Vemurafenib in pediatric patients with BRAFV600E mutated high-grade gliomas.

Abstract:

:We present three pediatric patients with BRAFV600E mutant high-grade gliomas treated by vemurafenib on a nominative authorization level at our institution. One patient with anaplastic ganglioglioma experienced confirmed partial tumor response and significant clinical improvement and she is alive 20 months after start of treatment. A second patient with ganglioglioma responded transiently to re-introduction of vemurafenib after immunotherapy. Pharmacokinetic studies suggest that maximum concentration and exposure of vemurafenib at steady-state is dose-dependent and similar in children to that reported in adults. These cases suggest that BRAFV600 is an oncogenic driver in pediatric gliomas. Further exploration in clinical studies is ongoing.

journal_name

Pediatr Blood Cancer

journal_title

Pediatric blood & cancer

authors

Bautista F,Paci A,Minard-Colin V,Dufour C,Grill J,Lacroix L,Varlet P,Valteau-Couanet D,Geoerger B

doi

10.1002/pbc.24891

subject

Has Abstract

pub_date

2014-06-01 00:00:00

pages

1101-3

issue

6

eissn

1545-5009

issn

1545-5017

journal_volume

61

pub_type

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