Abramson Zachary, Dayton Orrin L, Drane Walter E, Mendenhall William M, Kaye Frederic J
Department of Diagnostic Imaging, St. Jude Children's Research Hospital, Memphis, TN, United States.
Departments of Radiology, University of Florida College of Medicine, Gainesville, FL, United States.
Oral Oncol. 2022 May;128:105854. doi: 10.1016/j.oraloncology.2022.105854. Epub 2022 Apr 18.
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.
我们报告了首例携带BRAF V600E突变的4期成釉细胞瘤患者接受BRAF/MEK双重抑制治疗（BRAF/MEKi）的8年随访情况。在使用达拉非尼（BRAFi）和曲美替尼（MEKi）治疗期间，他经历了持久的临床反应，且无毒性反应，也没有出现耐药性肿瘤进展的迹象。在持续临床反应4年后自行停药时，他没有任何症状。2.5年后，他因下颌肿瘤复发伴双侧肺转移复发且疼痛而前来复诊。他再次接受达拉非尼/曲美替尼治疗，肿瘤迅速再次出现反应，并且在持续的双重靶向治疗下仍处于第二次持久临床缓解期（目前>16个月）。我们讨论了该病例研究对未来治疗策略的意义。
