Recent advances in the management of intrahepatic cholangiocarcinoma: the role of actionable mutations and targeted therapies.

Abstract

Introduction: The incidence of intrahepatic cholangiocarcinoma has continued to increase, with dismal rates of overall survival. While upfront surgery remains the mainstay of resectable intrahepatic cholangiocarcinoma, systemic therapy has gained increasing acceptance worldwide, especially for advanced and metastatic cholangiocarcinoma.

Areas covered: In this article, we review the different genetic mutations associated with cholangiocarcinoma, as well as recent advances made in the management of intrahepatic cholangiocarcinoma using therapies that directly target actionable mutations. We also review the clinical trials that led to the approval of these drugs, and the specific indications for their use.

Expert opinion: While significant progress has been made in identifying actionable mutations and developing drugs that target these mutations, several challenges exist in the management of intrahepatic cholangiocarcinoma using these targeted therapies. These challenges include issues with drug resistance, efficacy and cost. Furthermore, enrollment in clinical trials for cholangiocarcinoma is very limited and completed trials often lack the diversity needed to generalize their findings.