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序贯多分配随机试验设计样本量估算方法及应用

Methodological research on sample size estimation methods for SMART design
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摘要 序贯多分配随机试验(SMART)是一种结合序贯治疗与动态治疗策略进行的临床研究设计,其面临的主要挑战之一是确定适当的样本量。SMART样本量估算方法是根据4个主要研究目标的不同而估算方式不同。试点研究是SMART设计的前期筹备阶段,样本量估算方法主要基于精度和可行性两个方面。以估计一线和二线治疗措施的主要效果为目的SMART设计样本量估算方法与随机对照试验中常用的样本量估算方法相似。比较嵌入式自适应治疗措施包括:最优自适应治疗选择、配对优势检验、配对非劣性检验和与最佳者的多重比较。以开发优化为目标的SMART样本量估算主要包括以基于正态分布的样本量估算方式。本研究旨在通过实例进行展示,为SMART设计的样本量估算提供实用参考,以促进中医药领域SMART设计研究的发展。 The Sequential Multi-Assignment Randomized Trial(SMART)design incorporates sequential and dynamic treatment strategies in clinical research.Determining an appropriate sample size poses a significant challenge in SMART studies.Sample size estimation methods for SMART design vary according to four primary research objective.Pilot studies,conducted as preparatory stages,primarily focus on precision and feasibility in sample size estimation.Estimating the main effects of first-line and second-line treatments in SMART design aligns with the commonly used sample size estimation methods in randomized controlled trials.Comparative analyses of embedded adaptive treatment measures encompass optimal adaptive treatment selection,paired dominance testing,paired non-inferiority testing,and multiple comparisons with the best-performing interventions.Sample size estimation for SMART designs aimed at developmental optimization primarily employs methods based on the normal distribution.This study aims to provide practical guidance and examples for sample size estimation in SMART designs,thereby advancing research in SMART design within the field of TCM.
作者 卜志军 张雨欢 孙源 韩朝歌 王雪惠 徐耀宇 贺婷 陈婧如 李俊宏 黄智芮 刘冯双 刘建平 刘兆兰 BU Zhijun;ZHANG Yuhuan;SUN Yuan;HAN Zhaoge;WANG Xuehui;XU Yaoyu;HE Ting;CHEN Jingru;LI Junhong;HUANG Zhirui;LIU Fengshuang;LIU Jianping;LIU Zhaolan(Centre for Evidence-Based Medicine,Beijing University of Chinese Medicine,Beijing 100029;College of Acupuncture,Bone-setting and Traumatology,Hubei University of Chinese Medicine;School of Traditional Chinese Medicine,Hubei University of Chinese Medicine;The First Clinical College,Hubei University of Chinese Medicine;School of Pharmacy,Hubei University of Chinese Medicine;The Second Clinical College of Beijing University of Chinese Medicine)
出处 《现代中医临床》 2024年第4期39-43,共5页 Modern Chinese Clinical Medicine
基金 国家自然科学基金项目(No.82374298) 北京中医药大学学科后备带头人支持计划(No.90010960920033) 国家中医药管理局高水平中医药重点学科建设项目(No.zyyzdxk-2023249)。
关键词 序贯多分配随机试验 样本量估算 SMART设计 辨证论治 sequential multi-assignment randomized trial sample size estimation SMART design pattern differentiation and treatment
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