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Optimal designs for active controlled dose-finding trials with efficacy-toxicity outcomes
Biometrika ( IF 2.7 ) Pub Date : 2017-10-09 , DOI: 10.1093/biomet/asx057
K Schorning 1 , H Dette 1 , K Kettelhake 1 , W K Wong 2 , F Bretz 3
Affiliation  

Summary We derive optimal designs to estimate efficacy and toxicity in active controlled dose‐finding trials when the bivariate continuous outcomes are described using nonlinear regression models. We determine upper bounds on the required number of different doses and provide conditions under which the boundary points of the design space are included in the optimal design. We provide an analytical description of minimally supported optimal designs and show that they do not depend on the correlation between the bivariate outcomes.

中文翻译:

具有疗效-毒性结果的主动对照剂量探索试验的最佳设计

总结 当使用非线性回归模型描述双变量连续结果时,我们推导出最佳设计来估计主动控制剂量发现试验中的疗效和毒性。我们确定所需不同剂量数量的上限,并提供设计空间的边界点包含在最佳设计中的条件。我们提供了对最低支持最优设计的分析描述,并表明它们不依赖于双变量结果之间的相关性。
更新日期:2017-10-09
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