title: Clinical activity of regorafenib inPDGFRA-mutated gastrointestinal stromal tumor.
abstract: Gastrointestinal stromal tumor (GIST) is the most frequent mesenchymal tumor of the gastrointestinal tract and one of the most frequent sarcoma. Mutually exclusiveKITandPDGFRAmutations 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 frequentPDGFRAmutation (D842V) is associated with primary resistance to imatinib. Data related to regorafenib efficacy inPDGFRA-mutated GIST are lacking. We report here a case report of a prolonged response with regorafenib in a patient with aPDGFRA-mutated GIST.
authors.affiliation: Department of Medical Oncology, Institut Bergonié, Bordeaux, France; Department of Medical Oncology, Institut Bergonié, Bordeaux, France.
authors.forename: Thomas
authors.initials: T
authors.lastname: Grellety
authors.affiliation: Department of Radiology, Institut Bergonié, Bordeaux, France; Department of Radiology, Institut Bergonié, Bordeaux, France.
authors.forename: Michèle
authors.initials: M
authors.lastname: Kind
authors.affiliation: Department of Pathology, Institut Bergonié, Bordeaux, France; Department of Pathology, Institut Bergonié, Bordeaux, France.
authors.forename: Jean-Michel
authors.initials: JM
authors.lastname: Coindre
authors.affiliation: Department of Medical Oncology, Institut Bergonié, Bordeaux, France; Department of Medical Oncology, Institut Bergonié, Bordeaux, France.
authors.forename: Antoine
authors.initials: A
authors.lastname: Italiano
keywords: GIST; PDGFRA mutation; regorafenib