Profound clinical and radiological response to BRAF inhibition in a 2-month-old diencephalic child with hypothalamic/chiasmatic glioma.
Pediatr Blood Cancer
; 63(11): 2038-41, 2016 11.
Article
em En
| MEDLINE
| ID: mdl-27398937
ABSTRACT
Infants with low-grade glioma (LGG) have a poor survival. BRAFV600E mutation has been identified in pediatric LGG; however, the use of BRAF inhibitors in infants has never been reported. A 2-month-old with V600E mutant hypothalamic/chiasmatic glioma progressed on chemotherapy resulting in profound visual loss, massive ascites, and diencephalic syndrome. Initiation of dabrafenib resulted in rapid and sustained disappearance of clinical symptoms and a profound sustained cytoreduction. BRAF inhibition was safely tolerated with dramatic clinicoradiological response, suggesting early targeted therapy is a viable option in infants with LGG. A re-evaluation of current management paradigms in this population is warranted to leverage the potential benefit of upfront-targeted therapies.
Palavras-chave
Texto completo:
1
Bases de dados:
MEDLINE
Assunto principal:
Neoplasias Encefálicas
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Proteínas Proto-Oncogênicas B-raf
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Glioma
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Doenças Hipotalâmicas
Tipo de estudo:
Prognostic_studies
Limite:
Female
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Humans
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Infant
Idioma:
En
Revista:
Pediatr Blood Cancer
Assunto da revista:
HEMATOLOGIA
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NEOPLASIAS
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PEDIATRIA
Ano de publicação:
2016
Tipo de documento:
Article
País de afiliação:
Canadá