Precision therapy for BRAF V600E-mutated ameloblastoma: Systematic review insights

Although rare, ameloblastoma is a locally aggressive odontogenic tumor, with recent studies identifying the BRAF V600E mutation as its most common molecular alteration driving MAPK pathway activation. To elucidate its clinicopathological and therapeutic significance, we conducted a systematic review and meta-analysis of studies reporting both the prevalence of BRAF V600E and treatment outcomes in mutated cases. A total of 36 studies comprising 2034 patients were included, yielding a pooled mutation prevalence of 72.1% (95% CI: 67.1-76.6%), with marked enrichment in mandibular tumors (77.5%, 95% CI: 72.7-81.6%). For treatment outcomes, 12 studies involving 21 patients with BRAF V600E-mutated ameloblastoma who received targeted therapy were analyzed. Most patients demonstrated a partial or complete radiological response, suggesting promising but preliminary therapeutic efficacy. This systematic review indicates that BRAF V600E represents the predominant oncogenic driver in ameloblastoma and a promising therapeutic target. Integration of molecular testing and targeted therapy into clinical management may reduce surgical morbidity, warranting validation in prospective trials.