Homogeneity tests and interval estimations of risk differences for stratified bilateral and unilateral correlated data

Shuyi Liang, Kai-Tai Fang, Xin-Wei Huang, Yijing Xin and Chang-Xing Ma ()
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Shuyi Liang: University at Buffalo
Kai-Tai Fang: Beijing Normal University–Hong Kong Baptist University United International College
Xin-Wei Huang: University at Buffalo
Yijing Xin: The First Affiliated Hospital of Xiamen University
Chang-Xing Ma: University at Buffalo

Statistical Papers, 2024, vol. 65, issue 6, No 7, 3499-3543

Abstract: Abstract In clinical trials studying paired parts of a subject with binary outcomes, it is expected to collect measurements bilaterally. However, there are cases where subjects contribute measurements for only one part. By utilizing combined data, it is possible to gain additional information compared to using bilateral or unilateral data alone. With the combined data, this article investigates homogeneity tests of risk differences with the presence of stratification effects and proposes interval estimations of a common risk difference if stratification does not introduce underlying dissimilarities. Under Dallal’s model (Biometrics 44:253–257, 1988), we propose three test statistics and evaluate their performances regarding type I error controls and powers. Confidence intervals of a common risk difference with satisfactory coverage probabilities and interval length are constructed. Our simulation results show that the score test is the most robust and the profile likelihood confidence interval outperforms other methods proposed. Data from a study of acute otitis media is used to illustrate our proposed procedures.

Keywords: Dallal’s model; Homogeneity test; Confidence interval; Stratified design; Unilateral and bilateral data; Risk difference (search for similar items in EconPapers)
Date: 2024
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DOI: 10.1007/s00362-024-01532-6

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