聚类随机试验中死亡截断的加权方法。

IF 1.6 3区 医学 Q3 HEALTH CARE SCIENCES & SERVICES
Statistical Methods in Medical Research Pub Date : 2025-03-01 Epub Date: 2025-01-31 DOI:10.1177/09622802241309348
Dane Isenberg, Michael O Harhay, Nandita Mitra, Fan Li
{"title":"聚类随机试验中死亡截断的加权方法。","authors":"Dane Isenberg, Michael O Harhay, Nandita Mitra, Fan Li","doi":"10.1177/09622802241309348","DOIUrl":null,"url":null,"abstract":"<p><p>Patient-centered outcomes, such as quality of life and length of hospital stay, are the focus in a wide array of clinical studies. However, participants in randomized trials for elderly or critically and severely ill patient populations may have truncated or undefined non-mortality outcomes if they do not survive through the measurement time point. To address truncation by death, the survivor average causal effect has been proposed as a causally interpretable subgroup treatment effect defined under the principal stratification framework. However, the majority of methods for estimating the survivor average causal effect have been developed in the context of individually randomized trials. Only limited discussions have been centered around cluster-randomized trials, where methods typically involve strong distributional assumptions for outcome modeling. In this article, we propose two weighting methods to estimate the survivor average causal effect in cluster-randomized trials that obviate the need for potentially complicated outcome distribution modeling. We establish the requisite assumptions that address latent clustering effects to enable point identification of the survivor average causal effect, and we provide computationally efficient asymptotic variance estimators for each weighting estimator. In simulations, we evaluate our weighting estimators, demonstrating their finite-sample operating characteristics and robustness to certain departures from the identification assumptions. We illustrate our methods using data from a cluster-randomized trial to assess the impact of a sedation protocol on mechanical ventilation among children with acute respiratory failure.</p>","PeriodicalId":22038,"journal":{"name":"Statistical Methods in Medical Research","volume":" ","pages":"473-489"},"PeriodicalIF":1.6000,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11951466/pdf/","citationCount":"0","resultStr":"{\"title\":\"Weighting methods for truncation by death in cluster-randomized trials.\",\"authors\":\"Dane Isenberg, Michael O Harhay, Nandita Mitra, Fan Li\",\"doi\":\"10.1177/09622802241309348\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Patient-centered outcomes, such as quality of life and length of hospital stay, are the focus in a wide array of clinical studies. However, participants in randomized trials for elderly or critically and severely ill patient populations may have truncated or undefined non-mortality outcomes if they do not survive through the measurement time point. To address truncation by death, the survivor average causal effect has been proposed as a causally interpretable subgroup treatment effect defined under the principal stratification framework. However, the majority of methods for estimating the survivor average causal effect have been developed in the context of individually randomized trials. Only limited discussions have been centered around cluster-randomized trials, where methods typically involve strong distributional assumptions for outcome modeling. In this article, we propose two weighting methods to estimate the survivor average causal effect in cluster-randomized trials that obviate the need for potentially complicated outcome distribution modeling. We establish the requisite assumptions that address latent clustering effects to enable point identification of the survivor average causal effect, and we provide computationally efficient asymptotic variance estimators for each weighting estimator. In simulations, we evaluate our weighting estimators, demonstrating their finite-sample operating characteristics and robustness to certain departures from the identification assumptions. We illustrate our methods using data from a cluster-randomized trial to assess the impact of a sedation protocol on mechanical ventilation among children with acute respiratory failure.</p>\",\"PeriodicalId\":22038,\"journal\":{\"name\":\"Statistical Methods in Medical Research\",\"volume\":\" \",\"pages\":\"473-489\"},\"PeriodicalIF\":1.6000,\"publicationDate\":\"2025-03-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11951466/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Statistical Methods in Medical Research\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1177/09622802241309348\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2025/1/31 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/10.1177/09622802241309348","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/1/31 0:00:00","PubModel":"Epub","JCR":"Q3","JCRName":"HEALTH CARE SCIENCES & SERVICES","Score":null,"Total":0}
引用次数: 0

摘要

以患者为中心的结果,如生活质量和住院时间,是广泛临床研究的焦点。然而,在针对老年人或危重患者人群的随机试验中,如果参与者没有活过测量时间点,则可能出现截断或未定义的非死亡率结果。为了解决因死亡导致的截断问题,幸存者平均因果效应被提出作为在主要分层框架下定义的可因果解释的亚组治疗效应。然而,大多数估算幸存者平均因果效应的方法都是在单独随机试验的背景下发展起来的。只有有限的讨论集中在集群随机试验上,其中的方法通常涉及结果建模的强分布假设。在本文中,我们提出了两种加权方法来估计集群随机试验中的幸存者平均因果效应,从而避免了对可能复杂的结果分布建模的需要。我们建立了解决潜在聚类效应的必要假设,以实现幸存者平均因果效应的点识别,并且我们为每个加权估计器提供计算效率高的渐近方差估计器。在模拟中,我们评估了我们的加权估计器,展示了它们的有限样本操作特性和对某些偏离识别假设的鲁棒性。我们使用一项集群随机试验的数据来说明我们的方法,以评估镇静方案对急性呼吸衰竭儿童机械通气的影响。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Weighting methods for truncation by death in cluster-randomized trials.

Patient-centered outcomes, such as quality of life and length of hospital stay, are the focus in a wide array of clinical studies. However, participants in randomized trials for elderly or critically and severely ill patient populations may have truncated or undefined non-mortality outcomes if they do not survive through the measurement time point. To address truncation by death, the survivor average causal effect has been proposed as a causally interpretable subgroup treatment effect defined under the principal stratification framework. However, the majority of methods for estimating the survivor average causal effect have been developed in the context of individually randomized trials. Only limited discussions have been centered around cluster-randomized trials, where methods typically involve strong distributional assumptions for outcome modeling. In this article, we propose two weighting methods to estimate the survivor average causal effect in cluster-randomized trials that obviate the need for potentially complicated outcome distribution modeling. We establish the requisite assumptions that address latent clustering effects to enable point identification of the survivor average causal effect, and we provide computationally efficient asymptotic variance estimators for each weighting estimator. In simulations, we evaluate our weighting estimators, demonstrating their finite-sample operating characteristics and robustness to certain departures from the identification assumptions. We illustrate our methods using data from a cluster-randomized trial to assess the impact of a sedation protocol on mechanical ventilation among children with acute respiratory failure.

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)
×
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
确定
请完成安全验证×
copy
已复制链接
快去分享给好友吧!
我知道了
右上角分享
点击右上角分享
0
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 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信