Zhao Wenle, Hill Michael D, Palesch Yuko
Division of Biostatistics and Epidemiology, Medical University of South Carolina, USA
Department of Clinical Neurosciences and Hotchkiss Brain Institute, University of Calgary, Canada.
Stat Methods Med Res. 2015 Dec;24(6):989-1002. doi: 10.1177/0962280212436447. Epub 2012 Jan 26.
In many clinical trials, baseline covariates could affect the primary outcome. Commonly used strategies to balance baseline covariates include stratified constrained randomization and minimization. Stratification is limited to few categorical covariates. Minimization lacks the randomness of treatment allocation. Both apply only to categorical covariates. As a result, serious imbalances could occur in important baseline covariates not included in the randomization algorithm. Furthermore, randomness of treatment allocation could be significantly compromised because of the high proportion of deterministic assignments associated with stratified block randomization and minimization, potentially resulting in selection bias. Serious baseline covariate imbalances and selection biases often contribute to controversial interpretation of the trial results. The National Institute of Neurological Disorders and Stroke recombinant tissue plasminogen activator Stroke Trial and the Captopril Prevention Project are two examples. In this article, we propose a new randomization strategy, termed the minimal sufficient balance randomization, which will dually prevent serious imbalances in all important baseline covariates, including both categorical and continuous types, and preserve the randomness of treatment allocation. Computer simulations are conducted using the data from the National Institute of Neurological Disorders and Stroke recombinant tissue plasminogen activator Stroke Trial. Serious imbalances in four continuous and one categorical covariate are prevented with a small cost in treatment allocation randomness. A scenario of simultaneously balancing 11 baseline covariates is explored with similar promising results. The proposed minimal sufficient balance randomization algorithm can be easily implemented in computerized central randomization systems for large multicenter trials.
在许多临床试验中,基线协变量可能会影响主要结局。平衡基线协变量的常用策略包括分层约束随机化和最小化法。分层仅限于少数分类协变量。最小化法缺乏治疗分配的随机性。两者都仅适用于分类协变量。因此,在随机化算法未包含的重要基线协变量中可能会出现严重失衡。此外,由于与分层区组随机化和最小化法相关的确定性分配比例很高,治疗分配的随机性可能会受到显著影响,从而可能导致选择偏倚。严重的基线协变量失衡和选择偏倚往往会导致对试验结果的解释存在争议。美国国立神经疾病与中风研究所重组组织型纤溶酶原激活剂中风试验和卡托普利预防项目就是两个例子。在本文中,我们提出了一种新的随机化策略,称为最小充分平衡随机化,它将双重防止所有重要基线协变量(包括分类和连续类型)出现严重失衡,并保持治疗分配的随机性。使用美国国立神经疾病与中风研究所重组组织型纤溶酶原激活剂中风试验的数据进行了计算机模拟。在治疗分配随机性方面付出较小代价的情况下,防止了四个连续协变量和一个分类协变量出现严重失衡。探索了同时平衡11个基线协变量的情况,结果同样令人满意。所提出的最小充分平衡随机化算法可以很容易地在大型多中心试验的计算机化中央随机化系统中实现。