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一种基于Fine-Gray模型的治疗效果组序贯检验。

A group sequential test for treatment effect based on the Fine-Gray model.

作者信息

Martens Michael J, Logan Brent R

机构信息

The Emmes Corporation, Rockville, Maryland, U.S.A.

Division of Biostatistics, Medical College of Wisconsin, Milwaukee, Wisconsin, U.S.A.

出版信息

Biometrics. 2018 Sep;74(3):1006-1013. doi: 10.1111/biom.12871. Epub 2018 Mar 13.

Abstract

Competing risks endpoints arise when patients can fail therapy from several causes. Analyzing these outcomes allows one to assess directly the benefit of treatment on a primary cause of failure in a clinical trial setting. Regression models can be used in clinical trials to adjust for residual imbalances in patient characteristics, improving the power to detect treatment differences. But, none of the competing risks methods currently available for use in group sequential trials adjust for covariates. We propose a group sequential test for treatment effect that, because it is based on the Fine-Gray model, permits adjustment for covariates. Our derivations show that its sequence of test statistics has an asymptotic distribution with an independent increments structure, which allows standard techniques such as O'Brien-Fleming designs and error spending functions to be employed to meet type I error rate and power specifications. We demonstrate the test in a reanalysis of BMT CTN 0402, a phase III clinical trial that evaluated an experimental treatment for the prevention of adverse outcomes following blood and marrow transplant. Moreover, using a simulation study of randomized group sequential trials, we demonstrate that the proposed method preserves the type I error rate and power at their nominal levels in the presence of influential covariates.

摘要

当患者可能因多种原因治疗失败时,就会出现竞争风险终点。对这些结果进行分析可使人们在临床试验环境中直接评估治疗对主要失败原因的益处。回归模型可用于临床试验,以调整患者特征方面的残余不平衡,提高检测治疗差异的效能。但是,目前可用于成组序贯试验的竞争风险方法均未对协变量进行调整。我们提出了一种用于治疗效果的成组序贯检验,由于它基于Fine-Gray模型,因此允许对协变量进行调整。我们的推导表明,其检验统计量序列具有独立增量结构的渐近分布,这使得可以采用诸如O'Brien-Fleming设计和误差消耗函数等标准技术来满足I型错误率和效能规范。我们在对BMT CTN 0402的重新分析中展示了该检验,BMT CTN 0402是一项III期临床试验,评估了一种预防血液和骨髓移植后不良结局的实验性治疗方法。此外,通过对随机成组序贯试验的模拟研究,我们证明了所提出的方法在存在有影响的协变量时,能将I型错误率和效能维持在其名义水平。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/1708/6146968/ec85b75f97ba/nihms962843f1.jpg

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