2020 Year in Review - Cholangiocarcinoma

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.

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.

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.

Retrospective analysis data indicate that adjuvant chemoradiotherapy was associated with improved survival compared with chemotherapy alone in patients with extrahepatic CCA.

Preliminary data suggest that dual blockade with ipilimumab and nivolumab might have antitumor activity in a subset of patients with microsatellite-stable biliary tract cancer.

Phase 2 study results suggest that the addition of nivolumab and ipilimumab to chemotherapy as first-line therapy did not provide additional PFS benefit compared to standard-of-care chemotherapy in patients with advanced biliary tract cancer.

Comprehensive genomic profiling of tumor tissue and cfDNA of patients enrolled in the phase 2 study of infigratinib underscored the heterogeneity of CCA and the potential clinical utility of cfDNA to identify FGFR2 fusions.

Meta-analysis identifies upregulation of several novel genes that have not been previously described, and suggests the potential role of ERBB2 and extracellular genes in the pathogenesis of CCA.

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

Comprehensive genomic profiling data indicate that genetic profile of primary intrahepatic CCA was significantly different from metastatic intrahepatic CCA, with different rates of potentially targetable genetic alterations.