BRA Mutant Appendiceal Adenocarcinoma Differs From Colorectal Cancer but Responds to BRAF-Targeted Therapy

Authors

Vinay K Pattalachinti
Emaan Haque
Mahmoud Yousef
Abdelrahman Yousef
Saikat Chowdhury
Michael Overman
Christine M Parseghian
Van K Morris
Bryan Kee
Ryan W Huey
Kanwal Raghav
Colin M Court
John Paul Shen

Publication Date

2-5-2025

Journal

NPJ Precision Oncology

Abstract

Appendiceal Adenocarcinoma (AA) is a rare gastrointestinal cancer with no FDA-approved targeted therapies. Here, we retrospectively compare BRAF-mutant AA and colorectal cancer (CRC). BRAF mutation is rare in AA (3%). Unlike CRC, BRAFV600E AA is not associated with poor prognosis, female sex, microsatellite instability, mucinous histology, or poor differentiation. In both cancers, BRAFV600E but not atypical BRAF mutations are mutually exclusive with other Ras-activating mutations. BRAFV600E + EGFR inhibition shows efficacy in BRAFV600E AA (disease control rate = 80%, median progression-free survival = 7.1 months).

DOI

10.1038/s41698-025-00821-z

PMID

39910160

PMCID

PMC11799341

PubMedCentral® Posted Date

2-5-2025

PubMedCentral® Full Text Version

Post-print

Published Open-Access

yes