nejmoa1306494:Ponatinib in Philadelphia Chromosome–Positive Leukemias.pptx

Ponatinib in Philadelphia Chromosome–Positive

1. Original Article A Phase 2 Trial of Ponatinib in Philadelphia Chromosome–Positive Leukemias J.E. Cortes, D.-W. Kim, J. Pinilla-Ibarz, P. le Coutre, R. Paquette, C. Chuah, F.E. Nicolini, J.F. Apperley, H.J. Khoury, M. Talpaz, J. DiPersio, D.J. DeAngelo, E. Abruzzese, D. Rea, M. Baccarani, M.C. Müller, C. Gambacorti-Passerini, S. Wong, S. Lustgarten, V.M. Rivera, T. Clackson, C.D. Turner, F.G. Haluska, F. Guilhot, M.W. Deininger, A. Hochhaus, T. Hughes, J.M. Goldman, N.P. Shah, H. Kantarjian, for the PACE Investigators N Engl J Med Volume 369(19):1783-1796 November 7, 2013

2. Study Overview Several tyrosine kinase inhibitors have activity in Ph-positive chronic and acute leukemia, but resistant disease and unacceptable side effects may limit efficacy. Ponatinib showed a high level of activity in a phase 2 study of tyrosine kinase inhibitor–resistant disease but was associated with arterial thrombosis.

3. Response to Ponatinib According to Type of Leukemia, Resistance to or Unacceptable Side Effects from Previous Treatment with Dasatinib or Nilotinib, and T3151 Mutation Status. Cortes JE et al. N Engl J Med 2013;369:1783-1796

4. Demographic and Clinical Characteristics of the Patients at Baseline. Cortes JE et al. N Engl J Med 2013;369:1783-1796

5. Treatment Status of the Study Patients at the Data Cutoff Point. Cortes JE et al. N Engl J Med 2013;369:1783-1796

6. Response According to Previous Therapy in Patients with Chronic-Phase CML. Cortes JE et al. N Engl J Med 2013;369:1783-1796

7. Treatment-Related Adverse Events. Cortes JE et al. N Engl J Med 2013;369:1783-1796

8. Conclusions Ponatinib had significant antileukemic activity across categories of disease stage and mutation status.