单例实验设计系列。

Harvard data science review Pub Date : 2022-01-01 Epub Date: 2022-09-08 DOI:10.1162/99608f92.ff9300a8
Leonard H Epstein, Jesse Dallery
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摘要

单病例实验设计(SCEDs)是一种使用实验方法研究治疗对结果影响的研究设计。分析的基本单位是单个病例--可以是个人、诊所或社区--最好在病例内和/或病例间进行效果复制。这些设计具有灵活性和成本效益,可用于治疗开发、转化研究、个性化干预以及罕见疾病和失调的研究。本文概述了单病例实验设计系列,并列举了相应的实例,包括逆转设计、多基线设计、多基线/逆转组合设计,以及将单病例设计整合到更大规模的随机对照试验(RCT)中以确定个体的最佳治疗方法。个性化 N-of-1 试验可视为 SCED 的一个子类别,与逆转设计重叠。此外,还讨论了每种设计的相关问题,包括治疗方法的比较、随机化和盲法等设计问题、设计的标准以及补充单病例实验设计目测的统计方法。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

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The Family of Single-Case Experimental Designs.

Single-case experimental designs (SCEDs) represent a family of research designs that use experimental methods to study the effects of treatments on outcomes. The fundamental unit of analysis is the single case-which can be an individual, clinic, or community-ideally with replications of effects within and/or between cases. These designs are flexible and cost-effective and can be used for treatment development, translational research, personalized interventions, and the study of rare diseases and disorders. This article provides a broad overview of the family of single-case experimental designs with corresponding examples, including reversal designs, multiple baseline designs, combined multiple baseline/reversal designs, and integration of single-case designs to identify optimal treatments for individuals into larger randomized controlled trials (RCTs). Personalized N-of-1 trials can be considered a subcategory of SCEDs that overlaps with reversal designs. Relevant issues for each type of design-including comparisons of treatments, design issues such as randomization and blinding, standards for designs, and statistical approaches to complement visual inspection of single-case experimental designs-are also discussed.

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