2020 Year in Review: Cholangiocarcinoma

Preclinical data indicate that genetic analysis of ctDNA from patients with advanced CCA harboring IDH mutations was concordant with those of tissue samples and could detect emerging mutations associated with treatment resistance.
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Preclinical evidence presented at the AACR meeting implicates PKN2 signaling in resistance mechanisms in patients with FGFR2 fusion–positive intrahepatic CCA.
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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.
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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.
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Real-world data indicate that genetic testing and personalized medicine is rarely applied in the community setting for patients with hepatobiliary and pancreatic cancers.
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Retrospective chart review data indicate that intrahepatic CCA and extrahepatic CCA exhibit disparate clinical features and molecular profile, and divergent treatment patterns.
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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.
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The multidrug combination of toripalimab, lenvatinib plus chemotherapy with gemcitabine and oxaliplatin showed promising efficacy and tolerability in patients with intrahepatic CCA.
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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.
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Assessment of global epidemiologic trends in incidence of biliary tract cancers (BTCs) showed higher BTC incidence in Asian versus Western countries as well as intraregional variations within countries, which is consistent with previous reports.
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