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

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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 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.

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Author notes

  1. Roberto H. Nussenzveig

    Present address: ARUP Laboratories, Salt Lake City, UT, USA

Authors and Affiliations

  1. Department of Leukemia, M.D. Anderson Cancer Center, Box 428, 1515 Holcombe Boulevard, Houston, TX, 77030, USA

    Roberto H. Nussenzveig, Jorge Cortes, Matjaz Sever, Alfonso Quintás-Cardama, Pat Ault, Taghi Manshouri, Hagop Kantarjian & Srdan Verstovsek

  2. Department of Hematopathology, University of Texas M.D. Anderson Cancer Center, Houston, TX, USA

    Carlos Bueso-Ramos

  3. Division of Hematology, School of Medicine, University of Utah, Salt Lake City, UT, USA

    Josef T. Prchal

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  1. Roberto H. Nussenzveig
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Correspondence to Srdan Verstovsek.

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Nussenzveig, R.H., Cortes, J., Sever, M. et al. Imatinib mesylate therapy for polycythemia vera: final result of a phase II study initiated in 2001. Int J Hematol 90, 58–63 (2009). https://doi.org/10.1007/s12185-009-0345-y

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  • DOI: https://doi.org/10.1007/s12185-009-0345-y

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