Department of Biostatistics, University of Michigan, Ann Arbor, Michigan.
Vertex Pharmaceuticals, Boston, Massachusetts.
Stat Med. 2019 Nov 20;38(26):5133-5145. doi: 10.1002/sim.8356. Epub 2019 Sep 9.
Restricted mean survival time (RMST) has gained increased attention in biostatistical and clinical studies. Directly modeling RMST (as opposed to modeling then transforming the hazard function) is appealing computationally and in terms of interpreting covariate effects. We propose computationally convenient methods for evaluating center effects based on RMST. A multiplicative model for the RMST is assumed. Estimation proceeds through an algorithm analogous to stratification, which permits the evaluation of thousands of centers. We derive the asymptotic properties of the proposed estimators and evaluate finite sample performance through simulation. We demonstrate that considerable decreases in computational burden are achievable through the proposed methods, in terms of both storage requirements and run time. The methods are applied to evaluate more than 5000 US dialysis facilities using data from a national end-stage renal disease registry.
限制平均生存时间(RMST)在生物统计学和临床研究中受到了越来越多的关注。直接对 RMST 进行建模(而不是对危险函数进行建模然后转换)在计算和解释协变量效应方面具有吸引力。我们提出了基于 RMST 评估中心效应的计算方便的方法。假设 RMST 的乘法模型。估计通过类似于分层的算法进行,这允许评估数千个中心。我们推导出了所提出的估计量的渐近性质,并通过模拟评估了有限样本性能。我们证明,通过所提出的方法,可以在存储需求和运行时间方面实现计算负担的显著降低。该方法应用于使用全国终末期肾脏疾病登记处的数据评估超过 5000 个美国透析设施。