因严重急性呼吸系统综合征--冠状病毒-2(SARS-CoV-2)大流行而中断的临床试验的统计方法:综述。

IF 1.6 3区 医学 Q3 HEALTH CARE SCIENCES & SERVICES
Statistical Methods in Medical Research Pub Date : 2024-11-01 Epub Date: 2024-10-30 DOI:10.1177/09622802241288350
Joydeep Basu, Nicholas Parsons, Tim Friede, Nigel Stallard
{"title":"因严重急性呼吸系统综合征--冠状病毒-2(SARS-CoV-2)大流行而中断的临床试验的统计方法:综述。","authors":"Joydeep Basu, Nicholas Parsons, Tim Friede, Nigel Stallard","doi":"10.1177/09622802241288350","DOIUrl":null,"url":null,"abstract":"<p><p>Cancellation or delay of non-essential medical interventions, limitation of face-to-face assessments or outpatient attendance due to lockdown restrictions, illness or fear of hospital or healthcare centre visits, and halting of research to allow diversion of healthcare resources to focus on the pandemic led to the interruption of many clinical trials during the severe acute respiratory syndrome-coronavirus-2 (SARS-CoV-2) pandemic. Appropriate analysis approaches are now required for these interrupted trials. In trials with long follow-up and longitudinal outcomes, data may be available on early outcomes for many patients for whom final, primary outcome data were not observed. A natural question is then how these early data can best be used in the trial analysis. Although recommendations are available from regulators, funders, and methodologists, there is a lack of a review of recent work addressing this problem. This article reports a review of recent methods that can be used in the setting of the analysis of interrupted clinical trials with longitudinal outcomes with monotone missingness. A search for methodological papers published during the period 2020-2023 identified 43 relevant publications. We categorised these articles under the four broad themes of missing value imputation, modelling and covariate adjustment, simulation and estimands. Although motivated by the interruption due to SARS-CoV-2 and the resulting disease, the papers reviewed and methods discussed are also relevant to clinical trials interrupted for other reasons, with follow-up discontinued.</p>","PeriodicalId":22038,"journal":{"name":"Statistical Methods in Medical Research","volume":" ","pages":"2131-2143"},"PeriodicalIF":1.6000,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11577686/pdf/","citationCount":"0","resultStr":"{\"title\":\"Statistical methods for clinical trials interrupted by the severe acute respiratory syndrome-coronavirus-2 (SARS-CoV-2) pandemic: A review.\",\"authors\":\"Joydeep Basu, Nicholas Parsons, Tim Friede, Nigel Stallard\",\"doi\":\"10.1177/09622802241288350\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Cancellation or delay of non-essential medical interventions, limitation of face-to-face assessments or outpatient attendance due to lockdown restrictions, illness or fear of hospital or healthcare centre visits, and halting of research to allow diversion of healthcare resources to focus on the pandemic led to the interruption of many clinical trials during the severe acute respiratory syndrome-coronavirus-2 (SARS-CoV-2) pandemic. Appropriate analysis approaches are now required for these interrupted trials. In trials with long follow-up and longitudinal outcomes, data may be available on early outcomes for many patients for whom final, primary outcome data were not observed. A natural question is then how these early data can best be used in the trial analysis. Although recommendations are available from regulators, funders, and methodologists, there is a lack of a review of recent work addressing this problem. This article reports a review of recent methods that can be used in the setting of the analysis of interrupted clinical trials with longitudinal outcomes with monotone missingness. A search for methodological papers published during the period 2020-2023 identified 43 relevant publications. We categorised these articles under the four broad themes of missing value imputation, modelling and covariate adjustment, simulation and estimands. Although motivated by the interruption due to SARS-CoV-2 and the resulting disease, the papers reviewed and methods discussed are also relevant to clinical trials interrupted for other reasons, with follow-up discontinued.</p>\",\"PeriodicalId\":22038,\"journal\":{\"name\":\"Statistical Methods in Medical Research\",\"volume\":\" \",\"pages\":\"2131-2143\"},\"PeriodicalIF\":1.6000,\"publicationDate\":\"2024-11-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11577686/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Statistical Methods in Medical Research\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1177/09622802241288350\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2024/10/30 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/10.1177/09622802241288350","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/10/30 0:00:00","PubModel":"Epub","JCR":"Q3","JCRName":"HEALTH CARE SCIENCES & SERVICES","Score":null,"Total":0}
引用次数: 0

摘要

在严重急性呼吸系统综合症--冠状病毒 2 型(SARS-CoV-2)大流行期间,由于封锁限制、生病或害怕去医院或医疗中心就诊而取消或推迟非必要的医疗干预、限制面对面的评估或门诊就诊,以及停止研究以便将医疗资源转移到关注大流行病上,导致许多临床试验中断。现在需要对这些中断的试验采用适当的分析方法。在具有长期随访和纵向结果的试验中,可能会有许多患者的早期结果数据,而最终的主要结果数据并未被观察到。因此,一个很自然的问题就是如何在试验分析中更好地使用这些早期数据。虽然监管机构、资助者和方法论专家都提出了建议,但近期解决这一问题的工作还缺乏综述。本文综述了最近的一些方法,这些方法可用于分析具有单调缺失的纵向结果的中断临床试验。通过对 2020-2023 年间发表的方法学论文进行检索,我们发现了 43 篇相关论文。我们将这些文章归类为四大主题:缺失值估算、建模和协变量调整、模拟和估计。虽然研究的动机是由于 SARS-CoV-2 和由此引发的疾病而导致的临床试验中断,但所审查的论文和讨论的方法同样适用于因其他原因而中断的临床试验,这些临床试验的随访工作也已停止。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Statistical methods for clinical trials interrupted by the severe acute respiratory syndrome-coronavirus-2 (SARS-CoV-2) pandemic: A review.

Cancellation or delay of non-essential medical interventions, limitation of face-to-face assessments or outpatient attendance due to lockdown restrictions, illness or fear of hospital or healthcare centre visits, and halting of research to allow diversion of healthcare resources to focus on the pandemic led to the interruption of many clinical trials during the severe acute respiratory syndrome-coronavirus-2 (SARS-CoV-2) pandemic. Appropriate analysis approaches are now required for these interrupted trials. In trials with long follow-up and longitudinal outcomes, data may be available on early outcomes for many patients for whom final, primary outcome data were not observed. A natural question is then how these early data can best be used in the trial analysis. Although recommendations are available from regulators, funders, and methodologists, there is a lack of a review of recent work addressing this problem. This article reports a review of recent methods that can be used in the setting of the analysis of interrupted clinical trials with longitudinal outcomes with monotone missingness. A search for methodological papers published during the period 2020-2023 identified 43 relevant publications. We categorised these articles under the four broad themes of missing value imputation, modelling and covariate adjustment, simulation and estimands. Although motivated by the interruption due to SARS-CoV-2 and the resulting disease, the papers reviewed and methods discussed are also relevant to clinical trials interrupted for other reasons, with follow-up discontinued.

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