Evolution of definitions of response, progression-free survival and event-free survival in front-line studies of chronic myeloid leukemia

References

• Faderl S, Talpaz M, Estrov Z, . The biology of chronic myeloid leukemia. N Engl J Med 1999;341:164–172.
   PubMed Web of Science ®Google Scholar
• National Comprehensive Cancer Network. NCCN Clinical Practice Guidelines in Oncology™. Chronic myelogenous leukemia. v.2.2012. Available from: http://www.nccn.org/professionals/physician_gls/f_guidelines.asp
   Google Scholar
• Baccarani M, Saglio G, Goldman J, . Evolving concepts in the management of chronic myeloid leukemia: recommendations from an expert panel on behalf of the European LeukemiaNet. Blood 2006;108:1809–1820.
   PubMed Web of Science ®Google Scholar
• Baccarani M, Cortes J, Pane F, . Chronic myeloid leukemia: an update of concepts and management recommendations of European Leukemia Net. J Clin Oncol 2009;27:6041–6051.
   PubMed Web of Science ®Google Scholar
• Goldman J. ABC of clinical haematology: chronic myeloid leukaemia. Br Med J 1997;314:657–660.
   PubMed Web of Science ®Google Scholar
• Talpaz M, Silver RT, Druker BJ, . Imatinib induces durable hematologic and cytogenetic responses in patients with accelerated phase chronic myeloid leukemia: results of a phase 2 study. Blood 2002;99:1928–1937.
   PubMed Web of Science ®Google Scholar
• The Italian Cooperative Study Group on Chronic Myeloid Leukemia. Interferon alfa-2a as compared with conventional chemotherapy for the treatment of chronic myeloid leukemia. N Engl J Med 1994;330:820–825.
   PubMed Web of Science ®Google Scholar
• O’Brien SG, Guilhot F, Larson RA, . Imatinib compared with interferon and low-dose cytarabine for newly diagnosed chronic-phase chronic myeloid leukemia. N Engl J Med 2003;348:994–1004.
   PubMed Web of Science ®Google Scholar
• Cortes JE, Jones D, O’Brien S, . Nilotinib as front-line treatment for patients with chronic myeloid leukemia in early chronic phase. J Clin Oncol 2010;28:392–397.
   PubMed Web of Science ®Google Scholar
• Cortes JE, Jones D, O’Brien S, . Results of dasatinib therapy in patients with early chronic-phase chronic myeloid leukemia. J Clin Oncol 2010;28:398–404.
   PubMed Web of Science ®Google Scholar
• Druker BJ, Guilhot F, O’Brien SG, . Five-year follow-up of patients receiving imatinib for chronic myeloid leukemia. N Engl J Med 2006;355:2408–2417.
   PubMed Web of Science ®Google Scholar
• Kantarjian H, Shah NP, Hochhaus A, . Dasatinib versus imatinib in newly diagnosed chronic-phase chronic myeloid leukemia. N Engl J Med 2010;362:2260–2270.
   PubMed Web of Science ®Google Scholar
• Saglio G, Kim D-W, Issaragrisil S, . Nilotinib versus imatinib for newly diagnosed chronic myeloid leukemia. N Engl J Med 2010;362:2251–2259.
   PubMed Web of Science ®Google Scholar
• Kantarjian H, O’Brien S, Jabbour E, . Impact of treatment end point definitions on perceived differences in long-term outcome with tyrosine kinase inhibitor therapy in chronic myeloid leukemia. J Clin Oncol 2011;29:3173–3178.
   PubMed Web of Science ®Google Scholar
• Press RD, Love Z, Tronnes AA, . BCR-ABL mRNA levels at and after the time of a complete cytogenetic response (CCR) predict the duration of CCR in imatinib mesylate treated patients with CML. Blood 2006;107:4250–4256.
   PubMed Web of Science ®Google Scholar
• Press RD, Galderisi C, Yang R, . A half-log increase in BCR-ABL RNA predicts a higher risk of relapse in patients with chronic myeloid leukemia with an imatinib-induced complete cytogenetic response. Clin Cancer Res 2007;13:6136–6143.
   PubMed Web of Science ®Google Scholar
• Hughes TP, Kaeda J, Branford S, . Frequency of major molecular responses to imatinib or interferon alfa plus cytarabine in newly diagnosed chronic myeloid leukemia. N Engl J Med 2003; 349:1423–1432.
   PubMed Web of Science ®Google Scholar
• Hughes T, Deininger M, Hochhaus A, . 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 2006;108:28–37.
   PubMed Web of Science ®Google Scholar
• Hughes TP, Branford S. Monitoring disease response to tyrosine kinase inhibitor therapy in CML. Hematology Am Soc Hematol Educ Program 2009:477–487.
   PubMedGoogle Scholar
• Deininger M, O’Brien SG, Guilhot F, . International Randomized Study of Interferon vs ST1571 (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 2009;114(Suppl. 1):Abstract 1126.
