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两臂均衡分配临床试验中最大耐受不平衡限制的随机化设计的稳态统计特性与实现。

Steady-state statistical properties and implementation of randomization designs with maximum tolerated imbalance restriction for two-arm equal allocation clinical trials.

机构信息

Department of Public Health Sciences, Medical University of South Carolina, Charleston, South Carolina, USA.

Biometrics and Data Sciences Department, Boehringer-Ingelheim Pharmaceuticals Inc, Ridgefield, Connecticut, USA.

出版信息

Stat Med. 2024 Mar 15;43(6):1194-1212. doi: 10.1002/sim.10013. Epub 2024 Jan 20.

Abstract

In recent decades, several randomization designs have been proposed in the literature as better alternatives to the traditional permuted block design (PBD), providing higher allocation randomness under the same restriction of the maximum tolerated imbalance (MTI). However, PBD remains the most frequently used method for randomizing subjects in clinical trials. This status quo may reflect an inadequate awareness and appreciation of the statistical properties of these randomization designs, and a lack of simple methods for their implementation. This manuscript presents the analytic results of statistical properties for five randomization designs with MTI restriction based on their steady-state probabilities of the treatment imbalance Markov chain and compares them to those of the PBD. A unified framework for randomization sequence generation and real-time on-demand treatment assignment is proposed for the straightforward implementation of randomization algorithms with explicit formulas of conditional allocation probabilities. Topics associated with the evaluation, selection, and implementation of randomization designs are discussed. It is concluded that for two-arm equal allocation trials, several randomization designs offer stronger protection against selection bias than the PBD does, and their implementation is not necessarily more difficult than the implementation of the PBD.

摘要

近几十年来,文献中提出了几种随机化设计,作为传统区组随机化设计(PBD)的更好替代方案,在相同的最大允许不平衡限制(MTI)下提供更高的分配随机性。然而,PBD 仍然是临床试验中最常用的随机化受试者的方法。这种现状可能反映了对这些随机化设计的统计性质的认识和重视不足,以及缺乏简单的方法来实现这些设计。本文基于治疗失衡马尔可夫链的稳定状态概率,对五种具有 MTI 限制的随机化设计的统计性质进行了分析,并将其与 PBD 的结果进行了比较。提出了一种基于明确的条件分配概率公式的随机化序列生成和实时按需治疗分配的统一框架,用于直接实现随机化算法。讨论了与随机化设计的评估、选择和实现相关的主题。结论是,对于双臂等分配试验,几种随机化设计比 PBD 提供了更强的保护,防止选择偏倚,并且其实施不一定比 PBD 的实施更困难。

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