Complete clinical regression of a BRAF V600E-mutant pediatric glioblastoma multiforme after BRAF inhibitor therapy

التفاصيل البيبلوغرافية
العنوان: Complete clinical regression of a BRAF V600E-mutant pediatric glioblastoma multiforme after BRAF inhibitor therapy
المؤلفون: Robinson, Giles W, Orr, Brent A, Gajjar, Amar
بيانات النشر: BioMed Central Ltd.
سنة النشر: 2014
المجموعة: BioMed Central
مصطلحات موضوعية: High-grade glioma, Glioblastoma multiforme, BRAF mutations, V600E, Pediatric brain tumor, BRAF inhibitors
الوصف: Background Standard therapies for high grade glioma have failed to substantially improve survival and are associated with significant morbidity. At relapse, high grade gliomas, such as glioblastoma multiforme, are refractory to therapy and universally fatal. BRAF V600E -mutations have been described in a modest 6% to 7% of primary central nervous system (CNS) tumors, but with increased prevalence in the pediatric population and in certain brain tumor subtypes. The use of BRAF inhibitors have transformed melanoma therapy however their use in brain tumors remains unproven. Case presentation We describe the pediatric case of a now 12 year old Caucasian male who originally presented at age 9 with a right fronto-parietal glioblastoma multiforme that recurred 2 ½ years from diagnosis. Molecular analysis of the primary tumor revealed a BRAF V600E mutation and the patient was placed on the BRAF inhibitor vemurafenib. A complete response was observed after 4 months of therapy and remains sustained at 6 months. Conclusion This is the first report of a complete response of relapsed glioblastoma multiforme to targeted BRAF inhibitor therapy. While not a predominant mutation in glioblastoma multiforme, the increased prevalence of BRAF V600 mutations in pediatric CNS tumors and certain subtypes marks a population to whom this therapy could be applied. Response to this therapy suggests that BRAF inhibitors can affect primary CNS lesions when a documented and targetable mutation is present.
نوع الوثيقة: report
اللغة: English
العلاقة: http://www.biomedcentral.com/1471-2407/14/258Test
الإتاحة: http://www.biomedcentral.com/1471-2407/14/258Test
حقوق: Copyright 2014 Robinson et al.; licensee BioMed Central Ltd.
رقم الانضمام: edsbas.5A233A53
قاعدة البيانات: BASE