中医药治疗糖尿病肾病临床试验替代结局指标的探索与思考  

Exploration on Surrogate Outcome Measures in Clinical Trials of Traditional Chinese Medicine for Diabetic Kidney Disease

作　　者：曹灿[1] 孙卫卫[1] 马彤 王耀献 CAO Can;SUN Weiwei;MA Tong;WANG Yaoxian(Dongzhimen Hospital,Beijing University of Chinese Medicine(Tongzhou Branch),Beijing,101121;Dongzhimen Hospital,Beijing University of Chinese Medicine;Beijing University of Chinese Medicine;Henan University of Chinese Medicine)

机构地区：[1]北京中医药大学东直门医院通州院区,北京市101121 [2]北京中医药大学东直门医院 [3]北京中医药大学 [4]河南中医药大学

出　　处：《中医杂志》2025年第8期790-794,共5页Journal of Traditional Chinese Medicine

基　　金：北京中医药大学东直门医院临床研究和成果转化能力提升试点项目(DZMG-TZZX-24010);2022年度基本科研业务费揭榜挂帅项目(2022-JYB-JBZR-038);国家中医药管理局中医药传承与创新“百千万”人才工程(岐黄学者)(国中医药人教发〔2018〕12号)。

摘　　要：中医药在缓解糖尿病肾病(DKD)症状和延缓病程方面具有独特优势,但传统临床试验中以终末期肾病的发生作为终点指标,需长时间随访,增加了试验的复杂性和成本,限制了中医药临床试验的可行性。认为在中医药治疗DKD的临床试验中,肾小球滤过率(eGFR)变化率(≥30%)和eGFR斜率均可作为潜在的替代结局指标,若干预周期较短(<1年),建议使用eGFR变化率(≥30%)作为替代结局指标;而在长期(≥1年)干预的研究中,eGFR斜率则可能更优。在理化指标如eGFR斜率、尿白蛋白/肌酐比值(UACR)变化率的基础上,结合中医症状学评价、中医证候评价与生活质量量表,开发可被国际认可的中医患者报告结局指标,将是提升中医药治疗DKD疗效评估体系的关键步骤。Traditional Chinese medicine(TCM)has unique advantages in alleviating the symptoms of diabetic kidney disease(DKD)and slowing its progression.However,traditional clinical trials often use the occurrence of end-stage renal disease as the end point,requiring long-term follow-up,which increases trial complexity and costs,thereby limiting the feasibility of TCM clinical studies.This paper suggested that in clinical trials of TCM for DKD,both the estimated glomerular filtration rate(eGFR)change rate(≥30%)and eGFR slope can serve as potential surrogate outcome measures.If the intervention course is short(<1 year),the eGFR change rate(≥30%)is recommended as a surrogate outcome measure,whereas in long-term interventional studies(≥1 year),the eGFR slope may be more appropriate.Furthermore,based on biochemical indicators such as eGFR slope and urinary albumin-tocreatinine ratio(UACR)change rate,integrating TCM symptom evaluation,TCM syndrome evaluation,and quality of life scales can help develop internationally recognized patient-reported outcome measures(PROMs)for TCM clinical trials,which will be a key step in enhancing the evaluation system for the effectiveness of TCM in treating DKD.

关 键 词：糖尿病肾病 结局指标 替代指标 患者报告结局 生活质量 中医症状 

分 类 号：R259[医药卫生—中西医结合]