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

Author

Listed:
  • 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)

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.

Suggested Citation

  • Shuyi Liang & Kai-Tai Fang & Xin-Wei Huang & Yijing Xin & Chang-Xing Ma, 2024. "Homogeneity tests and interval estimations of risk differences for stratified bilateral and unilateral correlated data," Statistical Papers, Springer, vol. 65(6), pages 3499-3543, August.
    • Handle: RePEc:spr:stpapr:v:65:y:2024:i:6:d:10.1007_s00362-024-01532-6
    DOI: 10.1007/s00362-024-01532-6
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    References listed on IDEAS

    as
    1. Xi Shen & Chang-Xing Ma, 2018. "Testing homogeneity of difference of two proportions for stratified correlated paired binary data," Journal of Applied Statistics, Taylor & Francis Journals, vol. 45(8), pages 1410-1425, June.
    2. Shi-Fang Qiu & Li-Xuan Guo & G. Y. Zou & Dan Yu, 2019. "Tests for homogeneity of risk differences in stratified design with correlated bilateral data," Journal of Applied Statistics, Taylor & Francis Journals, vol. 46(14), pages 2491-2513, October.
    3. Zhiming Li & Changxing Ma & Mingyao Ai, 2020. "Statistical tests under Dallal’s model: Asymptotic and exact methods," PLOS ONE, Public Library of Science, vol. 15(11), pages 1-18, November.
    4. Tang, Nian-Sheng & Tang, Man-Lai & Qiu, Shi-Fang, 2008. "Testing the equality of proportions for correlated otolaryngologic data," Computational Statistics & Data Analysis, Elsevier, vol. 52(7), pages 3719-3729, March.
    5. Shi-Fang Qiu & Ji-Ran Tao, 2022. "Confidence intervals for assessing equivalence of two treatments with combined unilateral and bilateral data," Journal of Applied Statistics, Taylor & Francis Journals, vol. 49(13), pages 3414-3435, October.
    6. Changxing Ma & Guogen Shan & Song Liu, 2015. "Homogeneity Test for Correlated Binary Data," PLOS ONE, Public Library of Science, vol. 10(4), pages 1-12, April.
    7. Zhengyu Yang & Guo-Liang Tian & Xiaobin Liu & Chang-Xing Ma, 2021. "Simultaneous confidence interval construction for many-to-one comparisons of proportion differences based on correlated paired data," Journal of Applied Statistics, Taylor & Francis Journals, vol. 48(8), pages 1442-1456, June.
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