代谢相关脂肪性肝病中医临床疗效评价体系的构建

孙屿昕; 童光东

doi:10.12449/JCH260503

代谢相关脂肪性肝病中医临床疗效评价体系的构建

DOI: 10.12449/JCH260503

孙屿昕^{1, 2},
童光东^{1, 2, , ,}

1.
深圳市中医院肝病科，广东深圳 518000
2.
广州中医药大学附属第四临床医学院，广东深圳 518000

基金项目:

深圳市重点实验室计划 (ZDSYS20210623092000002);

深圳市“医疗卫生三名工程”项目 (SZZYSM202411013);

广东省横琴重点实验室 (HQCML-C-2024007)

利益冲突声明：本文不存在任何利益冲突。

作者贡献声明：童光东负责选题设计、研究思路确定及评价体系框架等关键内容审定；孙屿昕负责评价指标体系设计、资料分析及论文撰写；童光东和孙屿昕审阅并最终定稿。

详细信息

通信作者:
童光东， tgd755@163.com （ORCID： 0000-0002-8577-574X）

计量
- 文章访问数: 18
- HTML全文浏览量: 3
- PDF下载量: 6
- 被引次数: 0
出版历程
- 收稿日期: 2026-03-19
- 录用日期: 2026-04-15
- 出版日期: 2026-05-20

Construction of a clinical efficacy evaluation framework for traditional Chinese medicine treatment of metabolic dysfunction-associated fatty liver disease

Yuxin SUN^{1, 2},
Guangdong TONG^{1, 2
, ,
,}

1.
Department of Hepatology，Shenzhen Hospital of Traditional Chinese Medicine，Shenzhen，Guangdong 518000，China
2.
The Fourth Clinical Medical College of Guangzhou University of Chinese Medicine，Shenzhen，Guangdong 518000，China

Research funding:

Shenzhen Key Laboratory Program (ZDSYS20210623092000002);

Shenzhen Medical and Health "Three Famous Projects" (SZZYSM202411013);

Hengqin Key Laboratory of Guangdong Province (HQCML-C-2024007)

More Information

Corresponding author: TONG Guangdong， tgd755@163.com （ORCID： 0000-0002-8577-574X）

摘要

摘要: 本文在梳理代谢相关脂肪性肝病（MAFLD）临床试验评价指标和中医药治疗相关研究现状的基础上，结合MAFLD疾病特点及中医药疗效特征，提出了一个多维、分层、可操作的中医临床疗效评价体系框架。该体系涵盖肝脏组织学、影像学、生化指标、代谢指标、人体测量学、中医证候、患者报告结局和安全性指标等8个维度，并根据研究定位和疾病阶段进行分层设计。本文进一步探讨了证候评价标准化、研究设计规范、质量控制等实施路径，以期为开展高质量中医药临床研究提供方法学支撑，推动中医药疗效评价从“单指标”向“多维复合指标”转变。
- 代谢相关脂肪性肝病 /
- 中医药疗法 /
- 治疗结果
Abstract: Based on a review of clinical trial assessment indices for metabolic dysfunction-associated fatty liver disease （MAFLD） and the current status of traditional Chinese medicine （TCM） research， this article proposes a multi-dimensional， stratified， and operable clinical efficacy evaluation framework for TCM with reference to the features of MAFLD and the therapeutic effect of TCM. This system encompasses eight dimensions of liver histology， radiology， biochemical indicators， metabolic markers， anthropometry， TCM syndromes， patient-reported outcomes， and safety indicators， which are stratified according to research objectives and disease stages. This study further explores the implementation pathways for the standardization of syndrome evaluation， research design norms， and quality control， in order to provide methodological support for conducting high-quality TCM clinical research and promote the transition of TCM efficacy evaluation from “single indicators” to “multi-dimensional composite indicators”.
- Metabolic Dysfunction-Associated Fatty Liver Disease /
- Traditional Chinese Medicine Therapy /
- Treatment Outcome

HTML全文

表 1 MAFLD中医临床疗效评价指标体系的主要维度及核心指标建议

Table 1. Main dimensions and core indicator recommendations for the traditional Chinese medicine clinical efficacy evaluation index system of MAFLD

