Abstract

Introduction: Cholangiocarcinomas (CCA) are rare, aggressive tumors often diagnosed in advanced stages with limited evidence guiding therapy on progression. Case Report: We report a case of advanced CCA with rapid and aberrant progression, refractory to multiple lines of therapy, that resulted in severe hepatic dysfunction secondary to tumor burden with a BRAF V600E mutation and high tumor proportion score (TPS) of 99%. To our knowledge, this is the first reported use of BRAF/MEK inhibition to target BRAF V600E in a patient with severe hepatic dysfunction leading to rapid normalization of the patient’s liver dysfunction within days. No adverse events were recorded during either initial titration or maintenance periods. Programmed death-1 (PD-1) inhibitor was added to BRAF/MEK inhibition, and the patient continues to have clinical therapeutic response. Conclusion: This case highlights the use of BRAF/MEK inhibition in CCA with BRAF V600E mutations in hepatic dysfunction due to tumor burden and the role of combining immune checkpoint inhibitors.

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