Sustained Response to Targeted Therapy in a Patient With Disseminated Anaplastic Pleomorphic Xanthoastrocytoma

J Pediatr Hematol Oncol. 2018 Aug;40(6):478-482. doi: 10.1097/MPH.0000000000001032.

Abstract

Pleomorphic xanthoastrocytoma is a rare brain tumor with unique high frequency of BRAF V600E mutation which is plausible for targeted therapy. The anaplastic variant has generally worse prognosis. We present an adolescent patient with a disseminated relapse of anaplastic pleomorphic xanthoastrocytoma following surgery, radiotherapy, and chemotherapy. She had a dramatic and prolonged response to a BRAF inhibitor (Dabrafinib) and later to addition of a MEK inhibitor (Trametinib) on tumor progression. With minimal side effects and a good quality of life, the patient is alive more than 2 years after initiation of targeted therapy. This experience confirms the potential role of targeted treatments in high-grade BRAF-mutated brain tumors.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Amino Acid Substitution
  • Astrocytoma* / genetics
  • Astrocytoma* / therapy
  • Brain Neoplasms* / genetics
  • Brain Neoplasms* / therapy
  • Female
  • Humans
  • Imidazoles / administration & dosage*
  • Mutation, Missense*
  • Oximes / administration & dosage*
  • Proto-Oncogene Proteins B-raf / genetics*
  • Pyridones / administration & dosage*
  • Pyrimidinones / administration & dosage*

Substances

  • Imidazoles
  • Oximes
  • Pyridones
  • Pyrimidinones
  • trametinib
  • BRAF protein, human
  • Proto-Oncogene Proteins B-raf
  • dabrafenib