Langner Paula R, Juarez-Colunga Elizabeth, Marzec Lucas N, Grunwald Gary K, Rice John D
Denver/Seattle Center of Innovation, Department of Veterans Affairs Eastern Colorado Health Care System, 1700 North Wheeling Street, Aurora, 80045, CO, USA.
Department of Biostatistics and Informatics, Colorado School of Public Health, University of Colorado Anschutz Medical Campus, 13001 East 17th Place, Aurora, 80045, CO, USA.
Stat Biosci. 2025 Jan 14. doi: 10.1007/s12561-025-09473-w.
In studies with a recurrent event outcome, events may be captured as counts during subsequent intervals or follow-up times either by design or for ease of analysis. In many cases, recurrent events may be further coarsened such that only an indicator of one or more events in an interval is observed at the follow-up time, resulting in a loss of information relative to a record of all events. In this paper, we examine efficiency loss when coarsening longitudinally observed counts to binary indicators and aspects of the design which impact the ability to estimate a treatment effect of interest. The investigation was motivated by a study of patients with cardiac implantable electronic devices in which investigators aimed to examine the effect of a treatment on events detected by the devices over time. In order to study components of such a recurrent event process impacted by data coarsening, we derive the asymptotic relative efficiency (ARE) of a treatment effect estimator utilizing a coarsened binary outcome relative to an alternative estimator using the count outcome. We compare the efficiencies and consider conditions where the binary process maintains good efficiency in estimating a treatment effect. We present an application of the methods to a data set consisting of seizure counts in a sample of patients with epilepsy.
在具有复发事件结局的研究中，事件可以在后续时间段或随访期内作为计数被记录下来，这要么是出于设计目的，要么是为了便于分析。在许多情况下，复发事件可能会被进一步粗化，以至于在随访时仅观察到一个时间段内一个或多个事件的指标，这相对于所有事件的记录而言会导致信息丢失。在本文中，我们研究了将纵向观察到的计数粗化为二元指标时的效率损失，以及影响估计感兴趣的治疗效果能力的设计方面。这项研究的动机来自于一项针对心脏植入式电子设备患者的研究，在该研究中，研究人员旨在研究一种治疗方法对设备随时间检测到的事件的影响。为了研究受数据粗化影响的此类复发事件过程的组成部分，我们推导了使用粗化二元结局的治疗效果估计器相对于使用计数结局的替代估计器的渐近相对效率（ARE）。我们比较了效率，并考虑了二元过程在估计治疗效果时保持良好效率的条件。我们将这些方法应用于一个由癫痫患者样本中的癫痫发作计数组成的数据集。
