晚期胆管癌系统治疗：全球指南的分歧与区域临床实践

doi:10.16139/j.1007-9610.2025.02.01

摘要/Abstract

摘要：

胆管癌（CCA）是一类起源于胆道上皮的恶性肿瘤，晚期病人多以系统治疗为主。近年来，其分子分型及靶向治疗研究快速发展，推动胆管癌相关治疗指南的快速更新。本文主要针对晚期CCA的系统治疗方案进展以及国内外指南的相关推荐展开综述，旨在为CCA病人的临床管理和决策制定提供更多的循证医学证据。

关键词: 胆管癌, 系统治疗, 化疗, 免疫治疗, 分子靶向治疗

Abstract:

Cholangiocarcinoma (CCA) is a group of malignant tumors arising from the biliary epithelium, with systemic therapy serving as the primary treatment approach for patients with advanced disease. In recent years, advances in molecular classification and targeted therapy have led to rapid updates in treatment guidelines for CCA. This review summarized recent progress in systemic treatment strategies for advanced CCA and the relevant recommendations in both international and domestic guidelines, aiming to provide evidence-based references for clinical management and decision-making in patients with CCA.

Key words: Cholangiocarcinoma(CCA), Systemic therapy, Chemotherapy, Immunotherapy, Molecular targeted therapy

中图分类号:

R735.8

李淳, 马祖燚, 孙嘉, 李秉璐. 晚期胆管癌系统治疗：全球指南的分歧与区域临床实践[J]. 外科理论与实践, 2025, 30(2): 93-100.

LI Chun, MA Zuyi, SUN Jia, LI Binglu. Systemic therapy for advanced cholangiocarcinoma: divergences in global guidelines and regional clinical implementation[J]. Journal of Surgery Concepts & Practice, 2025, 30(2): 93-100.

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化疗方案	研究编号	研究阶段	研究人群来源	对比方案	纳入病人数量	主要终点	关键结果
GemCis	NCT00262769	Ⅲ期（ABC-02）	英国	GemCis 比吉西他滨	410	OS	11.7个月比8.1个月 (HR=0.64, P<0.001)
吉西他滨+替吉奥	UMIN000010667	Ⅲ期（JCOG1113）	日本	吉西他滨+替吉奥比吉西他滨	354	OS，非劣效性	15.1个月比13.4个月 (HR=0.945, P=0.0459)
GemCis+替吉奥	NCT02182778	Ⅲ期（KHBO1401- MITSUBA)	日本	GemCis+替吉奥比 GemCis	246	OS	13.5个月比12.6个月 (HR=0.79, P=0.046)
XELOX	NCT01470443	Ⅲ期	韩国	卡培他滨+奥沙利铂比吉西他滨+奥沙利铂	224	PFS率（6个月），非劣效性	46.7%比44.6%，符合非劣效标准

分子特征	药物	治疗阶段	研究阶段	文献来源
FGFR2融合	福巴替尼（futibatinib）	二线治疗	Ⅱ期（FIGHT-202、FOENIX-CCA2）	Vogel A 2024；Abou-Alfa GK 2020
FGFR2融合	培米替尼（pemigatinib）	二线治疗	Ⅱ期（FIGHT-202、FOENIX-CCA2）	Vogel A 2024；Abou-Alfa GK 2020
HER2阳性	德曲妥珠单抗（fam-trastuzumab deruxtecan-nxki）	二线治疗	Ⅱ期	Meric-Bernstam F 2024；Javle M 2021；Nakamura Y 2023
	曲妥珠单抗+帕妥珠单抗（trastuzumab + pertuzumab）
	妥卡替尼 + 曲妥珠单抗（tucatinib + trastuzumab）
IDH1突变	艾伏尼布（ivosidenib）（1类）	二线治疗	Ⅲ期（ClarIDHy）	Ghassan K Abou-Alfa 2020
NTRK基因融合阳性	恩曲替尼（entrectinib）	一线治疗、二线治疗	多瘤种Ⅱ期（basket）	Doebele RC 2020； Solomon BJ 2023
	拉罗替尼（larotrectinib）
	瑞普替尼（repotrectinib）
BRAF V600E突变	达拉非尼 + 曲美替尼（dabrafenib + trametinib）	二线治疗	Ⅱ期（ROAR）	Subbiah V 2020
RET基因融合阳性	普拉替尼（pralsetinib）（2B类）	一线治疗、二线治疗	Ⅰ/Ⅱ期	Subbiah V 2022
RET基因融合阳性	塞尔帕替尼（selpercatinib）（2B类）	一线治疗、二线治疗	Ⅰ/Ⅱ期	Subbiah V 2022
KRAS G12C突变阳性	阿达格拉西布（adagrasib）	二线治疗	Ⅱ期	Tanios S 2023