胆管癌靶向治疗研究进展

Review on Cholangiocarcinoma Target Therapy

胆管癌(CCA)是源于胆管上皮细胞恶性增殖的较为少见的恶性肿瘤。因疾病早期无特异临床表现,诊断困难,一经诊断多为中晚期,无法手术。即使采取手术切除,术后生存率也较低。无手术指征的患者一线标准治疗(SOC)为吉西他滨联合顺铂,中位生存期小于1年。2020年4月,成纤维细胞生长因子受体(FGFR)抑制剂培米替尼获美国FDA批准上市,成为全球首款胆管癌靶向药物。对近年胆管癌靶向治疗的进展进行综述,旨在为胆管癌的药物研发以及临床工作提供相关参考。

Cholangiocarcinoma(CCA)is a rare malignant tumor derived from uncontrolled proliferation of bile duct epithelial cells.Because of the asymptomatic early-stage manifestations,CCA is hard to diagnose until advanced stage,which is generally unresectable.The postoperative survival rate is quite low even after surgical resection.Gemcitabine combined with cisplatin is the first-line standard of care(SOC)for patients who do not need surgical resection,and their median overall survival time is less than 1 year.In April 2020,pemigatinib,an FGFR inhibitor approved by FDA,became the first target therapy for cholangiocarcinoma worldwide.This paper reviews the progress of target therapy for cholangiocarcinoma in recent years,aiming to provide reference for novel drug development and clinical practice for cholangiocarcinoma treatment.