Up to 13% of KIT and PDGFRA wild-type (WT) gastrointestinal stromal tumours (GIST) harbour a BRAF mutation, mostly involving the exon 15 V600E hot spot. Even if BRAF mutation is recognized as druggable target in other solid tumours, currently advanced BRAF-mutant GIST share the same therapeutical algorithm of KIT/PDGFRA mutants. We report a complete radiological response in a 51-year-old woman with V600E BRAF-mutated metastatic GIST who was treated with regorafenib (REG) as first-line therapy. REG represents the standard third-line therapy for advanced GIST patients progressing on or failing to respond to imatinib and sunitinib. However, according to its wide spectrum of action, with MAPK signalling pathway blockade at different levels, metastatic KIT/PDGFRA WT, including BRAF mutants, may benefit from REG upfront in first line.

Margherita Nannini, D.S.V. (2020). Complete radiological response to first-line regorafenib in a patient with abdominal relapse of BRAF V600E mutated GIST. THERAPEUTIC ADVANCES IN GASTROENTEROLOGY, 13, 1-4 [10.1177/1756284820927305].

Complete radiological response to first-line regorafenib in a patient with abdominal relapse of BRAF V600E mutated GIST

Margherita Nannini;Elisa Gruppioni;Maristella Saponara;Maria Abbondanza Pantaleo;
2020

Abstract

Up to 13% of KIT and PDGFRA wild-type (WT) gastrointestinal stromal tumours (GIST) harbour a BRAF mutation, mostly involving the exon 15 V600E hot spot. Even if BRAF mutation is recognized as druggable target in other solid tumours, currently advanced BRAF-mutant GIST share the same therapeutical algorithm of KIT/PDGFRA mutants. We report a complete radiological response in a 51-year-old woman with V600E BRAF-mutated metastatic GIST who was treated with regorafenib (REG) as first-line therapy. REG represents the standard third-line therapy for advanced GIST patients progressing on or failing to respond to imatinib and sunitinib. However, according to its wide spectrum of action, with MAPK signalling pathway blockade at different levels, metastatic KIT/PDGFRA WT, including BRAF mutants, may benefit from REG upfront in first line.
2020
Margherita Nannini, D.S.V. (2020). Complete radiological response to first-line regorafenib in a patient with abdominal relapse of BRAF V600E mutated GIST. THERAPEUTIC ADVANCES IN GASTROENTEROLOGY, 13, 1-4 [10.1177/1756284820927305].
Margherita Nannini, Di Scioscio Valerio, Elisa Gruppioni, Annalisa Altimari, Benedetta Chiusole, Maristella Saponara, Maria Abbondanza Pantaleo, Anton...espandi
File in questo prodotto:
File Dimensione Formato  
Nannini Ther Advanc Gastroenterol 2020.pdf

accesso aperto

Tipo: Versione (PDF) editoriale
Licenza: Licenza per Accesso Aperto. Creative Commons Attribuzione - Non commerciale (CCBYNC)
Dimensione 642.81 kB
Formato Adobe PDF
642.81 kB Adobe PDF Visualizza/Apri

I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.

Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11585/766685
Citazioni
  • ???jsp.display-item.citation.pmc??? ND
  • Scopus 2
  • ???jsp.display-item.citation.isi??? 2
social impact