Tan Serena, Pollack Jonathan R, Kaplan Michael J, Colevas A Dimitri, West Robert B
Department of Pathology, Stanford University School of Medicine, Stanford, CA, USA.
Department of Surgery, Stanford University School of Medicine, Stanford, CA, USA.
Oral Surg Oral Med Oral Pathol Oral Radiol. 2016 Jul;122(1):e5-7. doi: 10.1016/j.oooo.2015.12.016. Epub 2016 Feb 23.
Molecular characterization of ameloblastoma has indicated a high frequency of driver mutations in BRAF and SMO. Preclinical data suggest that Food and Drug Administration-approved BRAF-targeted therapies may be immediately relevant for patients with ameloblastoma positive for the BRAF V600E mutation.
A neoadjuvant treatment regime of dabrafenib was given to a patient with recurrent BRAF-mutant mandibular ameloblastoma. The patient subsequently underwent left mandible composite resection of the tumor and pathologic evaluation of treatment response.
The ameloblastoma had a slow but dramatic response with >90% tumor volume reduction. The inner areas of the tumor underwent degeneration and squamous differentiation, and intact ameloblastoma was present in the outer areas associated with bone.
Targeted neoadjuvant therapy for ameloblastoma may be useful in certain clinical settings of primary ameloblastoma. These might include tumors of advanced local stage when a neoadjuvant reduction could alter the extent of surgery and instances of local recurrence when surgical options are limited.
成釉细胞瘤的分子特征表明，BRAF和SMO基因中驱动突变的频率很高。临床前数据表明，美国食品药品监督管理局批准的BRAF靶向疗法可能对BRAF V600E突变阳性的成釉细胞瘤患者具有直接相关性。
对一名复发性BRAF突变型下颌成釉细胞瘤患者给予达拉非尼新辅助治疗方案。该患者随后接受了左侧下颌骨肿瘤复合切除术及治疗反应的病理评估。
成釉细胞瘤反应缓慢但显著，肿瘤体积缩小>90%。肿瘤内部区域发生退变和鳞状分化，与骨相关的外部区域存在完整的成釉细胞瘤。
成釉细胞瘤的靶向新辅助治疗在某些原发性成釉细胞瘤的临床情况下可能有用。这些情况可能包括局部晚期肿瘤，此时新辅助缩小可改变手术范围，以及手术选择有限的局部复发情况。
