Gastroenterology and Digestive Oncology Department, Cochin Hospital, Assistance Publique-Hôpitaux de Paris, Paris 75014, France; Unité INSERM U1016, University of Paris, France.
Gastroenterology and Digestive Oncology Department, Cochin Hospital, Assistance Publique-Hôpitaux de Paris, Paris 75014, France.
Pharmacol Ther. 2020 Jun;210:107517. doi: 10.1016/j.pharmthera.2020.107517. Epub 2020 Feb 25.
Biliary tract cancers (BTCs) represent a heterogeneous group that includes intrahepatic cholangiocarcinomas (CCAs), perihilar-CCAs or Klatskin tumors, extrahepatic-CCAs, and gallbladder adenocarcinoma. These entities have distinct demographics, risk factors, clinical presentation, and molecular characteristics. In advanced BTCs, the recommendations are mainly supporting a doublet chemotherapy regimen using cisplatin/gemcitabine (CisGem) with a 5-year overall survival rate close to 5% and median overall survival (mOS) of less than a year. The lack of overall efficacy stresses the need for personalized therapies. Recently, whole-genome and transcriptome sequencing highlighted the diversity of BTCs' subtypes. Distinct genetic alterations were retrieved according to the localization, with a high rate of potentially actionable alterations. Targeted therapies and immunotherapy have since then been tested for BTCs, trying to propose a more personalized treatment. This review describes the different therapeutic options, validated and in development, for patients with advanced BTCs.
胆道癌（BTC）是一组异质性肿瘤，包括肝内胆管癌（CCA）、肝门部胆管癌或 Klatskin 肿瘤、肝外胆管癌和胆囊腺癌。这些实体具有不同的人口统计学、危险因素、临床表现和分子特征。在晚期 BTC 中，推荐的治疗方案主要是使用顺铂/吉西他滨（CisGem）的双联化疗方案，5 年总生存率接近 5%，中位总生存期（mOS）不到一年。整体疗效不佳强调了需要个性化治疗。最近，全基因组和转录组测序突出了 BTC 亚型的多样性。根据定位，获得了不同的遗传改变，具有很高的潜在可操作性改变率。此后，针对 BTC 进行了靶向治疗和免疫治疗的测试，试图提出更个性化的治疗方法。本综述描述了晚期 BTC 患者目前已验证和正在开发的不同治疗选择。
