We present 8-year follow-up on the first patient with stage 4 ameloblastoma carrying a BRAF V600E mutation treated with dual BRAF/MEK inhibition (BRAF/MEKi). He experienced a durable clinical response while on dabrafenib (BRAFi) and trametinib (MEKi) without toxicity nor evidence for drug-resistant tumor progression. He was asymptomatic when he self-discontinued therapy after 4 years of sustained clinical response. He did not return for follow-up until 2.5 years later with onset of painful mandibular tumor recurrence associated with recurrent bilateral lung metastases. He was rechallenged with dabrafenib/trametinib and experienced another prompt tumor response and remains in a second durable clinical remission (currently > 16 months) on continuous dual targeted therapy. We discuss the implications of this case study for future treatment strategies.
Keywords: Ameloblastoma; BRAF gene; Head and neck cancer; MEK gene; Oral cancer; Targeted therapy.
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