PDF(Pubmed)
Abstract：
BACKGROUND: Ameloblastoma in 66% of the cases harbor a somatic mutation of the \"mitogen-activated protein kinase\" signaling pathway (BRAF V600E). In V600E mutations, BRAF is in the permanent \"on\" state and relays the growth-promoting signals independently of the EGFR pathway. Therefore, mutant BRAF represents a target for handful of new drugs.
METHODS: We conducted a literature search, with the search terms \"Vemurafenib, Dabrafenib, Ameloblastoma, and BRAF.\" These included seven case reports with nine patients who underwent monotherapy with Dabrafenib or Vemurafenib or combination therapy with Dabrafenib and Trametinib.
RESULTS: The patients age ranges from 10 years up to 86 years. The distribution of women and men is 4:5. Patients with an initial diagnosis of ameloblastoma, as well as recurrences or metastasized ameloblastoma were treated. Indications cover neoadjuvant therapy up to the use in metastasized patients in an irresectable state. Results ranging from \"only\" tumor size reduction to restitutio ad integrum.
CONCLUSIONS: We see the use of BRAF Inhibitors to reduce tumor size with consecutive surgical treatment as a reasonable option for therapy. However, we are aware that at present the data are based only on case reports with the longest follow-up of just 38 months. We encourage further clinical trials in the use of BRAF Inhibitors for selecting ameloblastoma patients in a multi-center setting.
METHODS: We conducted a literature search, with the search terms \"Vemurafenib, Dabrafenib, Ameloblastoma, and BRAF.\" These included seven case reports with nine patients who underwent monotherapy with Dabrafenib or Vemurafenib or combination therapy with Dabrafenib and Trametinib.
RESULTS: The patients age ranges from 10 years up to 86 years. The distribution of women and men is 4:5. Patients with an initial diagnosis of ameloblastoma, as well as recurrences or metastasized ameloblastoma were treated. Indications cover neoadjuvant therapy up to the use in metastasized patients in an irresectable state. Results ranging from \"only\" tumor size reduction to restitutio ad integrum.
CONCLUSIONS: We see the use of BRAF Inhibitors to reduce tumor size with consecutive surgical treatment as a reasonable option for therapy. However, we are aware that at present the data are based only on case reports with the longest follow-up of just 38 months. We encourage further clinical trials in the use of BRAF Inhibitors for selecting ameloblastoma patients in a multi-center setting.
摘要：
背景：66％的成釉细胞瘤具有“丝裂原活化蛋白激酶”信号通路（BRAFV600E）的体细胞突变。在V600E突变中，BRAF处于永久性“开启”状态，并独立于EGFR通路传递促进生长的信号。因此,突变型BRAF代表了少数新药的靶标。
方法：我们进行了文献检索，带有搜索词\“Vemurafenib，Dabrafenib,成釉细胞瘤,还有BRAF.“其中包括7例病例报告，其中9例患者接受了Dabrafenib或Vemurafenib的单一治疗或Dabrafenib和Trametinib的联合治疗。
结果：患者年龄从10岁到86岁不等。男女的分布是4:5。初步诊断为成釉细胞瘤的患者，以及复发或转移成釉细胞瘤的治疗。适应症涵盖新辅助治疗，直至在无法切除的转移患者中使用。结果从“唯一”肿瘤大小减小到整合素恢复。
结论：我们认为使用BRAF抑制剂以减少肿瘤大小并连续手术治疗是一种合理的治疗选择。然而,我们知道，目前的数据仅基于最长随访时间仅为38个月的病例报告。我们鼓励在多中心环境中使用BRAF抑制剂选择成釉细胞瘤患者的进一步临床试验。
方法：我们进行了文献检索，带有搜索词\“Vemurafenib，Dabrafenib,成釉细胞瘤,还有BRAF.“其中包括7例病例报告，其中9例患者接受了Dabrafenib或Vemurafenib的单一治疗或Dabrafenib和Trametinib的联合治疗。
结果：患者年龄从10岁到86岁不等。男女的分布是4:5。初步诊断为成釉细胞瘤的患者，以及复发或转移成釉细胞瘤的治疗。适应症涵盖新辅助治疗，直至在无法切除的转移患者中使用。结果从“唯一”肿瘤大小减小到整合素恢复。
结论：我们认为使用BRAF抑制剂以减少肿瘤大小并连续手术治疗是一种合理的治疗选择。然而,我们知道，目前的数据仅基于最长随访时间仅为38个月的病例报告。我们鼓励在多中心环境中使用BRAF抑制剂选择成釉细胞瘤患者的进一步临床试验。
