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n-of-1临床试验:个体化医疗的终极策略?

The n-of-1 clinical trial: the ultimate strategy for individualizing medicine?

作者信息

Lillie Elizabeth O, Patay Bradley, Diamant Joel, Issell Brian, Topol Eric J, Schork Nicholas J

机构信息

Scripps Health, La Jolla, CA 92037, USA.

出版信息

Per Med. 2011 Mar;8(2):161-173. doi: 10.2217/pme.11.7.

Abstract

N-of-1 or single subject clinical trials consider an individual patient as the sole unit of observation in a study investigating the efficacy or side-effect profiles of different interventions. The ultimate goal of an n-of-1 trial is to determine the optimal or best intervention for an individual patient using objective data-driven criteria. Such trials can leverage study design and statistical techniques associated with standard population-based clinical trials, including randomization, washout and crossover periods, as well as placebo controls. Despite their obvious appeal and wide use in educational settings, n-of-1 trials have been used sparingly in medical and general clinical settings. We briefly review the history, motivation and design of n-of-1 trials and emphasize the great utility of modern wireless medical monitoring devices in their execution. We ultimately argue that n-of-1 trials demand serious attention among the health research and clinical care communities given the contemporary focus on individualized medicine.

摘要

单病例临床试验将个体患者视为研究不同干预措施疗效或副作用特征时的唯一观察单位。单病例试验的最终目标是使用客观的数据驱动标准为个体患者确定最佳干预措施。此类试验可利用与基于标准人群的临床试验相关的研究设计和统计技术,包括随机化、洗脱期和交叉期,以及安慰剂对照。尽管单病例试验在教育环境中具有明显的吸引力且应用广泛,但在医学和一般临床环境中使用较少。我们简要回顾单病例试验的历史、动机和设计,并强调现代无线医疗监测设备在其实施过程中的巨大作用。鉴于当代对个性化医疗的关注,我们最终认为单病例试验应引起健康研究和临床护理界的高度重视。

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