International Journal of Hematology

, Volume 90, Issue 1, pp 58–63

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

Authors

  • Roberto H. Nussenzveig
    • Department of LeukemiaM.D. Anderson Cancer Center
    • ARUP Laboratories
  • Jorge Cortes
    • Department of LeukemiaM.D. Anderson Cancer Center
  • Matjaz Sever
    • Department of LeukemiaM.D. Anderson Cancer Center
  • Alfonso Quintás-Cardama
    • Department of LeukemiaM.D. Anderson Cancer Center
  • Pat Ault
    • Department of LeukemiaM.D. Anderson Cancer Center
  • Taghi Manshouri
    • Department of LeukemiaM.D. Anderson Cancer Center
  • Carlos Bueso-Ramos
    • Department of HematopathologyUniversity of Texas M.D. Anderson Cancer Center
  • Josef T. Prchal
    • Division of Hematology, School of MedicineUniversity of Utah
  • Hagop Kantarjian
    • Department of LeukemiaM.D. Anderson Cancer Center
    • Department of LeukemiaM.D. Anderson Cancer Center
Original Article

DOI: 10.1007/s12185-009-0345-y

Cite this article as:
Nussenzveig, R.H., Cortes, J., Sever, M. et al. Int J Hematol (2009) 90: 58. doi:10.1007/s12185-009-0345-y

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 JAK2V617F 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 mesylatePolycythemia veraJAK2 mutation

Copyright information

© The Japanese Society of Hematology 2009