Cholangiocarcinoma (CCA) is the second most common primary liver cancer and accounts for approximately 10–15% of all primary liver cancers. CCA is subdivided into intrahepatic CCA (iCCA), arising from bile ductules proximal to the second-order bile ducts; perihilar CCA (pCCA), arising in the right and/or left hepatic duct and/or at their junction; and distal CCA (dCCA), arising from the epithelium distal to the insertion of the cystic duct. pCCA and dCCA are collectively referred to extrahepatic CCA (eCCA). For patients with early-stage CCA, the treatment usually consists of a surgical resection followed by adjuvant chemotherapy, while systemic chemotherapy is the standard first-line treatment for patients with advanced stage disease. However, given the fact that nearly 40% of patients with biliary tract cancer harbour genetic alterations which are potential targets for precision medicine, molecular analysis should be carried out before or during firstline therapy to evaluate options for second and higher lines of treatment as early as possible in advanced disease.1 This review aims to discuss the most promising therapeutic molecular targets for CCA and the targeted agents that are available in this setting.
