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Ponatinib in chronic myeloid leukaemia and Philadelphia chromosome-positive acute lymphoblastic leukaemia: a guide to its use in the EU

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Abstract

Oral ponatinib (Iclusig®) is a novel kinase inhibitor that was designed to overcome resistance mutations in the ABL kinase domain, including T315I. In a phase 2 study, ponatinib therapy was associated with a major cytogenetic response within the first 12 months in >50 % of adults with chronic-phase chronic myeloid leukaemia (CML) and major haematological responses within the first 6 months in ≥50 % of adults with accelerated-phase CML and ≈34 % of adults with blast-phase CML or Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukaemia (ALL); responses were seen regardless of whether patients were resistant or intolerant to prior dasatinib or nilotinib, or had the T315I mutation. Ponatinib had a manageable tolerability profile; vascular occlusions, cardiac failure and pancreatitis have been reported with ponatinib. Ponatinib is a useful option for adults with chronic-, accelerated- or blast-phase CML or Ph+ ALL who are resistant or intolerant to prior tyrosine kinase inhibitor therapy or who have the T315I mutation.

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Acknowledgments

The review was updated from Drugs; 2014;74(7):793–806 [13], and was reviewed by: E. Abruzzese, Hematology, S. Eugenio Hospital, Tor Vergata University, Rome, Italy; M. Baccarani, Department of Hematology and Oncology, S. Orsola-Malpighi University Hospital, University of Bologna, Bologna, Italy. During the peer review process, the manufacturer of ponatinib was also offered an opportunity to review this article. Changes resulting from comments received were made on the basis of scientific and editorial merit.

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  1. Springer, Private Bag 65901, Mairangi Bay, 0754, Auckland, New Zealand

    Gillian M. Keating & Sheridan M. Hoy

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  1. Gillian M. Keating
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  2. Sheridan M. Hoy
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Correspondence to Gillian M. Keating.

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The preparation of this review was not supported by any external funding.

Conflict of interest

G.M. Keating and S.M. Hoy are salaried employees of Adis/Springer, are responsible for the article content and declare no conflicts of interest.

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Keating, G.M., Hoy, S.M. Ponatinib in chronic myeloid leukaemia and Philadelphia chromosome-positive acute lymphoblastic leukaemia: a guide to its use in the EU. Drugs Ther Perspect 32, 13–17 (2016). https://doi.org/10.1007/s40267-015-0265-2

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  • DOI: https://doi.org/10.1007/s40267-015-0265-2

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