ESMO E-Learning: Latest Advances in the Treatment of Biliary Tract Cancer

In this E-Learning module, the author provides an overview of anatomical subgroups of biliary tract cancer, features the rarity but increasing incidence of intrahepatic cholangiocarcinoma, contextualizes the findings from the clinical trials in the adjuvant setting and from recent trials in advanced disease, elaborates molecular subgroups and matched treatments in selected sub-populations, and tackles the future research agenda.

The term “biliary tract cancer” encompasses rare cancers with poor prognosis, late diagnosis, high inoperability rate and high relapse rate. The author underlines that anatomical subgroups of biliary tract cancer remain relevant for surgery and loco-regional therapies. Chemotherapy is a cornerstone of treatment in adjuvant and advanced disease settings, but more effective agents are needed.

This module nicely demonstrates recent developments in targeted treatments for advanced biliary tract cancer illustrated by the dissection and shift from anatomical to molecular subgrouping. The author provides an overview of the genomic alterations landscape in advanced cholangiocarcinoma, their actionability and results from the clinical trials with targeted agents, underpinning mechanisms of resistance.  

Besides explaining targeted treatment options for IDH1, FGFR, BRAF V600E, HER2, and KRAS aberrations, the author also elaborates the results from trials with PD1 blockade in biliary tract cancer with mismatch-repair deficiency, combination of immune checkpoint inhibitor and chemotherapy, issues around liquid biopsy, and provides a treatment algorithm from the recently published clinical practice guideline.

The author underlines that future efforts are needed to explore the prognostic significance, the impact of prior or post-study therapy, primary and secondary resistance, and the development of rational treatment combinations.