title: BRAF inhibitor treatment of primary BRAF-mutant ameloblastoma with pathologic assessment of response.
abstract: OBJECTIVE
  Molecular characterization of ameloblastoma has indicated a high frequency of driver mutations in BRAF and SMO. Preclinical data suggest that Food and Drug Administration-approved BRAF-targeted therapies may be immediately relevant for patients with ameloblastoma positive for the BRAF V600E mutation.
  
  
  METHODS
  A neoadjuvant treatment regime of dabrafenib was given to a patient with recurrent BRAF-mutant mandibular ameloblastoma. The patient subsequently underwent left mandible composite resection of the tumor and pathologic evaluation of treatment response.
  
  
  RESULTS
  The ameloblastoma had a slow but dramatic response with >90% tumor volume reduction. The inner areas of the tumor underwent degeneration and squamous differentiation, and intact ameloblastoma was present in the outer areas associated with bone.
  
  
  CONCLUSIONS
  Targeted neoadjuvant therapy for ameloblastoma may be useful in certain clinical settings of primary ameloblastoma. These might include tumors of advanced local stage when a neoadjuvant reduction could alter the extent of surgery and instances of local recurrence when surgical options are limited.
authors.affiliation: Department of Pathology, Stanford University School of Medicine, Stanford, CA, USA.
authors.forename: Serena
authors.initials: S
authors.lastname: Tan
authors.affiliation: Department of Pathology, Stanford University School of Medicine, Stanford, CA, USA.
authors.forename: Jonathan R
authors.initials: JR
authors.lastname: Pollack
authors.affiliation: Department of Surgery, Stanford University School of Medicine, Stanford, CA, USA.
authors.forename: Michael J
authors.initials: MJ
authors.lastname: Kaplan
authors.affiliation: Department of Medicine, Stanford University School of Medicine, Stanford, CA, USA.
authors.forename: A Dimitri
authors.initials: AD
authors.lastname: Colevas
authors.affiliation: Department of Pathology, Stanford University School of Medicine, Stanford, CA, USA. Electronic address: Rbwest@stanford.edu.
authors.forename: Robert B
authors.initials: RB
authors.lastname: West
mesh_terms: D000369:Aged, 80 and over; D000564:Ameloblastoma / Q000000981:diagnostic imaging / Q000188:drug therapy* / Q000235:genetics* / Q000601:surgery; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D006801:Humans; D007093:Imidazoles / Q000627:therapeutic use*; D008297:Male; D008339:Mandibular Neoplasms / Q000000981:diagnostic imaging / Q000188:drug therapy* / Q000235:genetics* / Q000601:surgery; D009154:Mutation; D020360:Neoadjuvant Therapy; D010091:Oximes / Q000627:therapeutic use*; D016133:Polymerase Chain Reaction; D048493:Proto-Oncogene Proteins B-raf / Q000235:genetics*; D014057:Tomography, X-Ray Computed
chemical_list: D000970:Antineoplastic Agents; D007093:Imidazoles; D010091:Oximes; D048493:Proto-Oncogene Proteins B-raf; C561627:dabrafenib