The emerging role of immunotherapy in biliary tract cancer: a review of new evidence and predictive biomarkers

Biliary tract cancers (BTCs) are frequently diagnosed in advanced stages and are highly lethal. Immunotherapy may play a role in the treatment of these patients. Promising results come from monotherapy or combination therapy studies in pretreated patients. In addition, several studies have demonstrated the safety and efficacy of immune checkpoint inhibitors (ICIs) in combination with chemotherapy in treatment-naive patients. Numerous biomarkers have been investigated to define their predictive role in response to ICIs. However, the full extent of the benefit of immunotherapies has not yet been fully established and, except for high microsatellite instability status, no other biomarkers were uniquely predictive of response to ICIs.

Automated summary

Biliary tract cancers (BTCs) are often diagnosed at advanced stages with high fatality rates. Immunotherapy, particularly in combination with chemotherapy and immune checkpoint inhibitors, has shown promise in the treatment of these patients. Although several biomarkers have been investigated for predicting the response to immunotherapies, only high microsatellite instability status has been identified as a unique predictive marker.