PDF(Pubmed)
Abstract：
A combination of BRAF and MEK inhibitors is reported to be effective for gliomas with the BRAF V600E mutation; however, its efficacy in gliomas with leptomeningeal metastases (LMM) is still unknown. In this report, we describe two pediatric patients with high-grade glioma featuring the BRAF V600E mutation who were treated with dabrafenib and trametinib for LMM. Both 2 cases underwent craniotomy for primary intracranial lesions and were diagnosed as a high-grade glioma with BRAF V600E mutation; one case was consistent with anaplastic pleomorphic xanthoastorocytoma, the other was epithelioid glioblastoma. They received standard treatment for the lesions but subsequently were found to have new lesions including multiple spinal dissemination. We started administering dabrafenib and trametinib. Within a few days of starting treatment, the symptoms improved dramatically and MRI performed one month after the prescription of the two drugs demonstrated remission of both brain and spinal lesions. This report shows that dabrafenib and trametinib are effective not only for recurrent lesions but also for LMM in pediatric patients.
摘要：
据报道，BRAF和MEK抑制剂的组合对具有BRAFV600E突变的神经胶质瘤有效；然而，其在伴有软脑膜转移（LMM）的神经胶质瘤中的疗效尚不清楚。在这份报告中,我们描述了两名患有BRAFV600E突变的高级别神经胶质瘤的儿科患者,他们分别接受了dabrafenib和trametinib治疗LMM.2例均因颅内原发病变行开颅手术，诊断为BRAFV600E突变的高级别胶质瘤；1例符合间变性多形性黄色母细胞瘤，另一个是上皮样胶质母细胞瘤。他们接受了病变的标准治疗，但随后发现有新的病变，包括多发性脊柱播散。我们开始服用达布拉非尼和曲美替尼。在开始治疗的几天内，两种药物的处方后1个月，症状得到了显着改善，MRI检查显示脑和脊髓病变均缓解。该报告显示dabrafenib和trametinib不仅对复发性病变有效，而且对儿科患者的LMM也有效。
