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

IF 3.9 3区医学 Q1 HEALTH CARE SCIENCES & SERVICES

BMC Medical Research Methodology Pub Date : 2025-03-18 DOI:10.1186/s12874-024-02410-3

Caroline Elzner, Amra Pepić, Oke Gerke, Antonia Zapf

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Abstract

Background: Randomized 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.

Methods: An 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.

Results: The 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.

Conclusions: It 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.

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来源期刊

BMC Medical Research Methodology 医学-卫生保健

CiteScore

6.50

自引率

2.50%

发文量

298

审稿时长

3-8 weeks

期刊介绍： BMC Medical Research Methodology is an open access journal publishing original peer-reviewed research articles in methodological approaches to healthcare research. Articles on the methodology of epidemiological research, clinical trials and meta-analysis/systematic review are particularly encouraged, as are empirical studies of the associations between choice of methodology and study outcomes. BMC Medical Research Methodology does not aim to publish articles describing scientific methods or techniques: these should be directed to the BMC journal covering the relevant biomedical subject area.