Targeted treatment of papillary craniopharyngiomas harboring BRAF V600E mutations

Cancer. 2019 Sep 1;125(17):2910-2914. doi: 10.1002/cncr.32197. Epub 2019 Jul 17.

Abstract

Papillary craniopharyngiomas (PCPs) are characterized by the presence of BRAF V600E mutations, which are emerging as a useful guide for diagnosis and treatment decision making. The ongoing multicenter phase 2 Alliance A071601 trial is evaluating the efficacy of BRAF and mitogen-activated protein kinase kinase (MEK) inhibitors for patients with PCPs. With continued successful responses, it is proposed that BRAF (and MEK) inhibitors be evaluated for the neoadjuvant treatment of patients with PCPs.

Publication types

  • Case Reports
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aged
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use
  • Craniopharyngioma / diagnostic imaging
  • Craniopharyngioma / drug therapy*
  • Craniopharyngioma / pathology
  • Craniopharyngioma / surgery
  • Humans
  • Male
  • Middle Aged
  • Molecular Targeted Therapy
  • Mutation*
  • Pituitary Neoplasms / diagnostic imaging
  • Pituitary Neoplasms / drug therapy*
  • Pituitary Neoplasms / pathology
  • Pituitary Neoplasms / surgery
  • Protein Kinase Inhibitors / therapeutic use*
  • Proto-Oncogene Proteins B-raf / antagonists & inhibitors
  • Proto-Oncogene Proteins B-raf / genetics*
  • Young Adult

Substances

  • Protein Kinase Inhibitors
  • BRAF protein, human
  • Proto-Oncogene Proteins B-raf