Evolution of clinical trial endpoints in chronic myeloid leukemia: Efficacious therapies require sensitive monitoring techniques

Leukemia Research - Tập 36 - Trang 664-671 - 2012
Raoul Tibes1, Ruben A. Mesa1
1Mayo Clinic, Scottsdale, AZ, United States

Tài liệu tham khảo

Druker, 2008, Translation of the Philadelphia chromosome into therapy for CML, Blood, 112, 4808, 10.1182/blood-2008-07-077958 Vardiman, 2009, Chronic myelogenous leukemia, BCR-ABL1+, Am J Clin Pathol, 132, 250, 10.1309/AJCPUN89CXERVOVH Kantarjian, 2007, Dasatinib or high-dose imatinib for chronic-phase chronic myeloid leukemia after failure of first-line imatinib: a randomized phase 2 trial, Blood, 109, 5143, 10.1182/blood-2006-11-056028 Kantarjian, 2010, Dasatinib versus imatinib in newly diagnosed chronic-phase chronic myeloid leukemia, N Engl J Med, 362, 2260, 10.1056/NEJMoa1002315 Kantarjian, 2007, Nilotinib (formerly AMN107), a highly selective BCR-ABL tyrosine kinase inhibitor, is effective in patients with Philadelphia chromosome-positive chronic myelogenous leukemia in chronic phase following imatinib resistance and intolerance, Blood, 110, 3540, 10.1182/blood-2007-03-080689 O’Brien, 2003, Imatinib compared with interferon and low-dose cytarabine for newly diagnosed chronic-phase chronic myeloid leukemia, N Engl J Med, 348, 994, 10.1056/NEJMoa022457 Saglio, 2010, Nilotinib versus imatinib for newly diagnosed chronic myeloid leukemia, N Engl J Med, 362, 2251, 10.1056/NEJMoa0912614 McCain, 2010, The ongoing evolution of clinical endpoints in oncology, Manag Care, 19, 1 Food and Drug Administration. Guidance for industry clinical trial endpoints for the approval of cancer drugs and biologics, 2007. Available at: http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm071590.pdf [accessed 26.06.11]. Baccarani, 2009, Response definitions and European LeukemiaNet Management recommendations, Best Pract Res Clin Haematol, 22, 331, 10.1016/j.beha.2009.10.001 NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®). Chronic Myelogenous Leukemia (Version 2.2012). © 2012 National Comprehensive Cancer Network, Inc. Available at: NCCN.org. Radich, 2009, How I monitor residual disease in chronic myeloid leukemia, Blood, 114, 3376, 10.1182/blood-2009-02-163485 Baccarani, 2008, Monitoring treatment of chronic myeloid leukemia, Haematologica, 93, 161, 10.3324/haematol.12588 Foroni, 2009, Technical aspects and clinical applications of measuring BCR-ABL1 transcripts number in chronic myeloid leukemia, American Journal of Hematology, 84, 517, 10.1002/ajh.21457 Baccarani, 2009, Chronic myeloid leukemia: an update of concepts and management recommendations of European LeukemiaNet, J Clin Oncol, 27, 6041, 10.1200/JCO.2009.25.0779 Hughes, 2003, Frequency of major molecular responses to imatinib or interferon alfa plus cytarabine in newly diagnosed chronic myeloid leukemia, N Engl J Med, 349, 1423, 10.1056/NEJMoa030513 Hughes, 2006, Monitoring CML patients responding to treatment with tyrosine kinase inhibitors: review and recommendations for harmonizing current methodology for detecting BCR-ABL transcripts and kinase domain mutations and for expressing results, Blood, 108, 28, 10.1182/blood-2006-01-0092 Hehlmann, 1993, Randomized comparison of busulfan and hydroxyurea in chronic myelogenous leukemia: prolongation of survival by hydroxyurea. The German CML Study Group, Blood, 82, 398, 10.1182/blood.V82.2.398.398 Italian Cooperative Study Group on Chronic Myeloid Leukemia, 1994, Interferon alfa-2a as compared with conventional chemotherapy for the treatment of chronic myeloid leukemia. The Italian Cooperative Study Group on Chronic Myeloid Leukemia, N Engl J Med, 330, 820, 10.1056/NEJM199403243301204 Ohnishi, 1995, A randomized trial comparing