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, BRAF V600E AA is not associated with poor prognosis, female s...
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| Format: | Article |
| Language: | English |
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Nature Portfolio
2025-02-01
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| Series: | npj Precision Oncology |
| Online Access: | https://doi.org/10.1038/s41698-025-00821-z |
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| author | 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 |
| author_facet | 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 |
| author_sort | Vinay K. Pattalachinti |
| collection | DOAJ |
| description | 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. BRAFV600E + EGFR inhibition shows efficacy in BRAF V600E AA (disease control rate = 80%, median progression-free survival = 7.1 months). |
| format | Article |
| id | doaj-art-f055df36a51941068ec70c80313917be |
| institution | Kabale University |
| issn | 2397-768X |
| language | English |
| publishDate | 2025-02-01 |
| publisher | Nature Portfolio |
| record_format | Article |
| series | npj Precision Oncology |
| spelling | doaj-art-f055df36a51941068ec70c80313917be2025-02-09T12:09:24ZengNature Portfolionpj Precision Oncology2397-768X2025-02-01911710.1038/s41698-025-00821-zBRAF mutant appendiceal adenocarcinoma differs from colorectal cancer but responds to BRAF-targeted therapyVinay K. Pattalachinti0Emaan Haque1Mahmoud Yousef2Abdelrahman Yousef3Saikat Chowdhury4Michael Overman5Christine M. Parseghian6Van K. Morris7Bryan Kee8Ryan W. Huey9Kanwal Raghav10Colin M. Court11John Paul Shen12Department of Gastrointestinal Medical Oncology, The University of Texas MD Anderson Cancer CenterDepartment of Gastrointestinal Medical Oncology, The University of Texas MD Anderson Cancer CenterDepartment of Gastrointestinal Medical Oncology, The University of Texas MD Anderson Cancer CenterInternal Medicine Department, University of New Mexico HospitalDepartment of Gastrointestinal Medical Oncology, The University of Texas MD Anderson Cancer CenterDepartment of Gastrointestinal Medical Oncology, The University of Texas MD Anderson Cancer CenterDepartment of Gastrointestinal Medical Oncology, The University of Texas MD Anderson Cancer CenterDepartment of Gastrointestinal Medical Oncology, The University of Texas MD Anderson Cancer CenterDepartment of Gastrointestinal Medical Oncology, The University of Texas MD Anderson Cancer CenterDepartment of Gastrointestinal Medical Oncology, The University of Texas MD Anderson Cancer CenterDepartment of Gastrointestinal Medical Oncology, The University of Texas MD Anderson Cancer CenterDepartment of Surgical Oncology and Endocrine Surgery, Mays Cancer Center, University of Texas Health San AntonioDepartment of Gastrointestinal Medical Oncology, The University of Texas MD Anderson Cancer CenterAbstract 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. BRAFV600E + EGFR inhibition shows efficacy in BRAF V600E AA (disease control rate = 80%, median progression-free survival = 7.1 months).https://doi.org/10.1038/s41698-025-00821-z |
| spellingShingle | 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 BRAF mutant appendiceal adenocarcinoma differs from colorectal cancer but responds to BRAF-targeted therapy npj Precision Oncology |
| title | BRAF mutant appendiceal adenocarcinoma differs from colorectal cancer but responds to BRAF-targeted therapy |
| title_full | BRAF mutant appendiceal adenocarcinoma differs from colorectal cancer but responds to BRAF-targeted therapy |
| title_fullStr | BRAF mutant appendiceal adenocarcinoma differs from colorectal cancer but responds to BRAF-targeted therapy |
| title_full_unstemmed | BRAF mutant appendiceal adenocarcinoma differs from colorectal cancer but responds to BRAF-targeted therapy |
| title_short | BRAF mutant appendiceal adenocarcinoma differs from colorectal cancer but responds to BRAF-targeted therapy |
| title_sort | braf mutant appendiceal adenocarcinoma differs from colorectal cancer but responds to braf targeted therapy |
| url | https://doi.org/10.1038/s41698-025-00821-z |
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