Managing stage 4 ameloblastoma with dual BRAF/MEK inhibition: A case report with 8-year clinical follow-up

Oral Oncol. 2022 May:128:105854. doi: 10.1016/j.oraloncology.2022.105854. Epub 2022 Apr 18.

Abstract

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.

Publication types

  • Case Reports
  • Letter

MeSH terms

  • Ameloblastoma* / drug therapy
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use
  • Follow-Up Studies
  • Humans
  • Male
  • Melanoma* / drug therapy
  • Mitogen-Activated Protein Kinase Kinases / genetics
  • Mitogen-Activated Protein Kinase Kinases / therapeutic use
  • Mutation
  • Proto-Oncogene Proteins B-raf / genetics
  • Pyridones / therapeutic use

Substances

  • Pyridones
  • BRAF protein, human
  • Proto-Oncogene Proteins B-raf
  • Mitogen-Activated Protein Kinase Kinases