Department of Medicine, Division of Medical Oncology, Washington University School of Medicine
Siteman Cancer Center at Barnes-Jewish Hospital and Washington University School of Medicine
J Natl Compr Canc Netw. 2018 Jan;16(1):4-10. doi: 10.6004/jnccn.2017.7032.
V600E mutations have been successfully treated with targeted therapy in melanoma, non-small cell lung cancer, and thyroid cancer. Interestingly, these mutations have also been identified in a subset of pediatric and adult brain tumors, with several cases reportedly responding to targeted therapy. However, these reports have been limited to single-agent BRAF inhibitor therapy and recurrent disease. Herein, we report dramatic clinical and radiographic responses to combination dabrafenib (BRAF inhibitor) and trametinib (MEK inhibitor) in 2 adults with high-grade gliomas (HGGs), with 1 patient treated in the first-line setting. These observations, together with prior case reports, advocate for routine screening of point mutations in adult HGGs, and suggest that treatment with dual-targeted therapy, even in newly diagnosed cases, is safe and effective.
V600E 突变已成功通过靶向治疗在黑色素瘤、非小细胞肺癌和甲状腺癌中得到治疗。有趣的是,这些突变也在一部分儿科和成人脑肿瘤中被发现,有报道称一些病例对靶向治疗有反应。然而,这些报告仅限于单一的 BRAF 抑制剂治疗和复发性疾病。在此,我们报告了 2 例高级别神经胶质瘤(HGG)患者联合使用 dabrafenib(BRAF 抑制剂)和 trametinib(MEK 抑制剂)的显著临床和影像学反应,其中 1 例患者在一线治疗中使用。这些观察结果,以及之前的病例报告,提倡对成人 HGG 进行点突变的常规筛查,并表明即使在新诊断的病例中,使用双重靶向治疗也是安全有效的。