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一种在具有删失生存数据的序贯临床试验中利用信息分数的有用设计。

A useful design utilizing the information fraction in a group sequential clinical trial with censored survival data.

作者信息

Hsu Chih-Yuan, Chen Chen-Hsin, Hsu Ken-Ning, Lu Ya-Hung

机构信息

Institute of Statistical Science, Academia Sinica, Taipei 11529, Taiwan, Republic of China.

Graduate Institute of Epidemiology and Preventive Medicine, National Taiwan University, Taipei 10055, Taiwan, Republic of China.

出版信息

Biometrics. 2019 Mar;75(1):133-143. doi: 10.1111/biom.12925. Epub 2018 Jul 13.

Abstract

Lan and DeMets (1983) proposed the alpha spending function for group sequential trials to permit the use of unspecified frequencies and timings of interim analyses in the trial design. Regarding a trial with censored time to endpoint, Lan and DeMets (1989) later defined information time at an interim analysis in a maximum duration trial. To compare two survival curves utilizing such a design, information times for group sequential logrank and Wilcoxon-type statistics have been developed by assuming that the survival time follows an exponential distribution or a Weibull distribution without considering the censoring distribution. To better address the practical concerns inherent in clinical trials with survival endpoints, we present a new approach to adequately design a group sequential trial using the Harrington-Fleming (1982) test based on our proposed information fractions by assuming the censoring distribution depends on the patient's accrual time according to various entry distributions and by extending the underlying survival distribution to the generalized gamma distribution. We also determine associated sample sizes, expected number of events and expected stopping time. Two phase III trials of non-small-cell lung cancer originally designed using fixed-sample tests are utilized to illustrate the potential advantages of using a group sequential design with the proposed approach. This enhanced method facilitates the design and analysis of group sequential clinical trials studying survival endpoints by increasing implemental flexibility.

摘要

兰和德梅茨(1983年)提出了用于成组序贯试验的α花费函数,以便在试验设计中允许使用未指定的中期分析频率和时间安排。对于终点时间存在删失的试验,兰和德梅茨(1989年)后来在最大持续时间试验中定义了中期分析时的信息时间。为了使用这样的设计比较两条生存曲线,通过假设生存时间服从指数分布或威布尔分布而不考虑删失分布,已经开发了成组序贯对数秩检验和威尔科克森型统计量的信息时间。为了更好地解决具有生存终点的临床试验中固有的实际问题,我们提出一种新方法,通过基于我们提出的信息分数,使用哈林顿 - 弗莱明(1982年)检验来充分设计成组序贯试验,假设删失分布根据各种入组分布取决于患者的入组时间,并将基础生存分布扩展到广义伽马分布。我们还确定了相关的样本量、预期事件数和预期停止时间。利用最初使用固定样本检验设计的两项非小细胞肺癌III期试验来说明使用所提出方法的成组序贯设计的潜在优势。这种改进的方法通过增加实施灵活性,促进了研究生存终点的成组序贯临床试验的设计和分析。

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