Facts and hopes in the systemic therapy of biliary tract carcinomas

Abstract

Biliary tract cancers (BTC) are a heterogeneous group of cancers that continue to present a particularly poor prognosis. Treatment of BTC is rapidly evolving but faces many challenges to improving patient outcomes and maximising benefit from treatment. Only a minority of patients are diagnosed with early-stage disease, and are suitable for curative resection. Current surgical strategies are limited by a high relapse rate and despite extensive efforts focused on adjuvant strategies, development of more effective adjuvant strategies remains a challenge. In addition, the role of locoregional strategies, liver transplant and neoadjuvant treatment remains unclear. Systemic treatment in the advanced setting is based on three main pillars. First, cytotoxic chemotherapy options, second, the addition of immunotherapy to chemotherapy and third, targeted therapies. The role of targeted therapies is oriented by many promising targets including IDH1 mutations, FGFR2 fusions, BRAF-V600E mutations, and HER2 amplifications. The aim of this review is to provide an overview of current facts and future hopes in the management of BTC, including an overview of the unmet need, and a particular focus on systemic therapies.