{"title":"聚类随机试验中复发事件的分析:PLEASANT试验案例研究。","authors":"Kelly Grant, Steven A Julious","doi":"10.1177/09622802251316972","DOIUrl":null,"url":null,"abstract":"<p><p>Recurrent events for many clinical conditions, such as asthma, can indicate poor health outcomes. Recurrent events data are often analysed using statistical methods such as Cox regression or negative binomial regression, suffering event or time information loss. This article re-analyses the preventing and lessening exacerbations of asthma in school-age children associated with a new term (PLEASANT) trial data as a case study, investigating the utility, extending recurrent events survival analysis methods to cluster randomised trials. A conditional frailty model is used, with the frailty term at the general practitioner practice level, accounting for clustering. A rare events bias adjustment is applied if few participants had recurrent events and truncation of small event risk sets is explored, to improve model accuracy. Global and event-specific estimates are presented, alongside a mean cumulative function plot to aid interpretation. The conditional frailty model global results are similar to PLEASANT results, but with greater precision (include time, recurrent events, within-participant dependence, and rare events adjustment). Event-specific results suggest an increasing risk reduction in medical appointments for the intervention group, in September-December 2013, as medical contacts increase over time. The conditional frailty model is recommended when recurrent events are a study outcome for clinical trials, including cluster randomised trials, to help explain changes in event risk over time, assisting clinical interpretation.</p>","PeriodicalId":22038,"journal":{"name":"Statistical Methods in Medical Research","volume":" ","pages":"9622802251316972"},"PeriodicalIF":1.6000,"publicationDate":"2025-05-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Analysis of recurrent events in cluster randomised trials: The PLEASANT trial case study.\",\"authors\":\"Kelly Grant, Steven A Julious\",\"doi\":\"10.1177/09622802251316972\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>Recurrent events for many clinical conditions, such as asthma, can indicate poor health outcomes. Recurrent events data are often analysed using statistical methods such as Cox regression or negative binomial regression, suffering event or time information loss. This article re-analyses the preventing and lessening exacerbations of asthma in school-age children associated with a new term (PLEASANT) trial data as a case study, investigating the utility, extending recurrent events survival analysis methods to cluster randomised trials. A conditional frailty model is used, with the frailty term at the general practitioner practice level, accounting for clustering. A rare events bias adjustment is applied if few participants had recurrent events and truncation of small event risk sets is explored, to improve model accuracy. Global and event-specific estimates are presented, alongside a mean cumulative function plot to aid interpretation. The conditional frailty model global results are similar to PLEASANT results, but with greater precision (include time, recurrent events, within-participant dependence, and rare events adjustment). Event-specific results suggest an increasing risk reduction in medical appointments for the intervention group, in September-December 2013, as medical contacts increase over time. The conditional frailty model is recommended when recurrent events are a study outcome for clinical trials, including cluster randomised trials, to help explain changes in event risk over time, assisting clinical interpretation.</p>\",\"PeriodicalId\":22038,\"journal\":{\"name\":\"Statistical Methods in Medical Research\",\"volume\":\" \",\"pages\":\"9622802251316972\"},\"PeriodicalIF\":1.6000,\"publicationDate\":\"2025-05-14\",\"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/10.1177/09622802251316972\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"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/10.1177/09622802251316972","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"HEALTH CARE SCIENCES & SERVICES","Score":null,"Total":0}
Analysis of recurrent events in cluster randomised trials: The PLEASANT trial case study.
Recurrent events for many clinical conditions, such as asthma, can indicate poor health outcomes. Recurrent events data are often analysed using statistical methods such as Cox regression or negative binomial regression, suffering event or time information loss. This article re-analyses the preventing and lessening exacerbations of asthma in school-age children associated with a new term (PLEASANT) trial data as a case study, investigating the utility, extending recurrent events survival analysis methods to cluster randomised trials. A conditional frailty model is used, with the frailty term at the general practitioner practice level, accounting for clustering. A rare events bias adjustment is applied if few participants had recurrent events and truncation of small event risk sets is explored, to improve model accuracy. Global and event-specific estimates are presented, alongside a mean cumulative function plot to aid interpretation. The conditional frailty model global results are similar to PLEASANT results, but with greater precision (include time, recurrent events, within-participant dependence, and rare events adjustment). Event-specific results suggest an increasing risk reduction in medical appointments for the intervention group, in September-December 2013, as medical contacts increase over time. The conditional frailty model is recommended when recurrent events are a study outcome for clinical trials, including cluster randomised trials, to help explain changes in event risk over time, assisting clinical interpretation.
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)