Anti-MAPK Targeted Therapy for Ameloblastoma: Case Report with a Systematic Review.

Simple Summary: Ameloblastoma is a type of tumor that usually forms in the jaw; while typically benign, it grows aggressively and often recurs after treatment. Traditional treatment involves extensive surgery, which can significantly affect a patient's quality of life. Recent research has focused on a new approach, targeting a specific cellular pathway known as the MAPK pathway, which appears to be involved in the development of these tumors. In this study, we reviewed the outcomes of 23 patients treated with this targeted therapy to assess its safety and effectiveness. The results were promising: most patients experienced significant tumor reduction, and the side effects were generally mild. This suggests that MAPK pathway inhibitors could be a viable alternative to surgery, potentially offering improved outcomes for patients with ameloblastoma, minimizing surgical risks, and preserving quality of life. This advancement could greatly impact the approach to treating this challenging condition. Ameloblastoma, a benign yet aggressive odontogenic tumor known for its recurrence and the severe morbidity from radical surgeries, may benefit from advancements in targeted therapy. We present a case of a 15-year-old girl with ameloblastoma successfully treated with targeted therapy and review the literature with this question: Is anti-MAPK targeted therapy safe and effective for treating ameloblastoma? This systematic review was registered in PROSPERO, adhered to PRISMA guidelines, and searched multiple databases up to December 2023, identifying 13 relevant studies out of 647 records, covering 23 patients treated with MAPK inhibitor therapies. The results were promising as nearly all patients showed a positive treatment response, with four achieving complete radiological remission and others showing substantial reductions in primary, recurrent, and metastatic ameloblastoma sizes. Side effects were mostly mild to moderate. This study presents anti-MAPK therapy as a significant shift from invasive surgical treatments, potentially enhancing life quality and clinical outcomes by offering a less invasive yet effective treatment alternative. This approach could signify a breakthrough in managing this challenging tumor, emphasizing the need for further research into molecular-targeted therapies. [ABSTRACT FROM AUTHOR]