Russek-Cohen E, Simon R M
Department of Animal Sciences, University of Maryland, College Park 20742, USA.
Stat Med. 1994 Jan 15;13(1):87-95. doi: 10.1002/sim.4780130110.
We propose a method for selecting the best treatment when a monotonic dose-response relationship exists. Because of side effects associated with higher doses, the highest dose may not be the optimum, particularly when a lower dose gives a similar response. Rather than assume a particular functional relationship of dose to response, we use isotonic regression techniques. We consider the case of three treatment levels, which is applicable to many clinical trials. The lowest treatment level may represent a placebo or no treatment control. While we focus primarily on Bernoulli response variables, we also discuss a model for normally distributed data. We suggest a two-stage procedure that we have investigated via simulation.
我们提出了一种在存在单调剂量反应关系时选择最佳治疗方法的方法。由于高剂量会带来副作用,最高剂量可能并非最佳选择,尤其是当较低剂量能产生类似反应时。我们不假设剂量与反应之间存在特定的函数关系,而是使用等渗回归技术。我们考虑了三个治疗水平的情况,这适用于许多临床试验。最低治疗水平可能代表安慰剂或无治疗对照。虽然我们主要关注伯努利反应变量,但我们也讨论了一个适用于正态分布数据的模型。我们建议了一个两阶段程序,并通过模拟对其进行了研究。