Cholangiocarcinoma

Retrospective chart review data indicate that intrahepatic CCA and extrahepatic CCA exhibit disparate clinical features and molecular profile, and divergent treatment patterns.

The multidrug combination of toripalimab, lenvatinib plus chemotherapy with gemcitabine and oxaliplatin showed promising efficacy and tolerability in patients with intrahepatic CCA.

The ongoing PROOF trial (NCT03773302) is evaluating the efficacy and safety of infigratinib versus gemcitabine plus cisplatin as frontline therapy in patients with advanced CCA harboring FGFR2 gene rearrangements.

Genomic data analysis of publicly available data supports the high prevalence of potentially actionable mutations in patients with CCA, supporting use of personalized therapies alone or in combination with chemotherapy.

Post-hoc analysis of the phase 2 FIGHT-202 study showed that second-line pemigatinib treatment resulted in a numerically longer PFS than standard systemic second-line treatment in patients with previously treated advanced/metastatic CCA.

Extracellular domain FGFR2 in-frame deletions might be a novel genomic alteration in intrahepatic CCA with potential clinical sensitivity to FGFR inhibitors.

Preliminary results of the FOENIX-CCA2 study show that futibatinib may be effective and well-tolerated in patients with locally advanced or metastatic unresectable intrahepatic CCA harboring FGFR2 gene fusions following failure of prior lines of chemotherapy.

Results of the global phase 2 FOENIX-CCA2 trial support the efficacy and safety of futibatinib treatment in patients with locally advanced or metastatic unresectable intrahepatic CCA and FGFR2 fusions or rearrangements, and indicate that futibatinib does not adversely affect patients’ quality of life.

Results of a comprehensive genomic profiling study indicate differences in genomic alterations detected from primary tumor, metastatic tumor tissue, and liquid biopsy in patients with intrahepatic CCA; IDH1 and FGFR2 genomic alterations are detectable in liquid biopsy, with potential practice-changing implications in clinical practice.