Pilomyxoid astrocytoma treated successfully with vemurafenib

Pediatr Blood Cancer. 2014 Nov;61(11):2099-100. doi: 10.1002/pbc.25084. Epub 2014 May 12.


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.

Keywords: CNS tumors; brain tumors; tumor markers.

Publication types

  • Case Reports

MeSH terms

  • Astrocytoma / drug therapy*
  • Astrocytoma / genetics
  • Brain Neoplasms / drug therapy*
  • Brain Neoplasms / genetics
  • Humans
  • Indoles / therapeutic use*
  • Infant
  • Male
  • Mutation
  • Proto-Oncogene Proteins B-raf / antagonists & inhibitors*
  • Proto-Oncogene Proteins B-raf / genetics
  • Sulfonamides / therapeutic use*
  • Vemurafenib


  • Indoles
  • Sulfonamides
  • Vemurafenib
  • BRAF protein, human
  • Proto-Oncogene Proteins B-raf