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中华乳腺病杂志(电子版) ›› 2026, Vol. 20 ›› Issue (02) : 69 -81. doi: 10.3877/cma.j.issn.1674-0807.2026.02.001

所属专题: 指南共识

指南与共识

乳腺癌内分泌治疗超说明书用药专家共识
中国临床肿瘤学会乳腺癌专家委员会, 中国医师协会肿瘤多学科诊疗专业委员会, 广东省药学会   
  1. 1. 510120 广州,中山大学孙逸仙纪念医院乳腺肿瘤中心
    2. 解放军总医院第五医学中心肿瘤医学部
  • 收稿日期:2025-10-10 出版日期:2026-04-01
  • 基金资助:
    国家科技重大专项项目(2020ZX09201-021); 癌症、心脑血管、呼吸和代谢性疾病防治研究国家科技重大专项(2024ZD0519800,2024ZD0519805); 广东省科技计划项目(2023B110005); 广州市科技计划基础研究计划(2023A03J0725)

Expert consensus on off-label use of endocrine therapy for breast cancer

Breast Cancer Committee of Chinese Society of Clinical Oncology, Multidisciplinary Committee on Oncology of Chinese Medical Doctor Association, Guangdong Pharmaceutical Association   

  1. 1. Breast Tumor Center, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou 510120, China
    2. Department of Oncology, Fifth Medical Center of Chinese PLA General Hospital, Beijing 100039, China
  • Received:2025-10-10 Published:2026-04-01
引用本文:

中国临床肿瘤学会乳腺癌专家委员会, 中国医师协会肿瘤多学科诊疗专业委员会, 广东省药学会. 乳腺癌内分泌治疗超说明书用药专家共识[J/OL]. 中华乳腺病杂志(电子版), 2026, 20(02): 69-81.

Breast Cancer Committee of Chinese Society of Clinical Oncology, Multidisciplinary Committee on Oncology of Chinese Medical Doctor Association, Guangdong Pharmaceutical Association. Expert consensus on off-label use of endocrine therapy for breast cancer[J/OL]. Chinese Journal of Breast Disease(Electronic Edition), 2026, 20(02): 69-81.

内分泌治疗是激素受体阳性乳腺癌的重要治疗手段,临床实践中普遍存在超说明书用药现象。中国临床肿瘤学会(CSCO)乳腺癌专家委员会、中国医师协会肿瘤多学科诊疗专业委员会、广东省药学会组织专家组,通过循证查证及系统评价乳腺癌内分泌超药品说明书用药治疗的依据,参考已公开发布的指南、共识、诊疗规范等外,对已上市的内分泌治疗药物的随机对照试验进行再评估,采用CSCO分级系统进行证据分级与推荐,最终形成针对21个临床问题的23条推荐意见,旨在为临床医师提供合理用药依据,并为药学部门管理提供参考。

Endocrine therapy is a crucial treatment modality for hormone receptor-positive breast cancer. However, off-label use of endocrine agents is widely observed in clinical practice. To provide evidence-based guidance, a panel of experts from the Breast Cancer Committee of the Chinese Society of Clinical Oncology (CSCO), the Oncology Multidisciplinary Committee of the Chinese Medical Doctor Association, and the Guangdong Pharmaceutical Association systematically reviewed and evaluated the evidence supporting off-label endocrine therapy for breast cancer. This process involved comprehensive literature searches, critical appraisal of existing guidelines, consensus statements, and practice standards, as well as a re-evaluation of randomized controlled trials (RCTs) involving approved endocrine agents. Using the CSCO evidence grading and recommendation system, the strength of evidence and recommendations were determined. Consequently, 23 recommendations addressing 21 clinical questions were developed. This consensus aims to provide clinicians with a basis for rational drug use and offers a reference for pharmacy departments in managing off-label endocrine therapies.

