胆道癌是第二常见的肝癌类型，由于其诊断较晚和预后不良，仍然是治疗上的挑战。近年来，很明显，经典化疗可能不是胆道癌患者的最佳治疗方法，特别是在一线治疗失败后。因此，寻找新的治疗方案和策略来提高这些患者的生存率至关重要。随着肿瘤患者基因检测的兴起和可用性的不断提高，最近提出了针对特定基因改变的新治疗方法，并在大量临床试验中证明了其安全性和有效性。在这篇综述中，我们将首先考虑化疗方案以及一线治疗中化疗与免疫检查点抑制剂相结合的新可能性。然后，我们将概述基因组改变及其靶向治疗尤其是二线治疗的潜力。除了最常见的改变（例如异柠檬酸脱氢酶 1 或 2 (IDH1/2) 突变、成纤维细胞生长因子受体 2 (FGFR2) 融合和改变）之外，我们还将讨论不太常见的改变，例如 BRAF V600E 突变和神经营养性酪氨酸激酶受体基因（NTRK）融合。我们强调分子分析在指导治疗决策中的重要性，并强调需要继续研究以优化和扩展这种侵袭性恶性肿瘤的靶向治疗策略。版权所有 © 2024 作者。由爱思唯尔有限公司出版。保留所有权利。

Biliary tract cancer, the second most common type of liver cancer, remains a therapeutic challenge due to its late diagnosis and poor prognosis. In recent years, it has become evident that classical chemotherapy might not be the optimal treatment for patients with biliary tract cancer, especially after failure of first-line therapy. Finding new treatment options and strategies to improve the survival of these patients is therefore crucial. With the rise and increasing availability of genetic testing in patients with tumor, novel treatment approaches targeting specific genetic alterations have recently been proposed and have demonstrated their safety and efficacy in numerous clinical trials. In this review, we will first consider chemotherapy options and the new possibility of combining chemotherapy with immune checkpoint inhibitors in first-line treatment. We will then provide an overview of genomic alterations and their potential for targeted therapy especially in second-line therapy. In addition to the most common alterations such as isocitrate dehydrogenase 1 or 2 (IDH1/2) mutations, fibroblast growth factor receptor 2 (FGFR2) fusions, and alterations, we will also discuss less frequently encountered alterations such as BRAF V600E mutation and neurotrophic tyrosine kinase receptor gene (NTRK) fusion. We highlight the importance of molecular profiling in guiding therapeutic decisions and emphasize the need for continued research to optimize and expand targeted treatment strategies for this aggressive malignancy.Copyright © 2024 The Author(s). Published by Elsevier Ltd.. All rights reserved.