在组序贯试验中不一致使用协变量调整的解释。

IF 1.8 4区 医学 Q3 MATHEMATICAL & COMPUTATIONAL BIOLOGY
Marlena S Bannick, Sonya L Heltshe, Noah Simon
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引用次数: 0

摘要

临床试验中的组序贯设计允许对中期疗效和无效性进行监测。对基线协变量的调整可以提高估计效果的能力和精度。然而,在整个组序试验阶段不一致地应用协变量调整可能导致I型误差膨胀、有偏的点估计和反保守的置信区间。我们提出了在这种情况下执行正确的临时监测、估计和推断的方法,以避免这些问题。我们专注于简单、平衡随机化和连续结果的双组试验。我们在模拟研究中研究了边界、估计和推理调整的性能。最后提出了协变量调整在组序贯设计中的应用建议。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Accounting for Inconsistent Use of Covariate Adjustment in Group Sequential Trials.

Group sequential designs in clinical trials allow for interim efficacy and futility monitoring. Adjustment for baseline covariates can increase power and precision of estimated effects. However, inconsistently applying covariate adjustment throughout the stages of a group sequential trial can result in inflation of type I error, biased point estimates, and anticonservative confidence intervals. We propose methods for performing correct interim monitoring, estimation, and inference in this setting that avoid these issues. We focus on two-arm trials with simple, balanced randomization and continuous outcomes. We study the performance of our boundary, estimation, and inference adjustments in simulation studies. We end with recommendations about the application of covariate adjustment in group sequential designs.

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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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