Single-patient (n-of-1) trials: a pragmatic clinical decision methodology for patient-centered comparative effectiveness research.
Single-patient (n-of-1) trials: a pragmatic clinical decision methodology for patient-centered comparative effectiveness research.
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DOI：
10.1016/j.jclinepi.2013.04.006
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发表时间：
2013-08
影响因子：
7.2
通讯作者：
Schmid, Christopher H.
中科院分区：
文献类型：
--
作者：
Duan, Naihua;Kravitz, Richard L.;Schmid, Christopher H.
关键词：
提高临床医生和流行病学家的认识，即单患者（n-1）试验可能有助于为慢性病患者提供个性化治疗决策。我们回顾了关于单患者试验方法和应用的临床和统计文献，然后批判性地评估了进一步方法学发展的需求。现有文献报告了108项研究中2，154项单患者试验在不同临床条件下的应用;最近的各种评论主张在临床决策中更广泛地应用此类试验。最近几项试点可接受性研究的初步证据表明，单患者试验有可能被患者和临床医生广泛接受，作为提高治疗精度的有效方式。贝叶斯和自适应统计方法有望提高单患者试验的信息产量，同时减少参与者的负担，但没有得到广泛应用。通过进一步开发和应用适应性试验设计、停止规则、网络荟萃分析、洗脱方法以及将试验结果传达给患者和临床医生的方法，可以增强单个患者试验的个性化应用。单患者试验可能会成为比较有效性研究和以患者为中心的结局研究的重要方法学组成部分。通过允许直接估计个体治疗效果，它们可以促进精细分级的个体化护理，提高治疗精度，改善患者结局并降低成本。
To raise awareness among clinicians and epidemiologists that single-patient (n-of-1) trials are potentially useful for informing personalized treatment decisions for patients with chronic conditions. We reviewed the clinical and statistical literature on methods and applications of single-patient trials and then critically evaluated the needs for further methodological developments. Existing literature reports application of 2,154 single-patient trials in 108 studies for diverse clinical conditions; various recent commentaries advocate for wider application of such trials in clinical decision making. Preliminary evidence from several recent pilot acceptability studies suggests that single-patient trials have the potential for widespread acceptance by patients and clinicians as an effective modality for increasing the therapeutic precision. Bayesian and adaptive statistical methods hold promise for increasing the informational yield of single-patient trials while reducing participant burden, but are not widely used. Personalized applications of single-patient trials can be enhanced through further development and application of methodologies on adaptive trial design, stopping rules, network meta-analysis, washout methods, and methods for communicating trial findings to patients and clinicians. Single-patient trials may be poised to emerge as an important part of the methodological armamentarium for comparative effectiveness research and patient-centered outcomes research. By permitting direct estimation of individual treatment effects, they can facilitate finely graded individualized care, enhance therapeutic precision, improve patient outcomes, and reduce costs.
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影响因子：
2.5
作者：
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通讯作者：
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