精准医学模式下的母方案篮式试验设计方法及其在中医药领域中的应用  

Design of master protocol basket trial in precision medicine and its application in traditional Chinese medicine

作　　者：陈莹 常静玲[3] 代恒恒[1,2] 张凯歌 张晶晶 张学成 万思琦[1,2] 关之玥 胡明智[1,2] 刘岩 商洪才 CHEN Ying;CHANG Jingling;DAI Hengheng;ZHANG Kaige;ZHANG Jingjing;ZHANG Xuecheng;WAN Siqi;GUAN Zhiyue;HU Mingzhi;LIU Yan;SHANG Hongcai(Key Laboratory of Chinese Internal Medicine of Ministry of Education,Dongzhimen Hospital,Beijing University of Chinese Medicine,Beijing 100700,P.R.China;Beijing University of Chinese Medicine,Beijing 100029,P.R.China;Department of Encephalopathy,Dongzhimen Hospital,Beijing University of Chinese Medicine,Beijing 100700,P.R.China)

机构地区：[1]北京中医药大学东直门医院中医内科学教育部和北京市重点实验室,北京100700 [2]北京中医药大学,北京100029 [3]北京中医药大学东直门医院脑病科,北京100700

出　　处：《中国循证医学杂志》2022年第4期483-489,共7页Chinese Journal of Evidence-based Medicine

基　　金：国家自然科学基金项目(编号:81725024)。

摘　　要：精准医学是根据患者生物学信息及临床症状和体征建立的个体化医疗体系,形成了新的临床研究模式和医疗实践路径。中医学的思想和精准医学的理念在很多方面高度契合,为精准医学开发的篮式试验设计亦适用于中医辨证论治体系的临床试验和疗效评价。篮式试验用于评价一种药物治疗多种疾病或疾病亚型的疗效,具有共享母方案设计、统一管理子研究、精简试验实施流程、统一进行数据分析、节约资源、减少预算、加快药物评价进度等优势,与中医学“异病同治”理念相似。本文通过介绍篮式试验的概念、设计方法,探讨其在中医药临床研究中的应用和优势,以期为中医药临床研究的方法学创新提供参考。Precision medicine is a personalized medical system based on patients’ individual biological information, clinical symptoms and signs, forming a new clinical research model and medical practice path. The basic idea of traditional Chinese medicine and the concept of precision medicine share many similarities. The basket trial developed for precision medicine is also suitable for clinical trials and evaluation of the efficacy of traditional Chinese medicine syndrome differentiation and treatment systems. Basket trials are used to evaluate the efficacy of a drug in the treatment of multiple diseases or disease subtypes. It has the advantages of sharing a master protocol, unifying management of subsidiary studies, simplifying the test implementation process, unifying statistical analysis, saving resources, reducing budgets and accelerating the drug evaluation progress. This is similar to the concept of using the "same treatment for different diseases" found in traditional Chinese medicine. This paper introduced the concept and method of basket trials and explored their application and advantages in clinical research into traditional Chinese medicine. This study is expected to provide references for the methodological innovation of clinical research into traditional Chinese medicine.

关 键 词：精准医学 母方案 篮式试验 异病同治 以证统病 中医药临床研究 疗效评价方法 

分 类 号：R2-03[医药卫生—中医学]