参数治愈模型下的单臂II期试验设计
Single-arm phase II trial design under parametric cure models.
作者信息
Wu Jianrong
机构信息
Department of Biostatistics, St. Jude Children's Research Hospital, 262 Danny Thomas Place, Memphis, 38105, TN, USA.
出版信息
Pharm Stat. 2015 May-Jun;14(3):226-32. doi: 10.1002/pst.1678. Epub 2015 Apr 1.
Abstract
The current practice of designing single-arm phase II survival trials is limited under the exponential model. Trial design under the exponential model may not be appropriate when a portion of patients are cured. There is no literature available for designing single-arm phase II trials under the parametric cure model. In this paper, a test statistic is proposed, and a sample size formula is derived for designing single-arm phase II trials under a class of parametric cure models. Extensive simulations showed that the proposed test and sample size formula perform very well under different scenarios.
摘要
在指数模型下，目前设计单臂II期生存试验的方法存在局限性。当一部分患者被治愈时，指数模型下的试验设计可能并不合适。目前尚无关于在参数治愈模型下设计单臂II期试验的文献。本文提出了一种检验统计量，并推导了在一类参数治愈模型下设计单臂II期试验的样本量公式。大量模拟结果表明，所提出的检验和样本量公式在不同情况下表现良好。
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