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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。
Abstract：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.
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基本信息:
DOI：10.13288/j.11-2166/r.2025.08.006
中图分类号:R259;R277.5
引用信息:
[1]曹灿,孙卫卫,马彤等.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[J].中医杂志,2025,66(08):790-794.DOI:10.13288/j.11-2166/r.2025.08.006.
基金信息:
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）