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动态治疗策略与序贯多重分配随机化简介。

Introduction to dynamic treatment strategies and sequential multiple assignment randomization.

作者信息

Lavori Philip W, Dawson Ree

机构信息

Department of Health Research and Policy, School of Medicine, Stanford University, Stanford, CA, USA

Frontier Science, Boston, MA, USA.

出版信息

Clin Trials. 2014 Aug;11(4):393-399. doi: 10.1177/1740774514527651. Epub 2014 May 1.

DOI:10.1177/1740774514527651
PMID:24784487
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC4216645/
Abstract

BACKGROUND

In June 2013, a 1-day workshop on Dynamic Treatment Strategies (DTSs) and Sequential Multiple Assignment Randomized Trials (SMARTs) was held at the University of Pennsylvania in Philadelphia, Pennsylvania. These two linked topics have generated a great deal of interest as researchers have recognized the importance of comparing entire strategies for managing chronic disease. A number of articles emerged from that workshop.

PURPOSE

The purpose of this survey of the DTS/SMART methodology (which is taken from the introductory talk in the workshop) is to provide the reader the collected articles presented in this volume with sufficient background to appreciate the more detailed discussions in the articles.

METHODS

The way that the DTS arises naturally in clinical practice is described, along with its connection to the well-known difficulties of interpreting the analysis by intention-to-treat. The SMART methodology for comparing DTS is described, and the basics of estimation and inference presented.

RESULTS

The DTS/SMART methodology can be a flexible and practical way to optimize ongoing clinical decision making, providing evidence (based on randomization) for comparative effectiveness.

LIMITATIONS

The DTS/SMART methodology is not a solution for unstandardized study protocols.

CONCLUSIONS

The DTS/SMART methodology has growing relevance to comparative effectiveness research and the needs of the learning healthcare system.

摘要

背景

2013年6月,一场关于动态治疗策略(DTS)和序贯多重分配随机试验(SMART)的为期一天的研讨会在宾夕法尼亚州费城的宾夕法尼亚大学举行。随着研究人员认识到比较慢性病管理的整体策略的重要性,这两个相关主题引发了极大的兴趣。该研讨会产生了多篇文章。

目的

本次对DTS/SMART方法的综述(取材于研讨会上的介绍性演讲)旨在为读者提供本卷中所收录文章的充分背景知识,以便理解文章中更详细的讨论。

方法

描述了DTS在临床实践中自然产生的方式,以及它与按意向性治疗解释分析时所面临的众所周知的困难之间的联系。描述了用于比较DTS的SMART方法,并介绍了估计和推断的基础知识。

结果

DTS/SMART方法可以是优化正在进行的临床决策的一种灵活且实用的方式,为比较疗效提供(基于随机化的)证据。

局限性

DTS/SMART方法并非未标准化研究方案的解决方案。

结论

DTS/SMART方法与比较疗效研究以及学习型医疗系统的需求越来越相关。

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