Clinical activity of regorafenib in PDGFRA-mutated gastrointestinal stromal tumor

Gastrointestinal stromal tumor (GIST) is the most frequent mesenchymal tumor of the gastrointestinal tract and one of the most frequent sarcoma. Mutually exclusive KIT and PDGFRA mutations are central events in GIST pathogenesis, and their understanding is crucial because specific treatment targetin...

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Detalles Bibliográficos
Autores principales: Grellety, Thomas, Kind, Michèle, Coindre, Jean-Michel, Italiano, Antoine
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Future Science Ltd 2015
Materias:
Case Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5137998/
https://www.ncbi.nlm.nih.gov/pubmed/28031906
http://dx.doi.org/10.4155/fso.15.33
Descripción
Sumario:Gastrointestinal stromal tumor (GIST) is the most frequent mesenchymal tumor of the gastrointestinal tract and one of the most frequent sarcoma. Mutually exclusive KIT and PDGFRA mutations are central events in GIST pathogenesis, and their understanding is crucial because specific treatment targeting oncogenic KIT and PDGFRA activation (especially imatinib) has become available. The most frequent PDGFRA mutation (D842V) is associated with primary resistance to imatinib. Data related to regorafenib efficacy in PDGFRA-mutated GIST are lacking. We report here a case report of a prolonged response with regorafenib in a patient with a PDGFRA-mutated GIST.

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