title: Rapid and clinically significant response to masitinib in the treatment of mucosal primary esophageal melanoma with somatic KIT exon 11 mutation involving brain metastases: A case report.
abstract: BACKGROUND
  Malignant melanoma in the gastrointestinal tract may be primary or metastatic. Mucosal melanoma is a quite rare and aggressive disease, growing hidden and diagnosed with a certain delay which makes treatment difficult.
  
  
  METHODS
  The authors present the first patient with c-kit exon 11 mutated primary esophageal melanoma treated with oral tyrosine kinase inhibitor masitinib. A 55-year-old-man presented with esophageal melanoma metastising into visceral organs and to the brain. The patient showed objective and clinical significant therapeutic response to masitinib. After initiation of masitinib, dysphagia and odynophagia disappeared within 1 week. Following 1 month of treatment, computed tomography showed a regression in the number and size of brain metastatic lesions and regression in visceral lesions. This therapeutic response, despite the aggressive disease on treatment initiation, effectively enabled the patient to have 6 months of quality life.
  
  
  CONCLUSIONS
  This report corroborates the plausibility of treating advanced melanoma carrying a mutation of KIT with masitinib. It also raises the question of masitinib treatment beyond progression. Additionally, the observed masitinib treatment effect on the brain suggests accumulation of therapeutically relevant concentration of masitinib in the central nervous system. This observation has possible ramifications for treatment of intracranial neoplasms.
authors.affiliation: Department of Clinical Oncology, County Hospital, Nachod, Czech Republic.
authors.forename: Jarmila
authors.initials: J
authors.lastname: Prosvicova
authors.affiliation: Department of Clinical Oncology, County Hospital, Nachod, Czech Republic.|Department of Clinical Microbiology, Faculty of Medicine in Hradec Kralove, Charles University in Prague, Hradec Kralove.
authors.forename: Sarka
authors.initials: S
authors.lastname: Lukesova
authors.affiliation: Department of Clinical Oncology and Radiotherapy, University Hospital in Hradec Kralove.
authors.forename: Jindrich
authors.initials: J
authors.lastname: Kopecky
authors.affiliation: Department of Clinical Oncology and Radiotherapy, University Hospital in Hradec Kralove.
authors.forename: Jiri
authors.initials: J
authors.lastname: Grim
authors.affiliation: Endoscopic Centre, County Hospital, Nachod.
authors.forename: Zdenek
authors.initials: Z
authors.lastname: Papik
authors.affiliation: Endoscopic Centre, County Hospital, Nachod.
authors.forename: Renata
authors.initials: R
authors.lastname: Kolarova
authors.affiliation: Department of Histopathology, County Hospital Nachod.
authors.forename: Blanka
authors.initials: B
authors.lastname: Navratilova
authors.affiliation: Inserm, U1068, CRCM (Signaling, Hematopoiesis and Mechanism of Oncogenesis); Institut Paoli-Calmettes, Aix-Marseille Univ, F-13284; CNRS, UMR7258, Marseille, F-13009, France.|AB Science, Paris, France.
authors.forename: Patrice
authors.initials: P
authors.lastname: Dubreuil
authors.affiliation: AB Science, Paris, France.
authors.forename: Julie
authors.initials: J
authors.lastname: Agopian
authors.affiliation: AB Science, Paris, France.
authors.forename: Colin
authors.initials: C
authors.lastname: Mansfield
authors.affiliation: AB Science, Paris, France.
authors.forename: Alan
authors.initials: A
authors.lastname: Moussy
authors.affiliation: AB Science, Paris, France.|Hospital Necker, Paris; CNRS UMR 8147, Department of Hematology, Universite Paris V Rene Descartes, Paris; Institut Imagine, Universite Sorbonne Paris cite, Paris, France.
authors.forename: Olivier
authors.initials: O
authors.lastname: Hermine
mesh_terms: D001549:Benzamides; D001932:Brain Neoplasms / Q000188:drug therapy / Q000556:secondary*; D004938:Esophageal Neoplasms / Q000188:drug therapy*; D005091:Exons; D017809:Fatal Outcome; D006801:Humans; D008297:Male; D008545:Melanoma / Q000188:drug therapy / Q000556:secondary*; D008875:Middle Aged; D009154:Mutation / Q000235:genetics; D010880:Piperidines; D047428:Protein Kinase Inhibitors / Q000627:therapeutic use*; D019009:Proto-Oncogene Proteins c-kit / Q000235:genetics*; D011725:Pyridines; D013844:Thiazoles / Q000627:therapeutic use*
chemical_list: D001549:Benzamides; D010880:Piperidines; D047428:Protein Kinase Inhibitors; D011725:Pyridines; D013844:Thiazoles; D019009:Proto-Oncogene Proteins c-kit; C526575:masitinib
keywords: esophageal melanoma; masitinib; targeted therapy