对复发和死亡事件进行限制性平均时间分析的研究设计。

IF 1.4 4区 数学 Q3 BIOLOGY Biometrics Pub Date : 2023-08-23 DOI:10.1111/biom.13923
Lu Mao
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引用次数: 0

摘要

复发事件和死亡的复合终点的分析工具箱中刚刚增加了治疗的受限平均支持时间(RMT-IF)。为了帮助从业人员根据这种方法设计新的试验,我们开发了计算样本量和功率的工具。具体来说,我们将结果表述为一个多状态马尔可夫过程,其中复发事件为一系列瞬态,死亡为一个吸收态。过渡强度(在本例中为另一个非致命事件或死亡的瞬时风险)被假定为时间均质的,但仍允许取决于过去事件的数量。利用 Coxian 分布的特性,我们得出了替代假设下的 RMT-IF 效果大小,它是治疗与控制强度比以及基线强度的函数,后者可以很容易地从历史数据中估算出来。我们还将非参数 RMT-IF 估计器的方差简化为标准删减设置下的可计算项。模拟研究表明,由此得出的公式能在现实环境中提供样本大小和功率的精确近似值。举例说明,我们分析了过去一项心血管试验的复发性住院和死亡率结果,以生成设计未来试验所需的参数。这些程序与原始方法一起被纳入了 R 档案综合网络(CRAN)上的 rmt 软件包。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

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Study design for restricted mean time analysis of recurrent events and death

The restricted mean time in favor (RMT-IF) of treatment has just been added to the analytic toolbox for composite endpoints of recurrent events and death. To help practitioners design new trials based on this method, we develop tools to calculate the sample size and power. Specifically, we formulate the outcomes as a multistate Markov process with a sequence of transient states for recurrent events and an absorbing state for death. The transition intensities, in this case the instantaneous risks of another nonfatal event or death, are assumed to be time-homogeneous but nonetheless allowed to depend on the number of past events. Using the properties of Coxian distributions, we derive the RMT-IF effect size under the alternative hypothesis as a function of the treatment-to-control intensity ratios along with the baseline intensities, the latter of which can be easily estimated from historical data. We also reduce the variance of the nonparametric RMT-IF estimator to calculable terms under a standard set-up for censoring. Simulation studies show that the resulting formulas provide accurate approximation to the sample size and power in realistic settings. For illustration, a past cardiovascular trial with recurrent-hospitalization and mortality outcomes is analyzed to generate the parameters needed to design a future trial. The procedures are incorporated into the rmt package along with the original methodology on the Comprehensive R Archive Network (CRAN).

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来源期刊
Biometrics
Biometrics 生物-生物学
CiteScore
2.70
自引率
5.30%
发文量
178
审稿时长
4-8 weeks
期刊介绍: The International Biometric Society is an international society promoting the development and application of statistical and mathematical theory and methods in the biosciences, including agriculture, biomedical science and public health, ecology, environmental sciences, forestry, and allied disciplines. The Society welcomes as members statisticians, mathematicians, biological scientists, and others devoted to interdisciplinary efforts in advancing the collection and interpretation of information in the biosciences. The Society sponsors the biennial International Biometric Conference, held in sites throughout the world; through its National Groups and Regions, it also Society sponsors regional and local meetings.
期刊最新文献
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