BRAF inhibitor treatment of primary BRAF-mutant ameloblastoma with pathologic assessment of response.

BRAF inhibitor treatment of primary BRAF-mutant ameloblastoma with pathologic assessment of response.

Oral Surg Oral Med Oral Pathol Oral Radiol. 2016 Feb 23;

Authors: Tan S, Pollack JR, Kaplan MJ, Colevas AD, West RB

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.

PMID: 27209484 [PubMed - as supplied by publisher]

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