Lim Lily Siok Hoon, Feldman Brian M, Lix Lisa M
Department of Pediatrics, Rady Faculty of Health Sciences, University of Manitoba, 501F-715 McDermot Avenue, Winnipeg, Manitoba R3E 3P4, Canada.
Division of Rheumatology, The Hospital for Sick Children, 555 University Avenue, Toronto, Ontario M5G1X8, Canada; Department of Pediatrics, Faculty of Medicine, Institute of Health Policy Management and Evaluation, Dana Lana School of Public Health, University of Toronto, Toronto, Ontario M5T 3M6, Canada.
Rheum Dis Clin North Am. 2018 May;44(2):189-201. doi: 10.1016/j.rdc.2018.01.001. Epub 2018 Feb 21.
Longitudinal cohort designs (with three or more measurement occasions) are invaluable to investigate between- and within-individual variation in outcomes. However, traditional longitudinal designs require a lengthy implementation and data collection period and impose a substantial burden on participants and investigators. We discuss alternative longitudinal designs, including planned missing data designs and retrospective cohort studies with secondary data, which require a shorter period for data accrual and reduce participant burden while maintaining statistical power. We also discuss analysis strategies to maximize data use and produce unbiased estimates of treatment effectiveness, including models for recurrent or multistate events and time-varying covariates.
纵向队列设计(有三次或更多次测量时机)对于研究个体间和个体内结果的差异非常重要。然而,传统的纵向设计需要很长的实施和数据收集期,给参与者和研究者带来很大负担。我们讨论了替代纵向设计,包括计划缺失数据设计和使用二次数据的回顾性队列研究,这些设计需要较短的数据积累期,在保持统计效力的同时减轻参与者负担。我们还讨论了最大化数据利用并产生无偏治疗效果估计的分析策略,包括针对复发或多状态事件以及时变协变量的模型。