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Profound clinical and radiological response to BRAF inhibition in a 2‐month‐old diencephalic child with hypothalamic/chiasmatic glioma
Author(s) -
Lassaletta Alvaro,
Guerreiro Stucklin Ana,
Ramaswamy Vijay,
Zapotocky Michal,
McKeown Tara,
Hawkins Cynthia,
Bouffet Eric,
Tabori Uri
Publication year - 2016
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.26086
Subject(s) - medicine , glioma , oncology , population , pediatrics , cancer research , environmental health
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.