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Complete clinical regression of a BRAF V600E-mutant pediatric glioblastoma multiforme after BRAF inhibitor therapy
by
Orr, Brent A
, Robinson, Giles W
, Gajjar, Amar
in
Biomedical and Life Sciences
/ Biomedicine
/ Brain research
/ Cancer Research
/ Case Report
/ Chemotherapy
/ Child
/ Clinical oncology
/ Glioblastoma - drug therapy
/ Glioblastoma - genetics
/ Glioblastoma - pathology
/ Health Promotion and Disease Prevention
/ Humans
/ Indoles - administration & dosage
/ Magnetic resonance imaging
/ Male
/ Medicine/Public Health
/ Melanoma
/ Mutation
/ Neoplasm Recurrence, Local - drug therapy
/ Neoplasm Recurrence, Local - genetics
/ Neoplasm Recurrence, Local - pathology
/ Oncology
/ Pediatrics
/ Proto-Oncogene Proteins B-raf - antagonists & inhibitors
/ Proto-Oncogene Proteins B-raf - genetics
/ Radiation
/ Sulfonamides - administration & dosage
/ Surgical Oncology
/ Tumors
2014
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Complete clinical regression of a BRAF V600E-mutant pediatric glioblastoma multiforme after BRAF inhibitor therapy
by
Orr, Brent A
, Robinson, Giles W
, Gajjar, Amar
in
Biomedical and Life Sciences
/ Biomedicine
/ Brain research
/ Cancer Research
/ Case Report
/ Chemotherapy
/ Child
/ Clinical oncology
/ Glioblastoma - drug therapy
/ Glioblastoma - genetics
/ Glioblastoma - pathology
/ Health Promotion and Disease Prevention
/ Humans
/ Indoles - administration & dosage
/ Magnetic resonance imaging
/ Male
/ Medicine/Public Health
/ Melanoma
/ Mutation
/ Neoplasm Recurrence, Local - drug therapy
/ Neoplasm Recurrence, Local - genetics
/ Neoplasm Recurrence, Local - pathology
/ Oncology
/ Pediatrics
/ Proto-Oncogene Proteins B-raf - antagonists & inhibitors
/ Proto-Oncogene Proteins B-raf - genetics
/ Radiation
/ Sulfonamides - administration & dosage
/ Surgical Oncology
/ Tumors
2014
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Complete clinical regression of a BRAF V600E-mutant pediatric glioblastoma multiforme after BRAF inhibitor therapy
by
Orr, Brent A
, Robinson, Giles W
, Gajjar, Amar
in
Biomedical and Life Sciences
/ Biomedicine
/ Brain research
/ Cancer Research
/ Case Report
/ Chemotherapy
/ Child
/ Clinical oncology
/ Glioblastoma - drug therapy
/ Glioblastoma - genetics
/ Glioblastoma - pathology
/ Health Promotion and Disease Prevention
/ Humans
/ Indoles - administration & dosage
/ Magnetic resonance imaging
/ Male
/ Medicine/Public Health
/ Melanoma
/ Mutation
/ Neoplasm Recurrence, Local - drug therapy
/ Neoplasm Recurrence, Local - genetics
/ Neoplasm Recurrence, Local - pathology
/ Oncology
/ Pediatrics
/ Proto-Oncogene Proteins B-raf - antagonists & inhibitors
/ Proto-Oncogene Proteins B-raf - genetics
/ Radiation
/ Sulfonamides - administration & dosage
/ Surgical Oncology
/ Tumors
2014
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Complete clinical regression of a BRAF V600E-mutant pediatric glioblastoma multiforme after BRAF inhibitor therapy
Journal Article
Complete clinical regression of a BRAF V600E-mutant pediatric glioblastoma multiforme after BRAF inhibitor therapy
2014
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Overview
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.
Publisher
BioMed Central,Springer Nature B.V
Subject
/ Child
/ Health Promotion and Disease Prevention
/ Humans
/ Indoles - administration & dosage
/ Male
/ Melanoma
/ Mutation
/ Neoplasm Recurrence, Local - drug therapy
/ Neoplasm Recurrence, Local - genetics
/ Neoplasm Recurrence, Local - pathology
/ Oncology
/ Proto-Oncogene Proteins B-raf - antagonists & inhibitors
/ Proto-Oncogene Proteins B-raf - genetics
/ Sulfonamides - administration & dosage
/ Tumors
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