Emerging pharmacotherapeutic strategies for cholangiocarcinoma.

Introduction: Although of limited clinical benefit, chemotherapy represents the cornerstone of management for patients with inoperable cholangiocarcinoma. The literature on chemotherapy in advanced cholangiocarcinoma is difficult to interpret because of the heterogeneity of cholangiocarcinoma, the use of various chemotherapeutic agents in different combinations and dosing regimens, and the small size of existing patient cohorts.

Areas Covered: The authors discuss the major advances in the therapy of cholangiocarcinoma over the past decade. The audience will become familiarized with the contemporary medical management of cholangiocarcinoma and potentially useful agents/combinations for the therapy of this entity in the future.

Expert Opinion: In recent years, gemcitabine-based chemotherapy has improved overall survival in advanced cholangiocarcinoma. Although gemcitabine-cisplatin combination represents the largely accepted standard for biliary cancers at present, very few studies so far have looked at the results in cholangiocarcinoma patients only. Therefore, large multicenter, randomized clinical trials are essential to identify the optimal treatment strategy for this rare tumor. In addition, regimens employing gemcitabine with oxaliplatin and/or fluoropyrimidines have shown promising results and warrant additional investigation in cholangiocarcinoma. The efficacy of a variety of newer agents is also now being tested in clinical trials.