Conflict of Interest: Nothing to declare.
Vemurafenib in pediatric patients with BRAFV600E mutated high-grade gliomas
Article first published online: 3 DEC 2013
© 2013 Wiley Periodicals, Inc.
Pediatric Blood & Cancer
Volume 61, Issue 6, pages 1101–1103, June 2014
How to Cite
Bautista, F., Paci, A., Minard-Colin, V., Dufour, C., Grill, J., Lacroix, L., Varlet, P., Valteau-Couanet, D. and Geoerger, B. (2014), Vemurafenib in pediatric patients with BRAFV600E mutated high-grade gliomas. Pediatr. Blood Cancer, 61: 1101–1103. doi: 10.1002/pbc.24891
- Issue published online: 16 APR 2014
- Article first published online: 3 DEC 2013
- Manuscript Accepted: 12 NOV 2013
- Manuscript Received: 16 OCT 2013
- anaplastic ganglioglioma;
- BRAF mutations;
- pediatric brain tumors;
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. Pediatr Blood Cancer 2014;61:1101–1103. © 2013 Wiley Periodicals, Inc.