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[单病例试验:独一无二]

[N-of-1 trials: the one and only].

作者信息

Wolters Thalijn L C, Van Vlijmen J

机构信息

Radboudumc, afd. Algemene Interne Geneeskunde, Nijmegen.

Contact: Thalijn L.C. Wolters (

出版信息

Ned Tijdschr Geneeskd. 2021 Sep 16;165:D6149.

PMID:34854642
Abstract

Personalized care is a topical issue: the demand for an optimal, individualized treatment instead of uniformity and strict guidelines in increasing. Implementing these ideals in clinical practice often appears to be challenging, as a result of heterogeneity in treatment effects and the influence of patient-specific factors. N-of-1 trials are double-blind, randomized, placebo-controlled trials in an individual patient and can be used to objectively examine the (side)effects of a specific drug, and to compare two or more drugs or variants of the same drug. The results can be used when aiming of deprescribing, but also for evaluating patient-reported intolerances or (in)effectiveness. This article discusses the use and advantages of N-of-1-trials in clinical practice, and addresses its specific concerns and possibilities. The N-of-1-trial is an effective, cost-reducing method to evaluate the effects of a certain drug, which is accessible to nearly every physician and can contribute to safe and effective personalized medicine.

摘要

个性化医疗是一个热门话题

对优化的个体化治疗而非统一和严格指南的需求正在增加。由于治疗效果的异质性和患者特定因素的影响,在临床实践中实施这些理想方案往往具有挑战性。单病例试验是针对个体患者的双盲、随机、安慰剂对照试验,可用于客观检验特定药物的(副)作用,并比较两种或更多种药物或同一药物的不同变体。这些结果可用于减药时的决策,也可用于评估患者报告的不耐受情况或(无)效性。本文讨论了单病例试验在临床实践中的应用和优势,并探讨了其具体问题和可能性。单病例试验是一种评估特定药物效果的有效且成本降低的方法,几乎每位医生都可采用,有助于实现安全有效的个性化医疗。

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