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整群随机试验中复发事件的分析:PLEASANT试验案例研究。

Analysis of recurrent events in cluster randomised trials: The PLEASANT trial case study.

作者信息

Grant Kelly, Julious Steven A

机构信息

Norwich Clinical Trials Unit, The University of East Anglia, Norwich, Norfolk, UK.

The University of Sheffield, Sheffield, UK.

出版信息

Stat Methods Med Res. 2025 Jun;34(6):1079-1096. doi: 10.1177/09622802251316972. Epub 2025 May 14.

Abstract

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.

摘要

许多临床病症(如哮喘)的复发事件可能预示着不良的健康结局。复发事件数据通常采用Cox回归或负二项回归等统计方法进行分析,但会出现事件或时间信息丢失的情况。本文以一项关于预防和减轻学龄儿童哮喘加重的新试验(PLEASANT)数据为例进行重新分析,研究将复发事件生存分析方法扩展到整群随机试验的效用。使用了一个条件脆弱模型,脆弱项设定在全科医生诊疗水平,以考虑聚类情况。如果很少有参与者出现复发事件,则应用罕见事件偏差调整,并探讨对小事件风险集的截断,以提高模型准确性。给出了总体估计和特定事件估计,以及一个平均累积函数图以辅助解读。条件脆弱模型的总体结果与PLEASANT试验结果相似,但精度更高(包括时间、复发事件、参与者内相关性和罕见事件调整)。特定事件结果表明,随着2013年9月至12月医疗接触次数随时间增加,干预组医疗预约的风险降低幅度不断增大。当复发事件是临床试验(包括整群随机试验)的研究结局时,建议使用条件脆弱模型,以帮助解释事件风险随时间的变化,辅助临床解读。

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本文引用的文献

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