测试多组配对二进制数据的响应率函数的相等性。

IF 1.6 3区 医学 Q3 HEALTH CARE SCIENCES & SERVICES Statistical Methods in Medical Research Pub Date : 2025-01-01 Epub Date: 2024-12-10 DOI:10.1177/09622802241292672
Yufei Liu, Zhiming Li, Keyi Mou, Junhong Du
{"title":"测试多组配对二进制数据的响应率函数的相等性。","authors":"Yufei Liu, Zhiming Li, Keyi Mou, Junhong Du","doi":"10.1177/09622802241292672","DOIUrl":null,"url":null,"abstract":"<p><p>In clinical trials, we often encounter observations from patients' paired organs. In paired correlated data, there exist various measures to evaluate the therapeutic responses, such as risk difference, relative risk ratio, and odds ratio. These measures are essentially some forms of response rate functions. Based on this point, this article aims to test the equality of response rate functions such that the homogeneity tests of the above measures are special cases. Under an interclass correlation model, the global and constrained maximum likelihood estimations are obtained through algorithms. Furthermore, we construct likelihood ratio, score, and Wald-type statistics and provide the explicit expressions of the corresponding tests based on the risk difference, relative risk ratio, and odds ratio. Monte Carlo simulations are conducted to compare the performance of the proposed methods in terms of the empirical type I error rates and powers. The results show that the score tests perform satisfactorily as their type I error rates are close to the specified nominal level, followed by the likelihood ratio test. The Wald-type tests exhibit poor performance, especially for small sample sizes. A real example is given to illustrate the three proposed test statistics.</p>","PeriodicalId":22038,"journal":{"name":"Statistical Methods in Medical Research","volume":" ","pages":"131-149"},"PeriodicalIF":1.6000,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Testing the equality of response rate functions for paired binary data with multiple groups.\",\"authors\":\"Yufei Liu, Zhiming Li, Keyi Mou, Junhong Du\",\"doi\":\"10.1177/09622802241292672\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>In clinical trials, we often encounter observations from patients' paired organs. In paired correlated data, there exist various measures to evaluate the therapeutic responses, such as risk difference, relative risk ratio, and odds ratio. These measures are essentially some forms of response rate functions. Based on this point, this article aims to test the equality of response rate functions such that the homogeneity tests of the above measures are special cases. Under an interclass correlation model, the global and constrained maximum likelihood estimations are obtained through algorithms. Furthermore, we construct likelihood ratio, score, and Wald-type statistics and provide the explicit expressions of the corresponding tests based on the risk difference, relative risk ratio, and odds ratio. Monte Carlo simulations are conducted to compare the performance of the proposed methods in terms of the empirical type I error rates and powers. The results show that the score tests perform satisfactorily as their type I error rates are close to the specified nominal level, followed by the likelihood ratio test. The Wald-type tests exhibit poor performance, especially for small sample sizes. A real example is given to illustrate the three proposed test statistics.</p>\",\"PeriodicalId\":22038,\"journal\":{\"name\":\"Statistical Methods in Medical Research\",\"volume\":\" \",\"pages\":\"131-149\"},\"PeriodicalIF\":1.6000,\"publicationDate\":\"2025-01-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Statistical Methods in Medical Research\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1177/09622802241292672\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2024/12/10 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q3\",\"JCRName\":\"HEALTH CARE SCIENCES & SERVICES\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Statistical Methods in Medical Research","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1177/09622802241292672","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/12/10 0:00:00","PubModel":"Epub","JCR":"Q3","JCRName":"HEALTH CARE SCIENCES & SERVICES","Score":null,"Total":0}
引用次数: 0

摘要

在临床试验中,我们经常会遇到来自患者配对器官的观察。在配对相关数据中,存在各种评价治疗反应的指标,如风险差异、相对风险比、优势比等。这些措施本质上是某种形式的响应率函数。基于这一点,本文旨在检验响应率函数的平等性,使上述测度的同质性检验成为特例。在类间相关模型下,通过算法得到全局和约束最大似然估计。此外,我们构建了似然比、得分和wald型统计,并根据风险差异、相对风险比和优势比给出了相应检验的显式表达式。通过蒙特卡罗仿真比较了所提出方法在经验I型错误率和功率方面的性能。结果表明,分数检验的ⅰ类错误率接近规定的标称水平,结果令人满意,其次是似然比检验。wald型测试表现出较差的性能,特别是对于小样本量。给出了一个实际的例子来说明这三种测试统计量。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
查看原文
分享 分享
微信好友 朋友圈 QQ好友 复制链接
本刊更多论文
Testing the equality of response rate functions for paired binary data with multiple groups.

In clinical trials, we often encounter observations from patients' paired organs. In paired correlated data, there exist various measures to evaluate the therapeutic responses, such as risk difference, relative risk ratio, and odds ratio. These measures are essentially some forms of response rate functions. Based on this point, this article aims to test the equality of response rate functions such that the homogeneity tests of the above measures are special cases. Under an interclass correlation model, the global and constrained maximum likelihood estimations are obtained through algorithms. Furthermore, we construct likelihood ratio, score, and Wald-type statistics and provide the explicit expressions of the corresponding tests based on the risk difference, relative risk ratio, and odds ratio. Monte Carlo simulations are conducted to compare the performance of the proposed methods in terms of the empirical type I error rates and powers. The results show that the score tests perform satisfactorily as their type I error rates are close to the specified nominal level, followed by the likelihood ratio test. The Wald-type tests exhibit poor performance, especially for small sample sizes. A real example is given to illustrate the three proposed test statistics.

求助全文
通过发布文献求助,成功后即可免费获取论文全文。 去求助
来源期刊
Statistical Methods in Medical Research
Statistical Methods in Medical Research 医学-数学与计算生物学
CiteScore
4.10
自引率
4.30%
发文量
127
审稿时长
>12 weeks
期刊介绍: Statistical Methods in Medical Research is a peer reviewed scholarly journal and is the leading vehicle for articles in all the main areas of medical statistics and an essential reference for all medical statisticians. This unique journal is devoted solely to statistics and medicine and aims to keep professionals abreast of the many powerful statistical techniques now available to the medical profession. This journal is a member of the Committee on Publication Ethics (COPE)
期刊最新文献
Extension of Fisher's least significant difference method to multi-armed group-sequential response-adaptive designs. Generalized framework for identifying meaningful heterogenous treatment effects in observational studies: A parametric data-adaptive G-computation approach. The relative efficiency of staircase and stepped wedge cluster randomised trial designs. Bayesian mixture models for phylogenetic source attribution from consensus sequences and time since infection estimates. Jointly assessing multiple endpoints in pilot and feasibility studies.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
现在去查看 取消
×
提示
确定
0
微信
客服QQ
Book学术公众号 扫码关注我们
反馈
×
意见反馈
请填写您的意见或建议
请填写您的手机或邮箱
已复制链接
已复制链接
快去分享给好友吧!
我知道了
×
扫码分享
扫码分享
Book学术官方微信
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术
文献互助 智能选刊 最新文献 互助须知 联系我们:info@booksci.cn
Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。
Copyright © 2023 Book学术 All rights reserved.
ghs 京公网安备 11010802042870号 京ICP备2023020795号-1