评价维度	评价内容	核心指标举例	中医药特色
肝脏组织学	脂肪变性、炎症活动、气球样变及纤维化程度	NAS评分、肝纤维化分期	为疗效评价金标准，可直接反映病理改善，但侵入性较强，临床普遍应用受限
肝脏影像学	肝脂肪含量、肝脏硬度、肝纤维化风险	MRI-PDFF、CAP、LSM	无创、可重复，适用于动态随访与疗效监测
肝脏生化学	肝细胞损伤、胆汁淤积及肝功能状态	ALT、AST、GGT、TBil	为常规客观评价指标，便于临床广泛应用
代谢指标	糖代谢、脂代谢、胰岛素抵抗及尿酸代谢	FBG、HOMA-IR、TC、TG、 HDL-C、LDL-C、UA	能较好体现MAFLD的代谢异常本质及肝外获益
人体测量学	肥胖程度、脂肪分布及体成分变化	BMI、腰围、体脂率、腰臀比	可反映生活方式及综合干预效果
中医证候	证候严重程度、证候演变及临床症状特征	证候积分、主症/次症评分、舌脉指标	为体现中医辨证论治特色的核心维度，有助于评价中医药整体调节效应
患者报告结局	生活质量、症状负担、疲劳及功能状态	SF-36、CLDQ-NAFLD、疲劳评分	体现患者主观获益与临床价值导向
安全性指标	不良事件及用药安全性	不良事件发生率、肝肾功能、血常规、心电图	用于综合评估干预措施的安全性与耐受性
注：对特定MAFLD人群，可补充评估饮酒情况、心脑血管事件风险及慢性肾脏病风险相关指标、全因死亡率，以辅助疗效解释及长期结局判断。MAFLD，代谢相关脂肪性肝病；NAS评分，MAFLD活动度评分；MRI-PDFF，磁共振质子密度脂肪分数；CAP，受控衰减参数；LSM，肝硬度值；ALT，丙氨酸氨基转移酶；AST，天冬氨酸氨基转移酶；GGT，γ-谷氨酰转移酶；TBil，总胆红素；FBG，空腹血糖；TC，总胆固醇；TG，甘油三酯；HDL-C，高密度脂蛋白胆固醇；LDL-C，低密度脂蛋白胆固醇；UA，尿酸；BMI，体重指数；SF-36，36项简短健康状况调查表；HOMA-IR，稳态模型评估的胰岛素抵抗指数；CLDQ-NAFLD，非酒精性脂肪性肝病生活质量问卷。

下载: 导出CSV

表 2 MAFLD中医临床疗效分层评价体系建议

Table 2. Suggestions for a stratified traditional Chinese medicine clinical efficacy evaluation system for MAFLD

分层维度	类别	评价建议
定位分层	探索性研究	以机制探索和信号发现为目标，可纳入多组学、影像学及新型生物标志物等探索性指标
定位分层	验证性研究	以疗效确证为目标，需预设主要终点和关键次要终点
疾病阶段分层	单纯性脂肪肝	主要终点可选择肝脂肪含量改善（MRI-PDFF/CAP）或代谢指标改善
	MASH伴轻度肝纤维化（F1～F2）	主要终点可选择ALT复常或MASH缓解
	MASH伴显著肝纤维化（F2～F3）	主要终点宜参考FDA认可的组织学复合终点，即MASH缓解且肝纤维化无恶化
证候类型分层	湿热蕴结证	重点关注肝酶、炎症相关指标及FGF21等
	脾虚湿盛证	重点关注胰岛素抵抗、脂代谢异常及疲劳症状等
	痰瘀互结证	重点关注肝纤维化指标、血脂及血液流变学指标等
注：MAFLD，代谢相关脂肪性肝病；MRI-PDFF，磁共振质子密度脂肪分数；CAP，受控衰减参数；MASH，代谢相关脂肪性肝炎；ALT，丙氨酸氨基转移酶；FDA，美国食品药品监督管理局；FGF21，成纤维细胞生长因子21。

下载: 导出CSV

表 3 MAFLD中医药临床研究核心指标集的初步建议

Table 3. Preliminary recommendations for the core outcome set in traditional Chinese medicine clinical research on MAFLD

终点层级	指标类别	具体指标建议
主要终点	根据研究目的选择其一	肝脂肪含量相对下降≥30%（MRI-PDFF）；ALT复常（基线升高者）；MASH缓解且肝纤维化无恶化；中医证候积分下降≥50%
次要终点	中医证候、与证候相关指标（选择一项或多项）	肝酶谱（ALT、AST、GGT）；糖代谢指标（FBG、HOMA-IR、HbA1c）；脂代谢指标（TC、TG、HDL- C、LDL-C）；影像学指标（CAP、LSM）；人体测量学指标（BMI、WHR）；中医证候积分（各证型分量表）
探索性终点	机制与前瞻性观察	新型生物标志物（FGF21、CK-18、Pro-C3）；肠道菌群及其代谢产物（SCFA、胆汁酸）；代谢组学/脂质组学特征
注：MAFLD，代谢相关脂肪性肝病；MRI-PDFF，磁共振质子密度脂肪分数；ALT，丙氨酸氨基转移酶；MASH，代谢相关脂肪性肝炎；AST，天冬氨酸氨基转移酶；GGT，γ-谷氨酰转移酶；FBG，空腹血糖；HOMA-IR，稳态模型评估的胰岛素抵抗指数；HbA1c，糖化血红蛋白；TC，总胆固醇；TG，甘油三酯；HDL-C，高密度脂蛋白胆固醇；LDL-C，低密度脂蛋白胆固醇；CAP，受控衰减参数；LSM，肝硬度值；BMI，体重指数；WHR，腰臀比；FGF21，成纤维细胞生长因子21；CK-18，细胞角蛋白18； Pro-C3，Ⅲ型前胶原C端肽；SCFA，短链脂肪酸。

下载: 导出CSV

参考文献(15)

