Toll Stephanie A, Tran Hung N, Cotter Jennifer, Judkins Alexander R, Tamrazi Benita, Biegel Jaclyn A, Dhall Girish, Robison Nathan J, Waters Kaaren, Patel Palak, Cooper Robert, Margol Ashley S
Division of Hematology, Oncology and Blood and Marrow Transplantation, Children's Center for Cancer and Blood Diseases, Children's Hospital Los Angeles, Los Angeles, CA, USA.
University of Southern California Keck School of Medicine, Los Angeles, CA, USA.
Oncotarget. 2019 Jan 11;10(4):551-557. doi: 10.18632/oncotarget.26560.
Outcomes for children with high-grade gliomas (HGG) remain dismal despite aggressive treatment strategies. The use of targeted therapy for BRAF mutated malignancies including HGG is being explored as a potentially well tolerated and effective therapeutic option. The results of adult melanoma studies demonstrating that combination therapy with BRAF inhibitors and MEK inhibitors results in prolonged survival led us to employ this treatment strategy in children with BRAF mutated HGG. In this case series, we describe three pediatric patients with HGG with confirmed BRAF mutation who demonstrated responses to combination therapy with dabrafenib and trametinib.
尽管采取了积极的治疗策略，但高级别胶质瘤（HGG）患儿的预后仍然很差。针对包括HGG在内的BRAF突变恶性肿瘤的靶向治疗正在被探索，作为一种潜在耐受性良好且有效的治疗选择。成人黑色素瘤研究结果表明，BRAF抑制剂和MEK抑制剂联合治疗可延长生存期，这促使我们对BRAF突变的HGG患儿采用这种治疗策略。在这个病例系列中，我们描述了三名确诊为BRAF突变的HGG儿科患者，他们对达拉非尼和曲美替尼联合治疗有反应。
