Targeted therapy for infants with diencephalic syndrome: A case report and review of management strategies.
Pediatr Blood Cancer
; 65(5): e26917, 2018 05.
Article
en En
| MEDLINE
| ID: mdl-29369501
ABSTRACT
Young children with emaciation caused by a hypothalamic glioma are considered to have diencephalic syndrome (DS), which is often poorly controlled with conventional treatment. We describe an infant with DS whose tumor progressed following chemotherapy. Biopsy was performed for molecular testing and demonstrated a BRAF fusion. Treatment with the MEK inhibitor trametinib for 18 months resulted in reduction of tumor size, normalization of his weight curve, and marked neurodevelopmental improvement. Our results build on earlier reports of using targeted agents for low-grade glioma, and we review the evolving management strategy for such patients in the era of precision medicine.
Palabras clave
Texto completo:
1
Bases de datos:
MEDLINE
Asunto principal:
Piridonas
/
Pirimidinonas
/
Proteínas Proto-Oncogénicas c-met
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Inhibidores de Proteínas Quinasas
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Terapia Molecular Dirigida
/
Enfermedades Hipotalámicas
Tipo de estudio:
Prognostic_studies
Límite:
Humans
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Infant
/
Male
Idioma:
En
Revista:
Pediatr Blood Cancer
Asunto de la revista:
HEMATOLOGIA
/
NEOPLASIAS
/
PEDIATRIA
Año:
2018
Tipo del documento:
Article