   Google Scholar
• Hughes TP, Hochhaus A, Branford S, ; IRIS Investigators. 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 and STI571 (IRIS). Blood 2010;116:3758–3765.
   PubMed Web of Science ®Google Scholar
• de Lavallade H, Apperley JF, Khorashad JS, . Imatinib for newly diagnosed patients with chronic myeloid leukemia: incidence of sustained responses in an intention-to-treat analysis. J Clin Oncol 2008;26:3358–3363.
   PubMed Web of Science ®Google Scholar
• Baccarani M, Rosti G, Castagnetti F, . Comparison of imatinib 400 mg and 800 mg daily in the front-line treatment of high-risk, Philadelphia-positive chronic myeloid leukemia: a European LeukemiaNet Study. Blood 2009;113:4497–4504.
   PubMed Web of Science ®Google Scholar
• Simonsson B, Porkka K, Richter J. Second-generation BCR-ABL kinase inhibitors in CML. N Engl J Med 2010;363:1673; author reply 1673–1675.
   PubMed Web of Science ®Google Scholar
• Kantarjian HM, Hochhaus A, Saglio G, . 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 Oncology 2011;12:841–851.
   PubMed Web of Science ®Google Scholar
• Larson RA, Kim D, Rosti G, . Comparison of nilotinib and imatinib in patients (pts) with newly diagnosed chronic myeloid leukemia in chronic phase (CML-CP): ENESTnd 24-month follow-up. J Clin Oncol 2011;29(Suppl.):Abstract 6511.
   Google Scholar
• A phase III study of dasatinib vs. imatinib in patients with newly diagnosed chronic phase CML (DASISION) [NCT00481247]. Available from: http://www.clinicaltrials.gov/nct00481247
   Google Scholar
• Kantarjian H, Shah NP, Cortes JE, . Dasatinib or imatinib (IM) in newly diagnosed chronic myeloid leukemia in chronic phase (CML-CP): two-year follow-up from DASISION. J Clin Oncol 2011;29(Suppl.):Abstract 6510.
   Google Scholar
• Freemantle N, Calvert M, Wood J, . Composite outcomes in randomized trials. Greater precision but with greater uncertainty? JAMA 2003;289:2554–2559.
   PubMed Web of Science ®Google Scholar
• Mell LK, Jeong J-H. Pitfalls of using composite primary end points in the presence of competing risks. J Clin Oncol 2010;28:4297–4299.
   PubMed Web of Science ®Google Scholar
• Craddock C, Szydlo R, Klein J, . Estimating leukemia-free survival after allografting for chronic myeloid leukemia: a new method that takes into account patients who relapse and are restored to complete remission. Blood 2000;96:86–90.
   PubMed Web of Science ®Google Scholar
• Al-Kali A, Kantarjian H, Shan J, . Current event-free survival after sequential tyrosine kinase inhibitor therapy for chronic myeloid leukemia. Cancer 2011;117:327–335.
   PubMed Web of Science ®Google Scholar
• Quintas-Cardama A, Cortes JE, O’Brien S, . Dasatinib early intervention after cytogenetic or hematologic resistance to imatinib in patients with chronic myeloid leukemia. Cancer 2009;115; 2912–2921.
   PubMed Web of Science ®Google Scholar
• Roy L, Guilhot J, Krahnke T, . Survival advantage from imatinib compared with the combination interferon-α plus cytarabine in chronic-phase chronic myelogenous leukemia: historical comparison between two phase 3 trials. Blood 2006;108:1478–1484.
   PubMed Web of Science ®Google Scholar
• Marin D, Milojkovic D, Olavarria E, . European Leukemia Net criteria for failure or sub-optimal response reliably identify patients with CML in early chronic phase treated with imatinib whose eventual outcome is poor. Blood 2008;112:4437–4444.
   PubMed Web of Science ®Google Scholar
• Jabbour E, Kantarjian H, O’Brien S, . The achievement of an early complete cytogenetic response is a major determinant for outcome in patients with early chronic phase chronic myeloid leukemia treated with tyrosine kinase inhibitors. Blood 2011;118:4541–4546; quiz 4759.
   PubMed Web of Science ®Google Scholar
• Kantarjian H, O’Brien S, Shan J, . Cytogenetic and molecular responses and outcome in chronic myelogenous leukemia. Need for new response definitions? Cancer 2008;112:837–845.
   PubMed Web of Science ®Google Scholar
• Palandri F, Iacobucci I, Soverini S, . Treatment of Philadelphia-positive chronic myeloid leukemia with imatinib: importance of a stable molecular response. Clin Cancer Res 2009;15:1059–1063.
   PubMed Web of Science ®Google Scholar
• Branford S, Fletcher L, Cross NCP, . Desirable performance characteristics of BCR-ABL measurement on an international reporting scale to allow consistent interpretation of individual patient response and comparison of response rates between clinical trials. Blood 2008;112:3330–3338.
   PubMed Web of Science ®Google Scholar