Biliary cancer is a group of rare tumors associated with a poor prognosis. About 40% of patients have genetic alterations, the most common of which are FGFR2 fusions and IDH1 mutations in approximately 15% of intrahepatic cholangiocarcinomas. This work is devoted to studying the effectiveness and application in real practice of molecular-targeted therapy (MTT) for biliary cancer.

OBJECTIVE

To evaluate the effectiveness of MTT for biliary cancer in the 2nd and subsequent lines in real clinical practice.

MATERIAL AND METHODS

This work is based on the analysis of retrospective and prospective data from the examination and treatment of 106 patients with biliary cancer, observed and treated at the National Medical Research Center for Oncology. N.N. Blokhin Russian Academy of Medical Sciences from 2015 to 2023. Patients with a histologically confirmed diagnosis underwent a molecular genetic study. Individuals with identified clinically significant genetic alterations, depending on the availability/availability of drugs, received MNT.

RESULTS

A genetic study of the molecular profile of the tumor was performed in 106 patients. Alterations were identified in 64 (60.4%) of them. The use of MNT in the 2nd line in patients with clinically significant alterations allows achieving progression-free survival of 14 months, which is statistically significantly better compared to the use of chemotherapy regimens. Overall survival (OS) of patients who received MNT was 35 months, which is statistically significantly longer than patients with alterations without the use of MNT (OS 16.5 months; p=0.0007) and without alterations (OS 19.1 months; p=.0002).

CONCLUSION

Biliary cancer remains a disease with a poor prognosis. Molecular profiling should be performed in patients with biliary cancer to decide on further treatment options 2nd line because this strategy has been clearly shown to provide clinical benefit to patients.