Vemurafenib in Pediatric Patients With BRAFV600E Mutated High-Grade Gliomas

被引：118

作者：

Bautista, Francisco ^{[1
]}

Paci, Angelo ^{[2
]}

Minard-Colin, Veronique ^{[1
]}

Dufour, Christelle ^{[1
]}

Grill, Jacques ^{[1
]}

Lacroix, Ludovic ^{[3
]}

Varlet, Pascale ^{[4
]}

Valteau-Couanet, Dominique ^{[1
]}

Geoerger, Birgit ^{[1
]}

机构：

[1] Gustave Roussy, Pediat & Adolescent Oncol Dept, F-94805 Villejuif, France

[2] Pharmacol & Drug Anal Dept, Villejuif, France

[3] Gustave Roussy, Dept Biol Clin, F-94805 Villejuif, France

[4] Ctr Hosp St Anne, Pathol & Neurooncol Dept, Paris, France

来源：

PEDIATRIC BLOOD & CANCER | 2014年 / 61卷 / 06期

关键词：

anaplastic ganglioglioma; BRAF mutations; pediatric brain tumors; pharmacokinetics; vemurafenib; BRAF; MELANOMA; METASTASES;

D O I：

10.1002/pbc.24891

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

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. (c) 2013 Wiley Periodicals, Inc.

引用

页码：1101 / 1103

页数：3

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