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Chronic Myeloproliferative Neoplasias

Nilotinib is effective in imatinib-resistant or -intolerant patients with chronic myeloid leukemia in blastic phase

Leukemia volume 26, pages 959–962 (2012)Cite this article

Abstract

Nilotinib is a selective inhibitor of BCR-ABL approved for use in newly diagnosed and imatinib-resistant or -intolerant patients with chronic myeloid leukemia (CML) in chronic phase. In this study, 400 mg of nilotinib was administered twice daily to the patients with myeloid (MBP, n=105) or lymphoid blastic phase (LBP, n=31) CML. After a minimum follow-up of 24 months, major hematologic responses were observed in 60% (MBP) and 59% (LBP) of patients. Major cytogenetic responses (MCyR) were attained in 38% (MBP) and 52% (LBP) of patients; and complete cytogenetic responses in 30% and 32%, respectively. Median duration of MCyR was 10.8 (MBP) and 3.2 months (LBP). Median overall survival was 10.1 (MBP) and 7.9 (LBP) months with 12- and 24-month survival of 42% (MBP 44%, LBP 35%) and 27% (MBP 32%, LBP 10%), respectively. Twelve MBP patients and two LBP patients received subsequent stem cell transplantation. Myelosuppression was frequent, with grade 3/4 neutropenia, thrombocytopenia, and anemia in 68%, 63% and 47% of patients, respectively. Grade 3/4 hypophosphatemia, hyperbilirubinemia and lipase elevation were observed in 15%, 11% and 11% of patients, respectively. Nilotinib has significant efficacy in patients with BP CML, but given the limited long-term survival of these patients, novel agents are needed.

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Acknowledgements

Research grant was supplied by Novartis Pharmaceuticals.

Author information

Authors and Affiliations

  1. HRB Clinical Research Facility, National University of Ireland Galway & Trinity College Dublin,, University Road, Galway, Ireland

    F J Giles

  2. Department of Leukemia,

    H M Kantarjian

  3. M.D Anderson Cancer Center, Houston, TX, USA

    H M Kantarjian

  4. Charité-Universitätsmedizin Berlin, Berlin, Germany

    P D le Coutre

  5. Institute of Hematology and Medical Oncology ‘L. e A. Seràgnoli,’ University of Bologna, Bologna, Italy

    M Baccarani

  6. Department of Leukemia, Universite Victor Segalen Bordeaux 2, Bordeaux Cedex, France

    F-X Mahon

  7. Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA

    R E Blakesley & K Gillis

  8. Novartis Pharma AG, Basel, Switzerland

    N J Gallagher

  9. Division of Leukemia, Hackensack University Medical Center, Hackensack, NJ, USA

    S L Goldberg

  10. University of Chicago, Chicago, IL, USA

    R A Larson

  11. Universitätsklinikum Jena, Jena, Germany

    A Hochhaus

  12. Johann Wolfgang Goethe Universität, Frankfurt, Germany

    O G Ottmann

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Correspondence to F J Giles.

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Competing interests

FJG, MB, F-XM, SLG, RAL, AH and OGO acted as a consultant for and received research funding and honoraria from Novartis; HMK acted as a consultant for and received research funding from Novartis; PD.LeC received research funding and honoraria from Novartis; REB, NJG and KG are employees of Novartis Pharmaceuticals.

Additional information

Data contained within this article were presented at the June 2010 European Hematology Association 14th Annual Congress in Berlin, Germany.

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Giles, F., Kantarjian, H., le Coutre, P. et al. Nilotinib is effective in imatinib-resistant or -intolerant patients with chronic myeloid leukemia in blastic phase. Leukemia 26, 959–962 (2012). https://doi.org/10.1038/leu.2011.355

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