Molecular-targeted therapy in ameloblastoma: a systematic review

Ameloblastoma is a benign odontogenic tumor that predominantly occurs in the mandible and is frequently associated with the BRAFV600E 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.

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.

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.

BRAF inhibitors demonstrate promising efficacy in the management of ameloblastoma, especially in patients harboring BRAFV600E 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.