[1]	MIAO L, TARGHER G, BYRNE CD, et al. Current status and future trends of the global burden of MASLD[J]. Trends Endocrinol Metab, 2024, 35( 8): 697- 707. DOI: 10.1016/j.tem.2024.02.007.
[2]	TANG Y, GU CH. Epidemiological transition of metabolic dysfunction-associated steatotic liver disease-related chronic liver disease and cirrhosis: A comparative study between China and the global population(1990—2021)[J]. Front Nutr, 2025, 12: 1624440. DOI: 10.3389/fnut.2025.1624440.
[3]	Chinese Society of Hepatology, Chinese Medical Association. Guidelines for the prevention and treatment of metabolic dysfunction-associated(non-alcoholic) fatty liver disease(Version 2024)[J]. J Pract Hepatol, 2024, 27( 4): 494- 510. DOI: 10.3760/cma.j.cn501113-20240327-00163. 中华医学会肝病学分会. 代谢相关(非酒精性)脂肪性肝病防治指南(2024年版)[J]. 实用肝脏病杂志, 2024, 27( 4): 494- 510. DOI: 10.3760/cma.j.cn501113-20240327-00163.
[4]	U.S. Food and Drug Administration. FDA approves first treatment for patients with liver scarring due to fatty liver disease[EB/OL].( 2024-03-14)[ 2026-04-15]. https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-patients-liver-scarring-due-fatty-liver-disease. https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-patients-liver-scarring-due-fatty-liver-disease
[5]	U.S. Food and Drug Administration. FDA approves treatment for serious liver disease known as‘MASH’[EB/OL].( 2025-08-15)[ 2026-04-15]. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-treatment-serious-liver-disease-known-mash. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-treatment-serious-liver-disease-known-mash
[6]	SUN ZL, WEI YH, XU YC, et al. The use of traditional Chinese medicine in the treatment of non-alcoholic fatty liver disease: A review[J]. Pharmacol Res Mod Chin Med, 2024, 12: 100475. DOI: 10.1016/j.prmcm.2024.100475.
[7]	LI JJ, JIAO RM, SU WQ, et al. Traditional Chinese medicine for non-alcoholic fatty liver disease: An overview of systematic reviews with evidence mapping and metabolic outcome assessment[J]. Front Pharmacol, 2025, 16: 1675793. DOI: 10.3389/fphar.2025.1675793.
[8]	Food and Drug Administration. Noncirrhotic nonalcoholic steatohepatitis with liver fibrosis: developing drugs for treatment guidance for industry[EB/OL].( 2018-12). https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM627376.pdf. https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM627376.pdf
[9]	YAO WW, LIU KJ, JI J, et al. Situation Analysis of the clinical outcomes of randomized controlled trials of traditional Chinese medicine intervention in non-alcoholic fatty liver disease[J]. Chin J Integr Tradit West Med Liver Dis, 2024, 34( 2): 136- 139. DOI: 10.3969/j.issn.1005-0264.2024.002.009. 姚伟伟, 刘可佳, 吉静, 等. 中医药干预非酒精性脂肪性肝病随机对照临床研究结局指标的现状分析[J]. 中西医结合肝病杂志, 2024, 34( 2): 136- 139. DOI: 10.3969/j.issn.1005-0264.2024.002.009.
[10]	COMET. Core outcome measures in effectiveness trials[EB/OL]. https://www.comet-initiative.org/. https://www.comet-initiative.org/
[11]	Food and Drug Administration. Patient-focused drug development: collecting comprehensive and representative input guidance for industry, FDA staff, and other stakeholders[EB/OL].( 2020-06). https://www.fda.gov/media/139088/download. https://www.fda.gov/media/139088/download
[12]	TONG GD, SUN YX. Theoretical and clinical practice of metabolic associated fatty liver disease based on the theory of“six stagnation diseases”[J]. J Clin Hepatol, 2024, 40( 10): 1949- 1953. DOI: 10.12449/JCH241004. 童光东, 孙屿昕. 从“六郁”论代谢相关脂肪性肝病的理论与临床实践[J]. 临床肝胆病杂志, 2024, 40( 10): 1949- 1953. DOI: 10.12449/JCH241004.
[13]	TANWAR SS, SHARMA S. Modulatory role of traditional Chinese medicine in gut–liver and adipose-liver axis dysfunction in MASLD[J]. Pharmacol Res Mod Chin Med, 2026, 18: 100743. DOI: 10.1016/j.prmcm.2025.100743.
[14]	DAI L, CHENG CW, TIAN R, et al. Standard protocol items for clinical trials with traditional Chinese medicine 2018: Recommendations, explanation and elaboration(SPIRIT-TCM extension 2018)[J]. Chin J Integr Med, 2019, 25( 1): 71- 79. DOI: 10.1007/s11655-018-2999-x.
[15]	MANYARA AM, DAVIES P, STEWART D, et al. Reporting of surrogate endpoints in randomised controlled trial reports(CONSORT-Surrogate): Extension checklist with explanation and elaboration[J]. BMJ, 2024, 386: e078524. DOI: 10.1136/bmj-2023-078524.