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<p>Appendiceal Adenocarcinoma (AA) is a rare gastrointestinal cancer with no FDA-approved targeted therapies. Here, we retrospectively compare <i>BRAF-</i>mutant AA and colorectal cancer (CRC). <i>BRAF</i> mutation is rare in AA (3%). Unlike CRC, <i>BRAF</i><sup><i>V600E</i></sup> AA is not associated with poor prognosis, female sex, microsatellite instability, mucinous histology, or poor differentiation. In both cancers, <i>BRAF</i><sup><i>V600E</i></sup> but not atypical <i>BRAF</i> mutations are mutually exclusive with other Ras-activating mutations. BRAF<sup>V600E</sup> + EGFR inhibition shows efficacy in <i>BRAF</i><sup><i>V600E</i></sup> AA (disease control rate = 80%, median progression-free survival = 7.1 months).</p>

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

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年2月5日

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  • 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

摘要

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).