Peng Y, Dear K B, Denham J W
Department of Statistics, University of Newcastle, NSW, Australia.
Stat Med. 1998 Apr 30;17(8):813-30. doi: 10.1002/(sici)1097-0258(19980430)17:8<813::aid-sim775>3.0.co;2-#.
Cure rate estimation is an important issue in clinical trials for diseases such as lymphoma and breast cancer and mixture models are the main statistical methods. In the last decade, mixture models under different distributions, such as exponential, Weibull, log-normal and Gompertz, have been discussed and used. However, these models involve stronger distributional assumptions than is desirable and inferences may not be robust to departures from these assumptions. In this paper, a mixture model is proposed using the generalized F distribution family. Although this family is seldom used because of computational difficulties, it has the advantage of being very flexible and including many commonly used distributions as special cases. The generalised F mixture model can relax the usual stronger distributional assumptions and allow the analyst to uncover structure in the data that might otherwise have been missed. This is illustrated by fitting the model to data from large-scale clinical trials with long follow-up of lymphoma patients. Computational problems with the model and model selection methods are discussed. Comparison of maximum likelihood estimates with those obtained from mixture models under other distributions are included.
治愈率估计是淋巴瘤和乳腺癌等疾病临床试验中的一个重要问题,混合模型是主要的统计方法。在过去十年中,已经讨论并使用了不同分布下的混合模型,如指数分布、威布尔分布、对数正态分布和冈珀茨分布。然而,这些模型涉及的分布假设比理想情况更强,并且推断可能对偏离这些假设的情况不稳健。在本文中,提出了一种使用广义F分布族的混合模型。尽管由于计算困难,这个族很少被使用,但它具有非常灵活的优点,并且包括许多常用分布作为特殊情况。广义F混合模型可以放宽通常更强的分布假设,并允许分析师发现数据中可能被遗漏的结构。通过将该模型拟合到对淋巴瘤患者进行长期随访的大规模临床试验数据中,对此进行了说明。讨论了该模型的计算问题和模型选择方法。还包括将最大似然估计与从其他分布下的混合模型获得的估计进行比较。