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

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

doi:10.1038/s41698-025-00821-z

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

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

Research output: Contribution to journal › Article › peer-review

1 Scopus citations

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, BRAF^V600E AA is not associated with poor prognosis, female sex, microsatellite instability, mucinous histology, or poor differentiation. In both cancers, BRAF^V600E but not atypical BRAF mutations are mutually exclusive with other Ras-activating mutations. BRAF^V600E + EGFR inhibition shows efficacy in BRAF^V600E AA (disease control rate = 80%, median progression-free survival = 7.1 months).

Original language	English (US)
Article number	38
Journal	npj Precision Oncology
Volume	9
Issue number	1
DOIs	https://doi.org/10.1038/s41698-025-00821-z
State	Published - Dec 2025
Externally published	Yes

ASJC Scopus subject areas

Oncology
Cancer Research

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10.1038/s41698-025-00821-z

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Pattalachinti, V. K., Haque, E., Yousef, M., Yousef, A., Chowdhury, S., Overman, M., Parseghian, C. M., Morris, V. K., Kee, B., Huey, R. W., Raghav, K., Court, C. M., & Shen, J. P. (2025). BRAF mutant appendiceal adenocarcinoma differs from colorectal cancer but responds to BRAF-targeted therapy. npj Precision Oncology, 9(1), Article 38. https://doi.org/10.1038/s41698-025-00821-z

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title = "BRAF mutant appendiceal adenocarcinoma differs from colorectal cancer but responds to BRAF-targeted therapy",

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).",

author = "Pattalachinti, \{Vinay K.\} and Emaan Haque and Mahmoud Yousef and Abdelrahman Yousef and Saikat Chowdhury and Michael Overman and Parseghian, \{Christine M.\} and Morris, \{Van K.\} and Bryan Kee and Huey, \{Ryan W.\} and Kanwal Raghav and Court, \{Colin M.\} and Shen, \{John Paul\}",

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doi = "10.1038/s41698-025-00821-z",

language = "English (US)",

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AU - Pattalachinti, Vinay K.

AU - Haque, Emaan

AU - Yousef, Mahmoud

AU - Yousef, Abdelrahman

AU - Chowdhury, Saikat

AU - Overman, Michael

AU - Parseghian, Christine M.

AU - Morris, Van K.

AU - Kee, Bryan

AU - Huey, Ryan W.

AU - Raghav, Kanwal

AU - Court, Colin M.

AU - Shen, John Paul

PY - 2025/12

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N2 - 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).

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

Abstract

ASJC Scopus subject areas

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