Bupathi Manojkumar, Ahn Daniel H, Bekaii-Saab Tanios
Medical Oncology, Ohio State University Medical Center, Columbus, OH 43210, USA.
Hematology/Medical Oncology, Mayo Clinic, Phoenix, AZ 85054, USA.
Hepatobiliary Surg Nutr. 2017 Apr;6(2):91-100. doi: 10.21037/hbsn.2016.12.12.
Biliary tract cancer (BTC) is a heterogeneous group of cancers, which is composed of intrahepatic cholangiocarcinoma (ICCA), extrahepatic cholangiocarcinoma (ECCA), gallbladder cancers and ampullary carcinomas. While all anatomic subgroups are treated uniformly, our understanding about the pathogenesis has allowed us to reason that each group represents a clinically and genetically diverse disease. The majority of patients present with locally advanced or metastatic disease, where the standard treatment is combination systemic cytotoxic chemotherapy with gemcitabine and cisplatin. While most receive a clinical benefit from chemotherapy, patients eventually progress where no standardized therapies are available in the refractory setting. With the use of next generation sequencing, we have come to understand that ICCA is a diverse genomic disease with many actionable alterations that may serve as potential therapeutic targets. Further studies investigating the role of novel targeted agents (as a single agent or with combination chemotherapy) will hopefully provide additional treatment options for this highly lethal disease.
胆管癌（BTC）是一组异质性癌症，由肝内胆管癌（ICCA）、肝外胆管癌（ECCA）、胆囊癌和壶腹癌组成。虽然所有解剖亚组的治疗方式相同，但我们对发病机制的了解使我们推断，每组都代表一种临床和基因上不同的疾病。大多数患者表现为局部晚期或转移性疾病，标准治疗是吉西他滨和顺铂联合全身细胞毒性化疗。虽然大多数患者从化疗中获得临床益处，但患者最终会进展，在难治性情况下没有标准化治疗方法。通过使用下一代测序，我们已经了解到ICCA是一种具有多种可操作改变的多样化基因组疾病，这些改变可能作为潜在的治疗靶点。进一步研究新型靶向药物（作为单一药物或与联合化疗）的作用有望为这种高致死性疾病提供更多治疗选择。
