Original Article

International Journal of Hematology

, Volume 90, Issue 1, pp 58-63

First online:

Imatinib mesylate therapy for polycythemia vera: final result of a phase II study initiated in 2001

  • Roberto H. NussenzveigAffiliated withDepartment of Leukemia, M.D. Anderson Cancer CenterARUP Laboratories
  • , Jorge CortesAffiliated withDepartment of Leukemia, M.D. Anderson Cancer Center
  • , Matjaz SeverAffiliated withDepartment of Leukemia, M.D. Anderson Cancer Center
  • , Alfonso Quintás-CardamaAffiliated withDepartment of Leukemia, M.D. Anderson Cancer Center
  • , Pat AultAffiliated withDepartment of Leukemia, M.D. Anderson Cancer Center
  • , Taghi ManshouriAffiliated withDepartment of Leukemia, M.D. Anderson Cancer Center
  • , Carlos Bueso-RamosAffiliated withDepartment of Hematopathology, University of Texas M.D. Anderson Cancer Center
  • , Josef T. PrchalAffiliated withDivision of Hematology, School of Medicine, University of Utah
  • , Hagop KantarjianAffiliated withDepartment of Leukemia, M.D. Anderson Cancer Center

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Abstract

Polycythemia vera (PV) is a chronic myeloproliferative neoplasm (MPN) characterized by excessive production of red blood cells. Patients with PV are at a risk of thrombosis, bleeding, and transformation to myelofibrosis or acute myeloid leukemia. Therapy for PV is based on the use of phlebotomy, aspirin, and in high-risk patients, cytoreductive agents such as hydroxyurea. Anecdotal evidence suggests that imatinib mesylate, a selective tyrosine kinase inhibitor of ABL1, ARG, PDGFR, and KIT kinases has activity in PV. We conducted an open-label phase II clinical trial of imatinib at the standard dose of 400 mg daily in 24 patients with PV. The median duration of imatinib therapy was 5.1 months (range 0.2–86.4). Overall, 4 (17%) patients responded: one had a complete and three partial hematological response. The median time to response was 17.5 months (range 6–28), and the median duration of response was 17 months (range 9–68). No significant changes in JAK2 V617F mutation burden were noted during imatinib therapy when compared with pretreatment values (P = 0.46). Therapy with imatinib was generally well tolerated. Our data indicate that imatinib has minimal clinical activity in PV.

Keywords

Imatinib mesylate Polycythemia vera JAK2 mutation