许多胆管癌（CCA）患者不适合手术治疗，化疗的生存益处少于12个月。最近在CCA中发现了多种基因突变和突变簇，其中一些可通过药物作用靶向治疗。靶向治疗的出现显著改变了CCA的治疗格局，并改善了晚期或转移性CCA的预后。本文旨在描述CCA过去和现有的治疗策略，重点关注FDA批准的靶向治疗。对截至2022年10月所有FDA批准的CCA靶向治疗进行了系统评估。从药品说明书和临床试验数据中收集了有关药理学、临床疗效和安全性的信息。截至本文撰写时，已批准了四种针对局部晚期或转移性CCA的靶向剂。这些药物包括IDH1抑制剂伊沃西布和FGFR2抑制剂佩米加替尼、因菲加替尼和库伯替尼。总体而言，这些药物为选择性先前接受局部晚期或不可切除CCA治疗的患者提供了额外的治疗选择。这些药物还为开发其他针对CCA的靶向疗法作出了贡献，并为探索新的治疗组合（如化疗和免疫治疗）打开了大门，这些组合最近已成为一线治疗选择。四种靶向小分子药物已成为CCA第二线治疗的有效疗法，这极大地改变了治疗格局，并直接促进了对靶向剂和免疫治疗作为CCA治疗的进一步研究。

Many patients with cholangiocarcinoma (CCA) are not surgical candidates, and the survival benefit of chemotherapy is less than 12 months. Several mutations and mutational clusters have recently been identified in CCA, some of which are pharmacologically targetable. The emergence of targeted therapies has significantly altered the treatment landscape of CCA and improved the prognosis for advanced or metastatic CCA. The purpose of this review is to describe past and current treatment strategies of CCA with a focus on FDA-approved targeted therapies.A systematic evaluation of all FDA-approved targeted treatments for CCA through October 2022 was conducted. Information related to pharmacology, clinical efficacy, and safety was gathered from the package insert, and clinical trial data.As of the writing of this review, four targeted agents are FDA approved for the treatment of locally advanced or metastatic CCA. These agents include the IDH1 inhibitor ivosidenib and the FGFR2 inhibitors pemigatinib, infigratinib, and futibatinib. Collectively, these agents have provided additional treatment options for select patients with previously treated locally advanced or unresectable CCA. These agents have also contributed to the development of other targeted therapies for the treatment of CCA and have opened the door for the exploration of novel treatment combinations such as chemotherapy and immunotherapy, which have recently become a front-line treatment option.Four targeted small molecule agents have emerged as effective therapies in the second-line setting for CCA, which has immensely changed the treatment landscape and directly led to further investigation of targeted agents and immunotherapy as treatment for CCA.