Sample size recalculation based on the overall success rate in a randomized test-treatment trial with restricting randomization to discordant pairs

被引：0

作者：

Elzner, Caroline ^{[1
]}

Pepic, Amra ^{[2
]}

Gerke, Oke ^{[3
]}

Zapf, Antonia ^{[2
]}

机构：

[1] ACOMED Stat, Fockestr 57, D-04275 Leipzig, Germany

[2] Univ Med Ctr Hamburg Eppendorf UKE, Inst Med Biometry & Epidemiol, Christoph-Probst Weg 1, D-20246 Hamburg, Germany

[3] Univ Southern Denmark, Dept Clin Res, Res Unit Clin Physiol & Nucl Med, Campusvej 55, DK-5230 Odense, Denmark

来源：

BMC MEDICAL RESEARCH METHODOLOGY | 2025年 / 25卷 / 01期

关键词：

Adaptive design; Sample size recalculation; Diagnostic test; Discordance design; Overall success rate;

D O I：

10.1186/s12874-024-02410-3

中图分类号：

R19 [保健组织与事业（卫生事业管理）];

学科分类号：

摘要：

BackgroundRandomized test-treatment studies are performed to evaluate the clinical effectiveness of diagnostic tests by assessing patient-relevant outcomes. The assumptions for a sample size calculation for such studies are often uncertain.MethodsAn adaptive design with a blinded sample size recalculation based on the overall success rate in a randomized test-treatment trial with restricting randomization to discordant pairs is proposed and evaluated by a simulation study. The results of the adaptive design are compared to those of the fixed design.ResultsThe empirical type I error rate is sufficiently controlled in the adaptive design as well as in the fixed design and the estimates are unbiased. The adaptive design achieves the desired theoretical power, whereas the fixed design tends to be over- or under-powered.ConclusionsIt may be advisable to consider blinded recalculation of sample size in a randomized test-treatment study with restriction of randomization to discordant pairs in order to improve the conduct of the study. However, there are a number of study-related limitations that affect the implementation of the method which need to be considered.

引用

页数：13

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