2020 Year in Review: Cholangiocarcinoma

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.
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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.
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Extracellular domain FGFR2 in-frame deletions might be a novel genomic alteration in intrahepatic CCA with potential clinical sensitivity to FGFR inhibitors.
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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.
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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.
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Retrospective analysis data suggest that second-line chemotherapy of capecitabine and cisplatin combination therapy was associated with modest efficacy in patients with advanced biliary tract cancer.
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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.
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Results of a phase 2 study indicate that mFOLFIRI was not superior to mFOLFOX in the second-line treatment of patients with biliary tract cancer.
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Results of the PK/PD analysis of the ClarIDHy study showed that the study dose of the IDH1 inhibitor ivosidenib resulted in good exposure and inhibition of oncometabolite D-2-HG.
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Results of the retrospective analysis of phase 2 study of the FGFR1-3 selective tyrosine kinase inhibitor infigratinib indicate that efficacy outcomes in patients with CCA and FGFR2 fusions were better with third- or later-line infigratinib therapy compared with standard second-line chemotherapy.
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