Streja Elani, Goldstein Leanne, Soohoo Melissa, Obi Yoshitsugu, Kalantar-Zadeh Kamyar, Rhee Connie M
Harold Simmons Center for Chronic Disease Research and Epidemiology, Division of Nephrology and Hypertension, University of California Irvine School of Medicine, Irvine, CA, USA.
Veterans Affairs Long Beach Healthcare System, Long Beach, CA, USA.
Nephrol Dial Transplant. 2017 Apr 1;32(suppl_2):ii77-ii83. doi: 10.1093/ndt/gfx015.
Nephrologists and kidney disease researchers are often interested in monitoring how patients' clinical and laboratory measures change over time, what factors may impact these changes, and how these changes may lead to differences in morbidity, mortality, and other outcomes. When longitudinal data with repeated measures over time in the same patients are available, there are a number of analytical approaches that could be employed to describe the trends and changes in these measures, and to explore the associations of these changes with outcomes. Researchers may choose a streamlined and simplified analytic approach to examine trajectories with subsequent outcomes such as estimating deltas (subtraction of the last observation from the first observation) or estimating per patient slopes with linear regression. Conversely, they could more fully address the data complexity by using a longitudinal mixed model to estimate change as a predictor or employ a joint model, which can simultaneously model the longitudinal effect and its impact on an outcome such as survival. In this review, we aim to assist nephrologists and clinical researchers by reviewing these approaches in modeling the association of longitudinal change in a marker with outcomes, while appropriately considering the data complexity. Namely, we will discuss the use of simplified approaches for creating predictor variables representing change in measurements including deltas and patient slopes, as well more sophisticated longitudinal models including joint models, which can be used in addition to simplified models based on the indications and objectives of the study as warranted.
肾病学家和肾脏疾病研究人员常常关注监测患者的临床和实验室指标如何随时间变化,哪些因素可能影响这些变化,以及这些变化如何导致发病率、死亡率和其他结局的差异。当有同一患者随时间重复测量的纵向数据时,可以采用多种分析方法来描述这些指标的趋势和变化,并探讨这些变化与结局之间的关联。研究人员可以选择一种简化的分析方法来检查轨迹及其后续结局,例如估计差值(首次观察值减去末次观察值)或通过线性回归估计每位患者的斜率。相反,他们可以使用纵向混合模型将变化作为预测因子进行估计,或者采用联合模型,该模型可以同时对纵向效应及其对生存等结局的影响进行建模,从而更全面地处理数据的复杂性。在本综述中,我们旨在通过回顾这些用于对标志物纵向变化与结局之间的关联进行建模的方法,同时适当考虑数据的复杂性,来帮助肾病学家和临床研究人员。具体而言,我们将讨论使用简化方法创建代表测量值变化的预测变量,包括差值和患者斜率,以及更复杂的纵向模型,如联合模型,这些模型可根据研究的指征和目标,在必要时与基于简化模型一起使用。