在数据缺失的情况下,将次要结果信息可靠地纳入主要结果分析。

IF 1.6 3区 医学 Q3 HEALTH CARE SCIENCES & SERVICES Statistical Methods in Medical Research Pub Date : 2024-07-01 Epub Date: 2024-05-20 DOI:10.1177/09622802241254195
Daxuan Deng, Vernon M Chinchilli, Hao Feng, Chixiang Chen, Ming Wang
{"title":"在数据缺失的情况下,将次要结果信息可靠地纳入主要结果分析。","authors":"Daxuan Deng, Vernon M Chinchilli, Hao Feng, Chixiang Chen, Ming Wang","doi":"10.1177/09622802241254195","DOIUrl":null,"url":null,"abstract":"<p><p>In clinical and observational studies, secondary outcomes are frequently collected alongside the primary outcome for each subject, yet their potential to improve the analysis efficiency remains underutilized. Moreover, missing data, commonly encountered in practice, can introduce bias to estimates if not appropriately addressed. This article presents an innovative approach that enhances the empirical likelihood-based information borrowing method by integrating missing-data techniques, ensuring robust data integration. We introduce a plug-in inverse probability weighting estimator to handle missingness in the primary analysis, demonstrating its equivalence to the standard joint estimator under mild conditions. To address potential bias from missing secondary outcomes, we propose a uniform mapping strategy, imputing incomplete secondary outcomes into a unified space. Extensive simulations highlight the effectiveness of our method, showing consistent, efficient, and robust estimators under various scenarios involving missing data and/or misspecified secondary models. Finally, we apply our proposal to the Uniform Data Set from the National Alzheimer's Coordinating Center, exemplifying its practical application.</p>","PeriodicalId":22038,"journal":{"name":"Statistical Methods in Medical Research","volume":" ","pages":"1249-1263"},"PeriodicalIF":1.6000,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Robust integration of secondary outcomes information into primary outcome analysis in the presence of missing data.\",\"authors\":\"Daxuan Deng, Vernon M Chinchilli, Hao Feng, Chixiang Chen, Ming Wang\",\"doi\":\"10.1177/09622802241254195\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>In clinical and observational studies, secondary outcomes are frequently collected alongside the primary outcome for each subject, yet their potential to improve the analysis efficiency remains underutilized. Moreover, missing data, commonly encountered in practice, can introduce bias to estimates if not appropriately addressed. This article presents an innovative approach that enhances the empirical likelihood-based information borrowing method by integrating missing-data techniques, ensuring robust data integration. We introduce a plug-in inverse probability weighting estimator to handle missingness in the primary analysis, demonstrating its equivalence to the standard joint estimator under mild conditions. To address potential bias from missing secondary outcomes, we propose a uniform mapping strategy, imputing incomplete secondary outcomes into a unified space. Extensive simulations highlight the effectiveness of our method, showing consistent, efficient, and robust estimators under various scenarios involving missing data and/or misspecified secondary models. Finally, we apply our proposal to the Uniform Data Set from the National Alzheimer's Coordinating Center, exemplifying its practical application.</p>\",\"PeriodicalId\":22038,\"journal\":{\"name\":\"Statistical Methods in Medical Research\",\"volume\":\" \",\"pages\":\"1249-1263\"},\"PeriodicalIF\":1.6000,\"publicationDate\":\"2024-07-01\",\"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/10.1177/09622802241254195\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2024/5/20 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/10.1177/09622802241254195","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/5/20 0:00:00","PubModel":"Epub","JCR":"Q3","JCRName":"HEALTH CARE SCIENCES & SERVICES","Score":null,"Total":0}
引用次数: 0

摘要

在临床和观察性研究中,在收集每个受试者的主要结果的同时,经常会收集次要结果,但它们在提高分析效率方面的潜力仍未得到充分利用。此外,在实践中经常会遇到数据缺失的情况,如果处理不当,会给估计结果带来偏差。本文提出了一种创新方法,通过整合缺失数据技术来增强基于经验似然法的信息借用方法,确保稳健的数据整合。我们引入了一种插件式反概率加权估计器来处理主要分析中的缺失,并证明了在温和条件下它与标准联合估计器的等效性。为了解决次要结果缺失可能造成的偏差,我们提出了一种统一映射策略,将不完整的次要结果归入一个统一的空间。大量的模拟突出了我们方法的有效性,显示了在涉及缺失数据和/或次级模型失当的各种情况下,我们的估计方法是一致、高效和稳健的。最后,我们将我们的建议应用于国家阿尔茨海默氏症协调中心的统一数据集,举例说明其实际应用。
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
Robust integration of secondary outcomes information into primary outcome analysis in the presence of missing data.

In clinical and observational studies, secondary outcomes are frequently collected alongside the primary outcome for each subject, yet their potential to improve the analysis efficiency remains underutilized. Moreover, missing data, commonly encountered in practice, can introduce bias to estimates if not appropriately addressed. This article presents an innovative approach that enhances the empirical likelihood-based information borrowing method by integrating missing-data techniques, ensuring robust data integration. We introduce a plug-in inverse probability weighting estimator to handle missingness in the primary analysis, demonstrating its equivalence to the standard joint estimator under mild conditions. To address potential bias from missing secondary outcomes, we propose a uniform mapping strategy, imputing incomplete secondary outcomes into a unified space. Extensive simulations highlight the effectiveness of our method, showing consistent, efficient, and robust estimators under various scenarios involving missing data and/or misspecified secondary models. Finally, we apply our proposal to the Uniform Data Set from the National Alzheimer's Coordinating Center, exemplifying its practical application.

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
LASSO-type instrumental variable selection methods with an application to Mendelian randomization. Estimating an adjusted risk difference in a cluster randomized trial with individual-level analyses. Testing for a treatment effect in a selected subgroup. Enhancing DHA supplementation adherence: A Bayesian approach with finite mixture models and irregular interim schedules in adaptive trial designs. Analysis of recurrent event data with spatial random effects using a Bayesian approach.
×
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