Phase II study of imatinib mesylate as therapy for patients with systemic mastocytosis

Leuk Res. 2009 Nov;33(11):1481-4. doi: 10.1016/j.leukres.2008.12.020. Epub 2009 Feb 3.


Gain-of-function D816V point mutation within the kinase domain of the transmembrane receptor KIT is found in the great majority of patients with systemic mastocytosis (SM) and is attractive therapeutic target. Twenty patients with SM were enrolled during 2003-2005 in phase II clinical trial with imatinib mesylate (400mg daily), a KIT inhibitor. Median time on therapy was 9 months (range, 0.5-44+). Only one patient, with D816V KIT mutation-negative FIP1L1-PDGFRalpha-negative SM-HES, achieved complete remission (now lasting for 44 months). Six other patients reported symptomatic improvement, including two with D816V KIT mutation-positive SM (one reported improvement in diarrhea and the other in fatigue). Other patients had no benefit. Imatinib was relatively well tolerated. Our study confirms that imatinib therapy does not result in appreciable clinical activity in patients with D816V mutation-positive SM, but may result in a significant benefit in occasional patient with D816V mutation-negative SM.

Publication types

  • Clinical Trial, Phase II

MeSH terms

  • Adult
  • Aged
  • Antineoplastic Agents / adverse effects
  • Antineoplastic Agents / therapeutic use*
  • Benzamides
  • Female
  • Humans
  • Imatinib Mesylate
  • Male
  • Mastocytosis, Systemic / drug therapy*
  • Mastocytosis, Systemic / genetics
  • Middle Aged
  • Piperazines / adverse effects
  • Piperazines / therapeutic use*
  • Point Mutation
  • Proto-Oncogene Proteins c-kit / genetics
  • Pyrimidines / adverse effects
  • Pyrimidines / therapeutic use*
  • Treatment Outcome


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