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Imatinib for systemic mast-cell disease.

A Pardanani1, M Elliott, T Reeder

  • 1Division of Hematology, Mayo Clinic, 200 First Street SW, Rochester, MN, USA.

Lancet (London, England)
|August 23, 2003
PubMed
Summary
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Imatinib effectively treats systemic mast-cell disease by targeting c-kit. Fifty percent of patients showed significant response, with some achieving complete remission, highlighting imatinib

Area of Science:

  • Hematology
  • Oncology
  • Pharmacology

Background:

  • Systemic mast-cell disease (SMCD) is a rare condition often driven by mutations in the c-kit proto-oncogene.
  • Imatinib is a tyrosine kinase inhibitor known to target c-kit and has shown efficacy in certain malignancies.

Purpose of the Study:

  • To evaluate the efficacy and safety of imatinib in adult patients with symptomatic systemic mast-cell disease.
  • To assess the response rates and clinical outcomes in patients treated with imatinib at different doses.

Main Methods:

  • A prospective study involving 12 adult patients with symptomatic SMCD.
  • Patients received imatinib at daily doses of 100 mg or 400 mg.
  • Response was assessed by clinical, histological, and hematological parameters, including mast cell cytoreduction and eosinophilia.

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Main Results:

  • Fifty percent (5 out of 10 assessable patients) demonstrated a measurable response to imatinib.
  • Four patients achieved significant mast cell cytoreduction, and two achieved complete clinical and histological remission.
  • Three out of five patients with eosinophilia achieved complete clinical and hematological remission. Non-responders possessed the c-kit D816V mutation.

Conclusions:

  • Imatinib demonstrates significant clinical activity in systemic mast-cell disease.
  • The drug appears effective against wild-type c-kit-driven disease, with limited efficacy in patients harboring the c-kit D816V mutation.
  • Imatinib may inhibit the growth-promoting role of wild-type c-kit or target other oncogenic kinases in SMCD.