Homogeneity Tests and Interval Estimations of Risk Differences for Stratified Bilateral and Unilateral Correlated Data

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Main Authors: Liang, Shuyi, Fang, Kai-Tai, Huang, Xin-Wei, Xin, Yijing, Ma, Chang-Xing
Format: Preprint
Published: 2023
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author Liang, Shuyi
Fang, Kai-Tai
Huang, Xin-Wei
Xin, Yijing
Ma, Chang-Xing
author_facet Liang, Shuyi
Fang, Kai-Tai
Huang, Xin-Wei
Xin, Yijing
Ma, Chang-Xing
contents 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 \citeyearpar{dallal1988paired}, 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.
format Preprint
id arxiv_https___arxiv_org_abs_2304_00162
institution arXiv
publishDate 2023
record_format arxiv
spellingShingle Homogeneity Tests and Interval Estimations of Risk Differences for Stratified Bilateral and Unilateral Correlated Data
Liang, Shuyi
Fang, Kai-Tai
Huang, Xin-Wei
Xin, Yijing
Ma, Chang-Xing
Applications
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 \citeyearpar{dallal1988paired}, 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.
title Homogeneity Tests and Interval Estimations of Risk Differences for Stratified Bilateral and Unilateral Correlated Data
topic Applications
url https://arxiv.org/abs/2304.00162