影响。我们通过重新设计一项真实的临床试验来说明我们的程序。

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