Cholangiocarcinoma

Updated data from the TOPAZ-1 study revealed improved median overall survival for the full patient population and no new safety signals.

Final analysis of the phase 2 FIGHT-202 trial demonstrated durable response and a tolerable safety profile for pemigatinib in patients with metastatic cholangiocarcinoma.

The BilT-04 study revealed that the addition of CPI-613 to gemcitabine/cisplatin was well-tolerated with potential activity in patients with advanced biliary tract cancers.

Updated FOENIX-CCA2 trial results were found to be consistent with the primary analysis, indicating durable efficacy and continued tolerability of futibatinib in patients with intrahepatic cholangiocarcinoma harboring FGFR2 fusions/rearrangements.

Phase 3 TOPAZ-1 clinical trial established durvalumab plus gemcitabine/cisplatin as a viable first-line standard-of-care treatment regimen for advanced biliary tract cancer.

A comprehensive genomic and immune characterization of IDH-mutated and wild-type intrahepatic CCA revealed significant differences in genetic alterations.

Cholangiocarcinoma (CCA) is an aggressive cancer of the bile duct that forms inside the liver (ie, intrahepatic) or outside the liver (ie, extrahepatic, including perihilar and distal tumors). Individuals with colitis or certain liver diseases may have an increased risk for CCA. Although the precise incidence of CCA is unknown, an estimated 8000 new cases of CCA are diagnosed annually in the United States. It is likely, however, that this number is higher, because CCA is difficult to diagnose and may be misclassified at times.

Findings from a retrospective analysis support the use of both tissue and liquid biopsy biomarker testing to guide therapy selection in patients with advanced CCA, particularly when tissue may not be readily available.

Results of a phase 2 study indicate that toripalimab and lenvatinib in combination with GemOx chemotherapy provide antitumor activity and reasonable tolerability in patients with advanced intrahepatic CCA.