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

Onderzoeksoutput: Bijdrage aan tijdschriftArtikelpeer review


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.

Originele taal-2Engels
Pagina's (van-tot)1101-3
Aantal pagina's3
TijdschriftPediatric blood & cancer
Nummer van het tijdschrift6
StatusGepubliceerd - jun. 2014
Extern gepubliceerdJa


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