精确医学统计方法手册
The statistical study and development of analytic methodology for individualization of treatments is no longer in its infancy. Many methods of study design, estimation, and inference exist, and the tools available to the analyst are ever growing. This handbook introduces the foundations of modern statistical approaches to precision medicine, bridging key ideas to active lines of current research in precision medicine. The contributions in this handbook vary in their level of assumed statistical knowledge; all contributions are accessible to a wide readership of statisticians and computer scientists including graduate students and new researchers in the area. Many contributions, particularly those that are more comprehensive reviews, are suitable for epidemiologists and clinical researchers with some statistical training. The handbook is split into three sections: Study Design for Precision Medicine, Estimation of Optimal Treatment Strategies, and Precision Medicine in High Dimensions. The first focuses on designed experiments, in many instances, building and extending on the notion of sequential multiple assignment randomized trials. Dose finding and simulation-based designs using agent-based modelling are also featured. The second section contains both introductory contributions and more advanced methods, suitable for estimating optimal adaptive treatment strategies from a variety of data sources including non-experimental (observational) studies. The final section turns to estimation in the many-covariate setting, providing approaches suitable to the challenges posed by electronic health records, wearable devices, or any other settings where the number of possible variables (whether confounders, tailoring variables, or other) is high. Together, these three sections bring together some of the foremost leaders in the field of precision medicine, offering new insights and ideas as this field moves towards its third decade.
统计研究和分析方法以实现治疗个性化的发展已经不再处于初级阶段。存在许多的研究设计、估计和推断的方法,分析师可用的工具也在不断增长。这本手册介绍了现代统计方法在精准医疗中的基础,将关键理念与当前精准医学研究的主要方向联系起来。这本手册的内容在所假设的统计知识水平方面有所不同;所有贡献都是面向广泛的统计数据学家和计算机科学家群体,包括该领域的研究生和新研究人员。许多贡献,特别是那些更综合的综述性文章,适合具有一定统计训练的流行病学专家和临床研究者。手册分为三个部分:精准医疗的研究设计、最优治疗策略的估计方法以及高维精准医学。第一部分专注于设计实验,在许多情况下,利用顺序多次分配随机试验的概念进行构建和扩展。还涵盖了基于代理建模的方法来实现剂量确定和模拟设计。第二部分包含初学者贡献和更先进的方法,适合从各种数据源(包括非实验性的观察性研究)中估计多种最优自适应治疗策略。最后一部分转向多协变量设置中的估计问题,提供了适用于电子健康记录、可穿戴设备或其他具有高变量数目的环境的挑战的方法。这三个部分汇集了一些精准医疗领域的最前沿领导者的新见解和想法,随着这一领域迈向其第三个十年。
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