Vemurafenib in pediatric patients with BRAFV600E mutated high-grade gliomas

Francisco Bautista; Angelo Paci; Veronique Minard-Colin; Christelle Dufour; Jacques Grill; Ludovic Lacroix; Pascale Varlet; Dominique Valteau-Couanet; Birgit Geoerger

doi:10.1002/pbc.24891

Vemurafenib in pediatric patients with BRAFV600E mutated high-grade gliomas

Francisco Bautista
, Angelo Paci
, Veronique Minard-Colin
, Christelle Dufour
, Jacques Grill
, Ludovic Lacroix
, Pascale Varlet
, Dominique Valteau-Couanet
, Birgit Geoerger

Research output: Contribution to journal › Article › peer-review

143 Citations (Scopus)

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.

Original language	English
Pages (from-to)	1101-1103
Number of pages	3
Journal	Pediatric Blood and Cancer
Volume	61
Issue number	6
DOIs	https://doi.org/10.1002/pbc.24891
Publication status	Published - Jun 2014
Externally published	Yes

Keywords

Anaplastic ganglioglioma
BRAF mutations
Pediatric brain tumors
Pharmacokinetics
Vemurafenib

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10.1002/pbc.24891

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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.",

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AU - Bautista, Francisco

AU - Paci, Angelo

AU - Minard-Colin, Veronique

AU - Dufour, Christelle

AU - Grill, Jacques

AU - Lacroix, Ludovic

AU - Varlet, Pascale

AU - Valteau-Couanet, Dominique

AU - Geoerger, Birgit

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AB - 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.

KW - Anaplastic ganglioglioma

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KW - Pediatric brain tumors

KW - Pharmacokinetics

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Vemurafenib in pediatric patients with BRAFV600E mutated high-grade gliomas

Abstract

Keywords

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