Pattalachinti Vinay K, Haque Emaan, Yousef Mahmoud, Yousef Abdelrahman, Chowdhury Saikat, Overman Michael, Parseghian Christine M, Morris Van K, Kee Bryan, Huey Ryan W, Raghav Kanwal, Court Colin M, Shen John Paul
Department of Gastrointestinal Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX, USA.
The Joe R. and Teresa Lozano Long School of Medicine, University of Texas Health Science Center San Antonio, San Antonio, TX, USA.
NPJ Precis Oncol. 2025 Feb 5;9(1):38. doi: 10.1038/s41698-025-00821-z.
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, BRAF AA is not associated with poor prognosis, female sex, microsatellite instability, mucinous histology, or poor differentiation. In both cancers, BRAF but not atypical BRAF mutations are mutually exclusive with other Ras-activating mutations. BRAF + EGFR inhibition shows efficacy in BRAF AA (disease control rate = 80%, median progression-free survival = 7.1 months).
阑尾腺癌（AA）是一种罕见的胃肠道癌症，目前尚无美国食品药品监督管理局（FDA）批准的靶向治疗方法。在此，我们对BRAF突变型AA和结直肠癌（CRC）进行回顾性比较。BRAF突变在AA中较为罕见（3%）。与CRC不同，BRAF AA与预后不良、女性、微卫星不稳定性、黏液组织学或低分化无关。在这两种癌症中，BRAF而非非典型BRAF突变与其他Ras激活突变相互排斥。BRAF联合表皮生长因子受体（EGFR）抑制在BRAF AA中显示出疗效（疾病控制率=80%，无进展生存期中位数=7.1个月）。
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