Clinical activity of regorafenib in PDGFRA-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 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.

This report describes the clinical efficacy of regorafenib in a patient with metastatic gastrointestinal stromal tumor bearing a mutation of the PDGFRA exon 18 gene.

Keywords::

• GIST
• PDGFRA mutation
• regorafenib

Financial & competing interests disclosure

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

No writing assistance was utilized in the production of this manuscript.

Informed consent disclosure

The authors state that they have obtained verbal and written informed consent from the patient/patients for the inclusion of their medical and treatment history within this case report.

Additional information

Funding

This work was supported by the charity: Mothers and Daughters, the University College London Hospitals, Experimental Cancer Medicine Center, a grant from CRUK (C6199/A16459) and a core center grant from the Wellcome Trust (075491/Z/04). The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.