Imatinib for systemic mast-cell disease

Lancet. 2003 Aug 16;362(9383):535-6. doi: 10.1016/s0140-6736(03)14115-3.


Imatinib has shown to be effective against malignant disease driven by ckit. We prospectively treated 12 adults with symptomatic systemic mast-cell disease at a dose of either 100 mg or 400 mg per day. Of the ten patients who we could assess for response, five (50%) had a measurable response to the drug, four of whom had important mast-cell cytoreduction and two who had complete clinical and histological remission. In the five patients with eosinophilia, three had complete clinical and haematological remission. The other two, who did not respond to treatment, were the only patients with the ckit D816V mutation. Our results suggest that imatinib either inhibits the growth-promoting role of wild type ckit, or targets an oncogenic kinase.

Publication types

  • Clinical Trial

MeSH terms

  • Adult
  • Antineoplastic Agents / therapeutic use*
  • Benzamides
  • Drug Administration Schedule
  • Humans
  • Imatinib Mesylate
  • Mastocytosis, Systemic / drug therapy*
  • Mastocytosis, Systemic / enzymology
  • Mastocytosis, Systemic / genetics
  • Mutation
  • Piperazines / therapeutic use*
  • Protein-Tyrosine Kinases / antagonists & inhibitors
  • Proto-Oncogene Proteins c-kit / drug effects*
  • Proto-Oncogene Proteins c-kit / genetics
  • Pyrimidines / therapeutic use*
  • Treatment Outcome


  • Antineoplastic Agents
  • Benzamides
  • Piperazines
  • Pyrimidines
  • Imatinib Mesylate
  • Protein-Tyrosine Kinases
  • Proto-Oncogene Proteins c-kit