BRAF mutant appendiceal adenocarcinoma differs from colorectal cancer but responds to BRAF-targeted therapy.

IF 6.8 1区 医学 Q1 ONCOLOGY NPJ Precision Oncology Pub Date : 2025-02-05 DOI:10.1038/s41698-025-00821-z
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
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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).

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BRAF突变阑尾腺癌不同于结直肠癌,但对BRAF靶向治疗有反应。
阑尾腺癌(AA)是一种罕见的胃肠道肿瘤,目前尚无fda批准的靶向治疗方法。在这里,我们回顾性比较braf突变AA和结直肠癌(CRC)。BRAF突变在AA中很少见(3%)。与CRC不同,BRAFV600E AA与预后不良、女性、微卫星不稳定、粘液组织学或分化不良无关。在这两种癌症中,BRAFV600E突变与其他ras激活突变相互排斥,但非典型BRAF突变除外。BRAFV600E + EGFR抑制对BRAFV600E AA有效(疾病控制率= 80%,中位无进展生存期= 7.1个月)。
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来源期刊
CiteScore
9.90
自引率
1.30%
发文量
87
审稿时长
18 weeks
期刊介绍: Online-only and open access, npj Precision Oncology is an international, peer-reviewed journal dedicated to showcasing cutting-edge scientific research in all facets of precision oncology, spanning from fundamental science to translational applications and clinical medicine.
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