Safety of current treatments for advanced cholangiocarcinoma

Biliary tract cancer (BTC) originates from the biliary epithelium of the small ducts within the liver (intrahepatic cholangiocarcinoma, IHCC), the main ducts of the hilum (extrahepatic cholangiocarcinoma, EHCC), or in the gallbladder (gallbladder cancer, GC). Due to presentation with nonspecific symptoms as well as absence of screening, most patients present with advanced disease and unfavorable prognosis.

The ABC-02 trial established the current first-line chemotherapy with gemcitabine/platinum for advanced BTC in 2010. Since then, multiple therapies have become available exploring different targetable alterations, emphasizing the importance of molecular profiling in all patients with BTC as well as understanding the distinct toxicity profile associated with these therapies. Besides chemotherapy, immunotherapy as well as targeted therapies for FGFR2, IDH1, and HER2 will be discussed in this manuscript. We performed a non-systematic review, largely based on high-quality articles on the topic of interest with no predefined protocol.

The primary objective of this manuscript is to conduct a thorough review of diverse aspects related to the safety of systemic treatment in BTC. As the benefit of these therapies depends on compliance and/or tolerance, the authors aim to discuss different toxicity profiles and to provide insights into strategies for overcoming them.