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

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• 作者：Gillian M. Keating ; Sheridan M. Hoy
• 刊名：Drugs & Therapy Perspectives
• 出版年：2016
• 出版时间：January 2016
• 年：2016
• 卷：32
• 期：1
• 页码：13-17
• 全文大小：405 KB
• 参考文献：1.O’Hare T, Deininger MWN, Eide CA, et al. Targeting the BCR-ABL signaling pathway in therapy-resistant Philadelphia chromosome-positive leukemia. Clin Cancer Res. 2011;17(2):212–21.PubMed CrossRef
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  4.O’Hare T, Shakespeare WC, Zhu X, et al. AP24534, a pan-BCR-ABL inhibitor for chronic myeloid leukemia, potently inhibits the T315I mutant and overcomes mutation-based resistance. Cancer Cell. 2009;16(5):401–12.PubMed PubMedCentral CrossRef
  5.Iclusig (ponatinib): EU summary of product characteristics. London: European Medicines Agency; 2015.
  6.Cortes JE, Kantarjian H, Shah NP, et al. Ponatinib in refractory Philadelphia chromosome-positive leukemias. N Engl J Med. 2012;367(22):2075–88.PubMed PubMedCentral CrossRef
  7.Cortes JE, Kim D-W, Pinilla-Ibarz J, et al. A phase 2 trial of ponatinib in Philadelphia chromosome-positive leukemias. N Engl J Med. 2013;369(19):1783–96.PubMed CrossRef
  8.Cortes JE, Kim D-W, Pinilla-Ibarz J, et al. Long-term follow-up of ponatinib efficacy and safety in the phase 2 PACE trial [abstract no. 3135 plus poster]. In: 56th ASH Annual Meeting and Exposition; 2014.
  9.Mueller MC, Baccarani M, Deininger MW, et al. Achieving early landmark response is predictive of outcomes in heavily pretreated patients with chronic phase chronic myeloid leukemia (CP-CML) treated with ponatinib [abstract no. 518]. In: 56th ASH Annual Meeting and Exposition; 2014.
  10.Hochhaus A, Kim D-W, Pinilla-Ibarz J, et al. Molecular responses with ponatinib in patients with Philadelphia chromosome positive (Ph+) leukemia: results from the PACE trial [abstract no. 3763 plus poster]. Blood. 2012;120(21).
  11.Knickerbocker R, Dorer DJ, Haluska FG, et al. Impact of dose intensity of ponatinib on selected adverse events: multivarirate analyses from a pooled population of clinical trial patients [abstract no. 4546]. In: 56th ASH Annual Meeting and Exposition; 2014.
  12.Ariad Pharmaceuticals. Ponatinib in patients with resistant chronic phase chronic myeloid leukemia (CML) to characterize the efficacy and safety of a range of doses (OPTIC) [ClinicalTrials.gov identifier NCT02467270]. US National Institutes of Health. Clinical Trials.gov. https://​clinicaltrials.​gov/​ct2/​show/​NCT02467270 . Accessed 30 Sep 2015.
  13.Hoy SM. Ponatinib: a review of its use in adults with chronic myeloid leukaemia or Philadelphia chromosome-positive acute lymphoblastic leukaemia. Drugs. 2014;74(7):793–806.PubMed CrossRef
  
• 作者单位：Gillian M. Keating (1)
  Sheridan M. Hoy (1)
  
  1. Springer, Private Bag 65901, Mairangi Bay, 0754, Auckland, New Zealand
  
• 刊物主题：Pharmacy; Pharmacotherapy;
• 出版者：Springer International Publishing
• ISSN：1179-1977

文摘

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.