Published online Jul 15, 2017. doi: 10.4251/wjgo.v9.i7.268
Peer-review started: October 7, 2016
First decision: December 1, 2016
Revised: January 5, 2017
Accepted: March 23, 2017
Article in press: March 24, 2017
Published online: July 15, 2017
Cholangiocarcinoma (CCA) is a rare cancer arising from the biliary tree with a poor prognosis and limited therapeutic options. Recent large scale molecular characterisation studies have identified recurrent genetic alterations in CCA which may be amenable to therapeutic targeting. In this review we explore the genomic landscape of CCA and examine results from trials of molecularly targeted agents and immunotherapy in this disease. Challenges in CCA diagnosis, treatment and trial design are discussed and we reflect on future directions which may lead to improved outcomes for CCA patients.
Core tip: Cholangiocarcinoma (CCA) is a clinically challenging malignancy; it is rare, molecularly heterogenous and associated with a poor prognosis. Here we review recent data on the genomic landscape of CCA and highlight the results of clinical trials using targeted agents and immunotherapy. We find a number of promising therapeutic agents in development and discuss strategies to improve diagnosis and outcomes in this patient group.