表1 目前国内获批用于乳腺癌内分泌治疗的药物
分类 通用名 国内已批准的适应证
SERM类 他莫昔芬 1. 复发或转移性乳腺癌
2.早期乳腺癌术后的辅助治疗
3.不排卵性不育症
托瑞米芬 绝经后妇女ER阳性/或不详的转移性乳腺癌
AI类 来曲唑 1.ER或PR阳性绝经后早期乳腺癌患者的辅助治疗
2.ER或PR阳性, 已经接受他莫昔芬辅助治疗5年的、绝经后早期乳腺癌患者的辅助治疗
3.自然绝经或人工诱导绝经后、ER及PR阳性或受体状况不明的晚期乳腺癌患者
阿那曲唑 1.对他莫昔芬呈现阳性的临床反应的ER阴性绝经后晚期乳腺癌
2.HR阳性的绝经后早期乳腺癌的辅助治疗
3.曾接受2~3年他莫昔芬辅助治疗的HR阳性绝经后早期乳腺癌的辅助治疗
依西美坦 1.他莫昔芬辅助治疗2~3年后 ER阳性的绝经后早期浸润性乳腺癌的辅助治疗,直至完成总共5年的辅助内分泌治疗
2.经他莫昔芬治疗后病情进展的自然或人工绝经后晚期乳腺癌
SERD类 氟维司群 1.在抗雌激素辅助治疗后或治疗中复发或进展的绝经后(自然绝经和人工绝经)ER阳性局部晚期或转移性乳腺癌
2.与阿贝西利联合治疗, 用于HR阳性、HER-2阴性局部晚期或转移性乳腺癌,或既往接受内分泌治疗后疾病进展者
CDK4/6抑制剂 哌柏西利 HR阳性、HER-2阴性局部晚期或转移性乳腺癌,应与AI联用作为绝经后女性患者的初始内分泌治疗
阿贝西利 1.联合内分泌治疗(他莫昔芬或AI)用于HR阳性、HER-2阴性、淋巴结阳性高复发风险的早期乳腺癌
2.HR阳性、HER-2阴性局部晚期或转移性乳腺癌:与AI联用作为绝经后患者的初始内分泌治疗;与氟维司群联合用于内分泌治疗后疾病进展的患者
瑞波西利 1.与AI联用,用于HR阳性、HER-2阴性高复发风险的早期乳腺癌的辅助治疗
2.与AI联用,用于HR阳性、HER-2阴性局部晚期或转移性乳腺癌的初始内分泌治疗
达尔西利 HR阳性、HER-2阴性局部晚期或转移性乳腺癌:与AI联用作为初始内分泌治疗;与氟维司群联合用于内分泌治疗后疾病进展的患者
表2 中国临床肿瘤学会诊疗指南证据类别
表3 中国临床肿瘤学会诊疗指南推荐等级
表4 乳腺癌内分泌治疗超说明书用药推荐意见汇总
临床问题 Ⅰ级推荐 Ⅱ级推荐 Ⅲ级推荐 不推荐
预防治疗
原位乳腺导管癌患者在接受手术和放射治疗后,用于降低浸润性乳腺癌的发生风险 常规剂量 TAMa 小剂量TAMb;EXE(绝经后)c;ANA(绝经后)c TOR
降低乳腺癌高危女性的发病风险预防治疗 小剂量 TAMb
乳腺癌不典型增生的预防治疗 TOR
术前新辅助治疗
需要术前新辅助治疗但不适合手术或无须即刻手术及新辅助化疗不敏感或疗效欠佳的HR阳性HER-2阴性乳腺癌患者或用于HR阳性HER-2阴性乳腺癌的新辅助内分泌治疗 AI(绝经后);OFS+AI(绝经前) AI+CDK4/6i(绝经后);OFS+AI+CDK4/6i(绝经前) TAM;FUL
术后辅助治疗
HR阳性早期乳腺癌术后辅助内分泌治疗 TOR(绝经前)
SERM用于HR阳性HER-2阴性乳腺癌的辅助治疗能否>5年 TAMd OFS+TAMe
AI用于绝经后HR阳性乳腺癌的辅助治疗5年,辅助内分泌疗程能否再延长3~5年 延长 AI 3~5年f
HR阳性绝经后乳腺癌辅助治疗,TAM治疗时间<2年或>3年时,能否序贯AI辅助内分泌治疗 TAM 治疗时间>3年,序贯AI(绝经后) TAM治疗时间<2年,序贯AI(绝经后)
HR阳性绝经后乳腺癌辅助治疗,TAM序贯AI总疗程能否>5年 总疗程可以超过5年
AI联合OFS能否用于绝经前HR阳性乳腺癌的术后辅助治疗 OFS+EXE ER弱阳性者(1%~10%)不推荐OFS+EXE
阿贝西利联合内分泌治疗能否用于HR阳性、HER-2阴性、N1、Ki-67>20%乳腺癌辅助治疗 阿贝西利
哌柏西利联合内分泌治疗能否用于HR阳性、HER-2阴性、高复发风险乳腺癌的辅助治疗 不推荐
男性乳腺癌
HR阳性男性乳腺癌术后辅助内分泌治疗 AI+GnRHag TOR
激素剥夺治疗药物能否单独用于HR阳性男性早期乳腺癌的治疗 激素剥夺治疗药物
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