interferon-alpha with busulfan for newly diagnosed chronic myelogenous leukemia in chronic phase, Blood, 86, 906, 10.1182/blood.V86.3.906.906 Druker, 2006, Five-year follow-up of patients receiving imatinib for chronic myeloid leukemia, N Engl J Med, 355, 2408, 10.1056/NEJMoa062867 Cortes, 2010, J Clin Oncol, 28, 424, 10.1200/JCO.2009.25.3724 O’Hare, 2005, In vitro activity of Bcr-Abl inhibitors AMN107 and BMS-354825 against clinically relevant imatinib-resistant Abl kinase domain mutants, Cancer Res, 65, 4500, 10.1158/0008-5472.CAN-05-0259 Weisberg, 2005, Characterization of AMN107, a selective inhibitor of native and mutant Bcr-Abl, Cancer Cell, 7, 129, 10.1016/j.ccr.2005.01.007 Kantarjian, 2011, Nilotinib is effective in patients with chronic myeloid leukemia in chronic phase after imatinib resistance or intolerance: 24-month follow-up results, Blood, 117, 1141, 10.1182/blood-2010-03-277152 Nicolini, 2011, Expanding Nilotinib Access in Clinical Trials (ENACT): an open-label, multicenter study of oral nilotinib in adult patients with imatinib-resistant or imatinib-intolerant Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase, Cancer, 118, 118, 10.1002/cncr.26249 Hochhaus, 2009, Dasatinib-associated major molecular responses in patients with chronic myeloid leukemia in chronic phase following imatinib failure: response dynamics and predictive value, Leukemia, 23, 1628, 10.1038/leu.2009.156 Kantarjian, 2009, Dasatinib or high-dose imatinib for chronic-phase chronic myeloid leukemia resistant to imatinib at a dose of 400 to 600 milligrams daily: two-year follow-up of a randomized phase 2 study (START-R), Cancer, 115, 4136, 10.1002/cncr.24504 Cortes, 2011, Safety and efficacy of bosutinib (SKI-606) in chronic phase Philadelphia chromosome-positive CML patients with resistance or intolerance to imatinib, Blood, 118, 4567, 10.1182/blood-2011-05-355594 Rosti, 2009, Nilotinib for the frontline treatment of Ph(+) chronic myeloid leukemia, Blood, 114, 4933, 10.1182/blood-2009-07-232595 Cortes, 2010, Nilotinib as front-line treatment for patients with chronic myeloid leukemia in early chronic phase, J Clin Oncol, 28, 392, 10.1200/JCO.2009.25.4896 Conneally, 2010, Nilotinib 300mg twice daily as first line treatment of Ph-positive chronic myeloid leukemia in chronic phase: updated results of the ICORG 0802 phase 2 study with analysis of the GeneXpert system versus IS BCR-ABL RQ PCR, Blood, 116, 3427, 10.1182/blood.V116.21.3427.3427 Kantarjian, 2011, Nilotinib versus imatinib for the treatment of patients with newly diagnosed chronic phase, Philadelphia chromosome-positive, chronic myeloid leukaemia: 24-month minimum follow-up of the phase 3 randomised ENESTnd trial, Lancet Oncol, 12, 841, 10.1016/S1470-2045(11)70201-7 Cortes, 2010, Results of dasatinib therapy in patients with early chronic-phase chronic myeloid leukemia, J Clin Oncol, 28, 398, 10.1200/JCO.2009.25.4920 Radich, 2010, A randomized phase II trial of dasatinib 100mg vs imatinib 400mg in newly diagnosed chronic myeloid leukemia in chronic phase (CML-CP): the S0325 intergroup trial, Blood, 116, 10.1182/blood.V116.21.LBA-6.LBA-6 Shah, 2010, Dasatinib versus imatinib in patients with newly diagnosed chronic myeloid leukemia in chronic phase (CML-CP) in the DASISION trial: 18-month follow-up, Blood, 116, 206, 10.1182/blood.V116.21.206.206 Gambacorti-Passerini, 2010, An ongoing phase 3 study of bosutinib (SKI-606) versus imatinib in patients with newly diagnosed chronic phase chronic myeloid leukemia, Blood, 116, 208, 10.1182/blood.V116.21.208.208 Gambacorti-Passerini, 2011, Bosutinib (BOS) versus imatinib (IM) in patients (pts) with chronic phase chronic myeloid leukemia (CP CML) in the BELA trial: 18-month follow-up, ASCO Meeting Abstracts, 29, 6509 