Cholangiocarcinoma

Patient-derived tumor organoids may have clinical application to predict drug responses in a personalized treatment setting; they have shown concordance with actionable genomic anchors and retrospective treatment outcomes.

Emerging data suggest that patient-derived 3-dimensional organoid model of intrahepatic CCA may allow personalized drug screening for active single agents or drug combinations similar to patient-derived xenograft models.

Real-world data indicate that genetic testing and personalized medicine is rarely applied in the community setting for patients with hepatobiliary and pancreatic cancers.

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.