单病例试验：个性化医疗的典范？
N-of-1 trials: The epitome of personalized medicine?
作者信息
Samuel Joyce P, Wootton Susan H, Tyson Jon E
机构信息
Department of Pediatrics, Center for Clinical Research and Evidence-Based Medicine, McGovern Medical School, The University of Texas Health Science Center at Houston, Houston, TX, USA.
出版信息
J Clin Transl Sci. 2023 Jul 3;7(1):e161. doi: 10.1017/cts.2023.583. eCollection 2023.
Abstract
Observational studies are notoriously susceptible to bias, and parallel-group randomized trials are important to identify the best overall treatment for eligible patients. Yet, such trials can be expected to be a misleading indicator of the best treatment for some subgroups or individual patients. In selected circumstances, patients can be treated in n-of-1 trials to address the inherent heterogeneity of treatment response in clinical populations. Such trials help to accomplish the ultimate goal of all biomedical research, to optimize the care of individual patients.
摘要
观察性研究极易受到偏倚影响，平行组随机试验对于确定适合患者的最佳总体治疗方法很重要。然而，对于某些亚组或个体患者而言，此类试验可能会成为最佳治疗方法的误导性指标。在特定情况下，可以采用单病例试验对患者进行治疗，以解决临床人群中治疗反应固有的异质性问题。此类试验有助于实现所有生物医学研究的最终目标，即优化个体患者的护理。
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