@article{0c15b60a7e7a4e82af3462810cefc88a,
title = "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.",
keywords = "Antibodies, Monoclonal, Humanized/administration & dosage, Antineoplastic Agents/adverse effects, Antineoplastic Combined Chemotherapy Protocols/therapeutic use, Astrocytoma/drug therapy, Bevacizumab, Brain Neoplasms/drug therapy, Camptothecin/administration & dosage, Cancer Vaccines/therapeutic use, Child, Combined Modality Therapy, Cranial Irradiation, Dacarbazine/administration & dosage, Doxorubicin/administration & dosage, Drug Evaluation, Fatal Outcome, Ganglioglioma/drug therapy, Humans, Immunotherapy, Indoles/adverse effects, Infant, Irinotecan, Male, Mutation, Missense, Neoplasm Proteins/antagonists & inhibitors, Point Mutation, Polyethylene Glycols/administration & dosage, Protein Kinase Inhibitors/adverse effects, Proto-Oncogene Proteins B-raf/antagonists & inhibitors, Salvage Therapy, Sulfonamides/adverse effects, Temozolomide, Thalamus, Treatment Outcome, Vemurafenib",
author = "Francisco Bautista and Angelo Paci and Veronique Minard-Colin and Christelle Dufour and Jacques Grill and Ludovic Lacroix and Pascale Varlet and Dominique Valteau-Couanet and Birgit Geoerger",
note = "{\textcopyright} 2013 Wiley Periodicals, Inc.",
year = "2014",
month = jun,
doi = "10.1002/pbc.24891",
language = "English",
volume = "61",
pages = "1101--3",
journal = "Pediatric blood & cancer",
issn = "1545-5009",
publisher = "Wiley-Liss Inc.",
number = "6",
}