Vemurafenib in pediatric patients with <scp><i>BRAFV</i></scp><i>600E</i> mutated high‐grade gliomas

TLDR

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. Further exploration in clinical studies is ongoing. 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 achieved a confirmed partial response and remains alive 20 months after treatment initiation, a second patient with ganglioglioma had a transient response to vemurafenib re‑introduction after immunotherapy, and the cases collectively support BRAFV600E as an oncogenic driver in pediatric gliomas. Pediatr Blood Cancer 2014;61:1101–1103; © 2013 Wiley Periodicals, Inc.

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. Pediatr Blood Cancer 2014;61:1101–1103. © 2013 Wiley Periodicals, Inc.