Progression of RAS-mutant leukemia during RAF inhibitor treatment

N Engl J Med. 2012 Dec 13;367(24):2316-21. doi: 10.1056/NEJMoa1208958. Epub 2012 Nov 7.

Abstract

Vemurafenib, a selective RAF inhibitor, extends survival among patients with BRAF V600E-mutant melanoma. Vemurafenib inhibits ERK signaling in BRAF V600E-mutant cells but activates ERK signaling in BRAF wild-type cells. This paradoxical activation of ERK signaling is the mechanistic basis for the development of RAS-mutant squamous-cell skin cancers in patients treated with RAF inhibitors. We report the accelerated growth of a previously unsuspected RAS-mutant leukemia in a patient with melanoma who was receiving vemurafenib. Exposure to vemurafenib induced hyperactivation of ERK signaling and proliferation of the leukemic cell population, an effect that was reversed on drug withdrawal.

Publication types

  • Case Reports

MeSH terms

  • Aged
  • Cell Proliferation / drug effects
  • Disease Progression
  • Genes, ras*
  • Humans
  • Indoles / adverse effects*
  • Indoles / therapeutic use
  • Leukemia, Myelomonocytic, Chronic / chemically induced
  • Leukemia, Myelomonocytic, Chronic / genetics*
  • Leukocyte Count
  • Male
  • Melanoma / drug therapy
  • Melanoma / genetics*
  • Mutation
  • Protein Kinase Inhibitors / adverse effects*
  • Protein Kinase Inhibitors / therapeutic use
  • Proto-Oncogene Proteins B-raf / antagonists & inhibitors*
  • Proto-Oncogene Proteins B-raf / genetics
  • Sulfonamides / adverse effects*
  • Sulfonamides / therapeutic use
  • Vemurafenib

Substances

  • Indoles
  • Protein Kinase Inhibitors
  • Sulfonamides
  • Vemurafenib
  • Proto-Oncogene Proteins B-raf