Management of Inoperable Supra-Sellar Low-Grade Glioma With BRAF Mutation in Young Children.
braf-v600e
inoperable
liquid biopsy
low grade gliomas
molecular biomarker
pediatric
Journal
Cureus
ISSN: 2168-8184
Titre abrégé: Cureus
Pays: United States
ID NLM: 101596737
Informations de publication
Date de publication:
Nov 2021
Nov 2021
Historique:
accepted:
08
11
2021
entrez:
20
12
2021
pubmed:
21
12
2021
medline:
21
12
2021
Statut:
epublish
Résumé
Pediatric low-grade gliomas (PLGGs) are the most common central nervous system (CNS) tumors in children. The current standard of care for surgically unresectable and/or progressive cases of PLGGs includes combination chemotherapy. PLGGs are molecularly characterized by alterations in the RAS/RAF/MAPK/ERK pathway in a majority of tumors. PLGGs harboring the BRAF-V600E mutation respond poorly to current chemotherapy strategies. We present a case of a two-year-old female with biopsy-proven low-grade glioma (LGG, pilocytic astrocytoma) involving the hypothalamic/optic chiasm region. At presentation, she had obstructive hydrocephalus, bitemporal hemianopia, central hypothyroidism, and right-sided hemiparesis due to the location/mass effect of the tumor. She was initially treated with chemotherapy (vincristine/carboplatin), but her tumor progressed at six weeks of treatment. She was subsequently started on dabrafenib as her tumor was positive for BRAF-V600E mutation. Dabrafenib monotherapy resulted in dramatic improvement in her clinical symptoms and near-complete resolution of tumor. Our experience and review of the literature suggest that LGGs with BRAF-V600E mutations may benefit from upfront targeted therapy in children. There is an urgent need for prospective clinical trials comparing the efficacy of upfront BRAF inhibitors versus standard chemotherapy in PLGGs with BRAF mutations.
Identifiants
pubmed: 34926002
doi: 10.7759/cureus.19400
pmc: PMC8656291
doi:
Types de publication
Case Reports
Langues
eng
Pagination
e19400Informations de copyright
Copyright © 2021, Howden et al.
Déclaration de conflit d'intérêts
The authors have declared that no competing interests exist.
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