Successful Treatment of BRAF V600E-Mutant Intrahepatic Cholangiocarcinoma With Encorafenib, Binimetinib, and Cetuximab

BRAF mutations, particularly BRAF V600E, are rare but clinically significant molecular alterations in intrahepatic cholangiocarcinoma (IHCC), often linked to aggressive disease and poor prognosis. While targeted therapies have shown efficacy in BRAF-mutant colorectal cancer, their role in IHCC remains uncertain. This report describes the case of a 59-year-old woman with advanced BRAF V600E-mutated IHCC (cT3N3M1c, stage IVc) treated with encorafenib, binimetinib, and cetuximab. The BRAF V600E mutation was identified through next-generation sequencing. Initially, the patient was given gemcitabine, cisplatin, and durvalumab as first-line chemotherapy; however, her metastatic lesions progressed rapidly within one month. Due to the lack of response, second-line treatment with encorafenib, binimetinib, and cetuximab was started, resulting in a favorable initial response. Unfortunately, the disease progressed five months after initiating this regimen. This case highlights the potential benefit of combined BRAF/MEK/EGFR inhibition in BRAF V600E-mutated IHCC and underscores the need for further research to optimize treatment strategies for this patient population.

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Publication Details

Journal
Cureus
Published
2025-06-18
DOI
https://doi.org/10.7759/cureus.86263
Primary Topic
Cholangiocarcinoma and Gallbladder Cancer Studies
Type
article
Field-Weighted Citation Impact
0.00
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article

Successful Treatment of BRAF V600E-Mutant Intrahepatic Cholangiocarcinoma With Encorafenib, Binimetinib, and Cetuximab

Hiroki Nishikawa, Yoshinobu Hirose, Akiko Kagotani, Hiroki Yukami et al.
Cureus
Cholangiocarcinoma and Gallbladder Cancer Studies
article

Successful Treatment of BRAF V600E-Mutant Intrahepatic Cholangiocarcinoma With Encorafenib, Binimetinib, and Cetuximab

Hiroki Nishikawa, Yoshinobu Hirose, Akiko Kagotani, Hiroki Yukami, Elham Fakhrejahani, Hiroko Kodama, Toshifumi Yamaguchi, N. Matsuo, Yasutaka Nakazawa, Shin Kameishi
article en

Abstract

BRAF mutations, particularly BRAF V600E, are rare but clinically significant molecular alterations in intrahepatic cholangiocarcinoma (IHCC), often linked to aggressive disease and poor prognosis. While targeted therapies have shown efficacy in BRAF-mutant colorectal cancer, their role in IHCC remains uncertain. This report describes the case of a 59-year-old woman with advanced BRAF V600E-mutated IHCC (cT3N3M1c, stage IVc) treated with encorafenib, binimetinib, and cetuximab. The BRAF V600E mutation was identified through next-generation sequencing. Initially, the patient was given gemcitabine, cisplatin, and durvalumab as first-line chemotherapy; however, her metastatic lesions progressed rapidly within one month. Due to the lack of response, second-line treatment with encorafenib, binimetinib, and cetuximab was started, resulting in a favorable initial response. Unfortunately, the disease progressed five months after initiating this regimen. This case highlights the potential benefit of combined BRAF/MEK/EGFR inhibition in BRAF V600E-mutated IHCC and underscores the need for further research to optimize treatment strategies for this patient population.

CureusVol. 17(6)
Kyoto University (JP), Osaka International Cancer Institute (JP), Osaka University Hospital (JP), Osaka University of Pharmaceutical Sciences (JP)
Good health and well-being
Openalex Percentile: Top 76%
Cholangiocarcinoma and Gallbladder Cancer Studies
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Successful Treatment of BRAF V600E-Mutant Intrahepatic Cholangiocarcinoma With Encorafenib, Binimetinib, and Cetuximab — Hiroki Nishikawa, Yoshinobu Hirose, et al. · Cureus (2025) | TGRS Research Map | TGRS