Division of Hematology and Oncology, University of Arizona Cancer Center, Tucson, AZ.
J Clin Oncol. 2022 Aug 20;40(24):2716-2734. doi: 10.1200/JCO.21.02576. Epub 2022 Jul 15.
Precision medicine has become a dominant theme in the treatment of biliary tract cancers (BTCs). Although prognosis remains poor, technologies for improved molecular characterization along with the US Food and Drug Administration approval of several targeted therapies have changed the therapeutic landscape of advanced BTC. The hallmark of BTC oncogenesis is chronic inflammation of the liver and biliary tract regardless of the anatomical subtype. Subtypes of BTC correspond to distinct molecular characteristics, making BTC a molecularly heterogenous collection of tumors. Collectively, up to 40% of BTCs harbor a potentially targetable molecular abnormality, and the National Comprehensive Cancer Network guidelines recommend molecular profiling for all patients with advanced BTC. Use of circulating tumor DNA, immunohistochemistry, and next-generation sequencing continues to expand the utility for biomarker-driven management and molecular monitoring of BTC. Improving outcomes using biomarker-agnostic treatment for nontargetable tumors also remains a priority, and combinational treatment strategies such as immune checkpoint inhibition plus chemotherapy hold promise for this subgroup of patients.
精准医学已成为治疗胆管癌（BTC）的主流主题。尽管预后仍然较差，但随着分子特征改善技术的进步以及美国食品和药物管理局批准了几种靶向治疗药物，BTC 的治疗前景已经发生了变化。BTC 发生的标志是无论解剖亚型如何，肝脏和胆道的慢性炎症。BTC 的亚型对应于不同的分子特征，使得 BTC 成为一种分子上异质性的肿瘤集合。总的来说，多达 40%的 BTC 存在潜在可靶向的分子异常，国家综合癌症网络指南建议对所有晚期 BTC 患者进行分子分析。循环肿瘤 DNA、免疫组织化学和下一代测序的应用不断扩大了用于生物标志物驱动管理和 BTC 分子监测的用途。使用无生物标志物的治疗方法来治疗不可靶向的肿瘤以改善预后仍然是一个优先事项，免疫检查点抑制加化疗等联合治疗策略为这组患者带来了希望。
