Randomized Trials With Repeatedly Measured Outcomes: Handling Irregular and Potentially Informative Assessment Times.

IF 5.2 2区 医学 Q1 PUBLIC, ENVIRONMENTAL & OCCUPATIONAL HEALTH
Eleanor M Pullenayegum, Daniel O Scharfstein
{"title":"Randomized Trials With Repeatedly Measured Outcomes: Handling Irregular and Potentially Informative Assessment Times.","authors":"Eleanor M Pullenayegum,&nbsp;Daniel O Scharfstein","doi":"10.1093/epirev/mxac010","DOIUrl":null,"url":null,"abstract":"<p><p>Randomized trials are often designed to collect outcomes at fixed points in time after randomization. In practice, the number and timing of outcome assessments can vary among participants (i.e., irregular assessment). In fact, the timing of assessments may be associated with the outcome of interest (i.e., informative assessment). For example, in a trial evaluating the effectiveness of treatments for major depressive disorder, not only did the timings of outcome assessments vary among participants but symptom scores were associated with assessment frequency. This type of informative observation requires appropriate statistical analysis. Although analytic methods have been developed, they are rarely used. In this article, we review the literature on irregular assessments with a view toward developing recommendations for analyzing trials with irregular and potentially informative assessment times. We show how the choice of analytic approach hinges on assumptions about the relationship between the assessment and outcome processes. We argue that irregular assessment should be treated with the same care as missing data, and we propose that trialists adopt strategies to minimize the extent of irregularity; describe the extent of irregularity in assessment times; make their assumptions about the relationships between assessment times and outcomes explicit; adopt analytic techniques that are appropriate to their assumptions; and assess the sensitivity of trial results to their assumptions.</p>","PeriodicalId":50510,"journal":{"name":"Epidemiologic Reviews","volume":"44 1","pages":"121-137"},"PeriodicalIF":5.2000,"publicationDate":"2022-12-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10362939/pdf/","citationCount":"6","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Epidemiologic Reviews","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1093/epirev/mxac010","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"PUBLIC, ENVIRONMENTAL & OCCUPATIONAL HEALTH","Score":null,"Total":0}
引用次数: 6

Abstract

Randomized trials are often designed to collect outcomes at fixed points in time after randomization. In practice, the number and timing of outcome assessments can vary among participants (i.e., irregular assessment). In fact, the timing of assessments may be associated with the outcome of interest (i.e., informative assessment). For example, in a trial evaluating the effectiveness of treatments for major depressive disorder, not only did the timings of outcome assessments vary among participants but symptom scores were associated with assessment frequency. This type of informative observation requires appropriate statistical analysis. Although analytic methods have been developed, they are rarely used. In this article, we review the literature on irregular assessments with a view toward developing recommendations for analyzing trials with irregular and potentially informative assessment times. We show how the choice of analytic approach hinges on assumptions about the relationship between the assessment and outcome processes. We argue that irregular assessment should be treated with the same care as missing data, and we propose that trialists adopt strategies to minimize the extent of irregularity; describe the extent of irregularity in assessment times; make their assumptions about the relationships between assessment times and outcomes explicit; adopt analytic techniques that are appropriate to their assumptions; and assess the sensitivity of trial results to their assumptions.

重复测量结果的随机试验:处理不规则和潜在的信息评估时间。
随机化试验通常被设计为在随机化后的固定时间点收集结果。在实践中,结果评估的数量和时间可能因参与者而异(即不定期评估)。事实上,评估的时间可能与感兴趣的结果(即信息性评估)有关。例如,在一项评估重度抑郁障碍治疗有效性的试验中,参与者之间不仅结果评估的时间不同,而且症状评分与评估频率相关。这种类型的信息性观察需要适当的统计分析。尽管分析方法已经发展起来,但很少使用。在这篇文章中,我们回顾了关于不规则评估的文献,以期为分析具有不规则和潜在信息评估时间的试验制定建议。我们展示了分析方法的选择如何取决于对评估和结果过程之间关系的假设。我们认为,应该像对待缺失数据一样谨慎对待不规则评估,我们建议试验人员采取策略,最大限度地减少不规则的程度;描述评估时间的不规则程度;明确他们对评估时间和结果之间关系的假设;采用适合其假设的分析技术;并评估试验结果对其假设的敏感性。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
求助全文
约1分钟内获得全文 求助全文
来源期刊
Epidemiologic Reviews
Epidemiologic Reviews 医学-公共卫生、环境卫生与职业卫生
CiteScore
8.10
自引率
0.00%
发文量
10
期刊介绍: Epidemiologic Reviews is a leading review journal in public health. Published once a year, issues collect review articles on a particular subject. Recent issues have focused on The Obesity Epidemic, Epidemiologic Research on Health Disparities, and Epidemiologic Approaches to Global Health.
×
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