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Case Report
1 Department of Oncology, Johns Hopkins Hospital, 1800 Orleans St., Baltimore, MD 21287, United States
2 Department of Pathology, Johns Hopkins Hospital, 1800 Orleans St., Baltimore, MD 21287, United States
Address correspondence to:
Chester Kao
MD, Johns Hopkins Sidney Kimmel Comprehensive Cancer Center, 401 N Broadway St., Baltimore, MD 21287,
United States
Message to Corresponding Author
Article ID: 100117Z10AB2023
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.
Keywords: BRAF/MEK inhibition, BRAF V600E, Cholangiocarcinoma, Immune-checkpoint inhibition, Targeted therapy
Aanika Balaji - Acquisition of data, Analysis of data, Interpretation of data, Drafting the article, Revising it critically for important intellectual content, Final approval of the version to be published
Kayla Garzio - Substantial contributions to conception and design, Acquisition of data, Analysis of data, Interpretation of data, Drafting the article, Revising it critically for important intellectual content, Final approval of the version to be published
Kiyoko Oshima - Acquisition of data, Revising it critically for important intellectual content, Final approval of the version to be published
Rachel Klein - Acquisition of data, Revising it critically for important intellectual content, Final approval of the version to be published
Nilofer Azad - Substantial contributions to conception and design, Acquisition of data, Analysis of data, Interpretation of data, Revising it critically for important intellectual content, Final approval of the version to be published
Chester Kao - Substantial contributions to conception and design, Acquisition of data, Analysis of data, Interpretation of data, Drafting the article, Revising it critically for important intellectual content, Final approval of the version to be published
Guaranter of SubmissionThe corresponding author is the guarantor of submission.
Source of SupportNone
Consent StatementAll institutional IRB guidelines were followed, and patient data were de-identified prior to publication.
Data AvailabilityAll relevant data are within the paper and its Supporting Information files.
Conflict of InterestAuthors declare no conflict of interest.
Copyright© 2023 Aanika Balaji et al. This article is distributed under the terms of Creative Commons Attribution License which permits unrestricted use, distribution and reproduction in any medium provided the original author(s) and original publisher are properly credited. Please see the copyright policy on the journal website for more information.