Hughes, 2010, ENESTnd update: continued superiority of nilotinib versus imatinib in patients with newly diagnosed chronic myeloid leukemia in chronic phase (CML-CP), Blood, 116, 207, 10.1182/blood.V116.21.207.207 Kantarjian, 2011, Dasatinib or imatinib (IM) in newly diagnosed chronic myeloid leukemia in chronic phase (CML-CP): two-year follow-up from DASISION, ASCO Meeting Abstracts, 29, 6510 Kantarjian, 2003, Complete cytogenetic and molecular responses to interferon-alpha-based therapy for chronic myelogenous leukemia are associated with excellent long-term prognosis, Cancer, 97, 1033, 10.1002/cncr.11223 Deininger, 2009, International randomized study of interferon versus STI571 (IRIS) 8-year follow up: sustained survival and low risk for progression or events in patients with newly diagnosed chronic myeloid leukemia in chronic phase (CML-CP) treated with imatinib, Blood, 114, 1126, 10.1182/blood.V114.22.1126.1126 Hughes, 2010, Long-term prognostic significance of early molecular response to imatinib in newly diagnosed chronic myeloid leukemia: an analysis from the International Randomized Study of Interferon versus STI571 (IRIS), Blood, 116, 3758, 10.1182/blood-2010-03-273979 Hehlmann, 2011, Tolerability-adapted imatinib 800mg/d versus 400mg/d versus 400mg/d plus interferon-{alpha} in newly diagnosed chronic myeloid leukemia, J Clin Oncol, 29, 1634, 10.1200/JCO.2010.32.0598 Hanfstein, 2011, Dynamics of mutant BCR-ABL-positive clones after cessation of tyrosine kinase inhibitor therapy, Haematologica, 96, 360, 10.3324/haematol.2010.030999 Kim, 2010, Comprehensive evaluation of time-to-response parameter as a predictor of treatment failure following imatinib therapy in chronic phase chronic myeloid leukemia: which parameter at which time-point does matter?, Am J Hematol, 85, 856, 10.1002/ajh.21850 Naqvi, 2011, Kinetics of molecular response with different tyrosine kinase inhibitors (TKI) used as frontline therapy in chronic myeloid leukemia-chronic phase (CML CP), Blood, 118, 3784, 10.1182/blood.V118.21.3784.3784 Hochhaus, 2011, Blood, 118, 2767, 10.1182/blood.V118.21.2767.2767 Hanfstein, 2011, Molecular and cytogenetic response after 3months of imatinib treatment is predictive for the risk of disease progression and death in newly diagnosed chronic myeloid leukemia patients—a follow-up analysis of the German CML Study IV, Blood, 118, 783, 10.1182/blood.V118.21.783.783 Mahon, 2010, Discontinuation of imatinib in patients with chronic myeloid leukaemia who have maintained complete molecular remission for at least 2 years: the prospective, multicentre Stop Imatinib (STIM) trial, Lancet Oncol, 11, 1029, 10.1016/S1470-2045(10)70233-3 Rea, 2011, Cessation of dasatinib or nilotinib therapy in chronic-phase chronic myeloid leukaemia patients with sustained complete molecular responses Ross, 2011, Durable complete molecular remission of chronic myeloid leukemia following dasatinib cessation, despite adverse disease features, Haematologica, 96, 1720, 10.3324/haematol.2011.048165 Noens, 2009, Prevalence, determinants, and outcomes of nonadherence to imatinib therapy in patients with chronic myeloid leukemia: the ADAGIO study, Blood, 113, 5401, 10.1182/blood-2008-12-196543 Marin, 2010, Adherence is the critical factor for achieving molecular responses in patients with chronic myeloid leukemia who achieve complete cytogenetic responses on imatinib, J Clin Oncol, 28, 2381, 10.1200/JCO.2009.26.3087 Ibrahim, 2011, Poor adherence is the main reason for loss of CCyR and imatinib failure for chronic myeloid leukemia patients on long-term therapy, Blood, 117, 3733, 10.1182/blood-2010-10-309807 Faderl, 1999, Chronic myelogenous leukemia: biology and therapy, Ann Intern Med, 131, 207, 10.7326/0003-4819-131-3-199908030-00008