Pilomyxoid astrocytoma treated successfully with vemurafenib

Mary Skrypek, Nicholas Foreman, Daniel Guillaume, Christopher Moertel

Research output: Contribution to journalArticlepeer-review

44 Scopus citations


The BRAF V600E missense mutation is known to be present in a subset of central nervous system tumors. We report a patient with a BRAF V600E mutated pilomyxoid astrocytoma who failed multiple conventional chemotherapy regimens. Treatment with vemurafenib, a molecularly targeted therapy against the mutant BRAF V600E kinase, combined with vinblastine resulted in tumor regression. Furthermore, this patient experienced almost immediate progression of disease after holding vemurafenib for only 2-3 weeks, suggesting that the tumor response is vemurafenib dependent. This population of patients may benefit from targeted therapy and testing of individual tumors for BRAF mutations is justified.

Original languageEnglish (US)
Pages (from-to)2099-2100
Number of pages2
JournalPediatric Blood and Cancer
Issue number11
StatePublished - Nov 1 2014


  • Brain tumors
  • CNS tumors
  • Tumor markers

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