双臂均等分配临床试验的稳态统计特性和具有最大可容忍不平衡限制的随机化设计的实施。

IF 1.8 4区 医学 Q3 MATHEMATICAL & COMPUTATIONAL BIOLOGY
Statistics in Medicine Pub Date : 2024-03-15 Epub Date: 2024-01-20 DOI:10.1002/sim.10013
Wenle Zhao, Kerstine Carter, Oleksandr Sverdlov, Annika Scheffold, Yevgen Ryeznik, Christy Cassarly, Vance W Berger
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引用次数: 0

摘要

近几十年来,文献中提出了几种随机化设计,作为传统的包块设计(PBD)的更好替代方案,在相同的最大可容忍不平衡(MTI)限制下提供了更高的分配随机性。然而,PBD 仍是临床试验中最常用的受试者随机分配方法。这种现状可能反映了人们对这些随机化设计的统计特性认识不足,缺乏简单的实施方法。本手稿基于治疗不平衡马尔可夫链的稳态概率,介绍了五种具有 MTI 限制的随机化设计的统计特性分析结果,并将其与 PBD 的统计特性进行了比较。本文提出了随机化序列生成和实时按需治疗分配的统一框架,以便直接实施具有明确条件分配概率公式的随机化算法。还讨论了与随机化设计的评估、选择和实施相关的主题。结论是,对于双臂等额分配试验,有几种随机化设计比 PBD 能更有效地防止选择偏差,而且它们的实施并不一定比 PBD 的实施更困难。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Steady-state statistical properties and implementation of randomization designs with maximum tolerated imbalance restriction for two-arm equal allocation clinical trials.

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.

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来源期刊
Statistics in Medicine
Statistics in Medicine 医学-公共卫生、环境卫生与职业卫生
CiteScore
3.40
自引率
10.00%
发文量
334
审稿时长
2-4 weeks
期刊介绍: The journal aims to influence practice in medicine and its associated sciences through the publication of papers on statistical and other quantitative methods. Papers will explain new methods and demonstrate their application, preferably through a substantive, real, motivating example or a comprehensive evaluation based on an illustrative example. Alternatively, papers will report on case-studies where creative use or technical generalizations of established methodology is directed towards a substantive application. Reviews of, and tutorials on, general topics relevant to the application of statistics to medicine will also be published. The main criteria for publication are appropriateness of the statistical methods to a particular medical problem and clarity of exposition. Papers with primarily mathematical content will be excluded. The journal aims to enhance communication between statisticians, clinicians and medical researchers.
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