Molecular-targeted therapy in ameloblastoma: a systematic review

Background: Ameloblastoma is a benign odontogenic tumor that predominantly occurs in the mandible and is frequently associated with the BRAF^V600E mutation, which activates the mitogen-activated protein kinase (MAPK) signaling pathway. These mutations indicate potential targets for molecular therapies. This systematic review evaluated the effectiveness of molecular-targeted therapies, particularly BRAF inhibitors such as dabrafenib and vemurafenib, in the treatment of ameloblastoma and their effects on clinical outcomes and quality of life. Methods: In accordance with PRISMA guidelines (PROSPERO: CRD42024627944), a comprehensive search of databases including PubMed/MEDLINE and Scopus identified 4,620 studies, of which eight case reports met the inclusion criteria for analysis. Results: The selected case reports involved patients aged 13 to 85, most of whom had experienced prior surgical recurrences. Treatment with BRAF/MEK (mitogen-activated protein kinase kinase) inhibitors resulted in significant tumor regression and improved quality of life, although some manageable side effects were observed. Conclusion: BRAF inhibitors demonstrate promising efficacy in the management of ameloblastoma, especially in patients harboring BRAF^V600E mutations. These therapies may reduce the necessity for extensive surgical procedures and enhance patient outcomes. Further research is needed to establish standardized treatment protocols and to assess long-term effects on recurrence and quality of life.