Developmental Therapeutics Consortium report on study design effects on trial outcomes in chronic myeloid leukaemia

European Journal of Clinical Investigation

Volumn 42, Issue 9, 2012, Pages 1016-1026

  Giles, Francis ^a   Mahon, François Xavier ^b   Gjertsen, Bjorn ^c   Swords, Ronan ^d   Labar, Boris ^e   Turkina, Anna ^f   Rosti, Gianantonio ^g  

^a TRINITY COLLEGE DUBLIN   (Ireland)

^b BORDEAUX UNIVERSITY HOSPITAL   (France)

^c HAUKELAND UNIVERSITY HOSPITAL   (Norway)

^d UNIVERSITY OF MIAMI   (United States)

^e KBC Zagreb   (Croatia)

^f NATIONAL RESEARCH CENTER FOR HEMATOLOGY   (Russian Federation)

^g UNIVERSITY OF BOLOGNA   (Italy)

Author keywords

Chronic myeloid leukaemia; Dasatinib; Developmental Therapeutics Consortium; Imatinib; Nilotinib; Tyrosine kinase inhibitors

Indexed keywords

DASATINIB; IMATINIB; NILOTINIB; PROTEIN TYROSINE KINASE INHIBITOR;

ALOPECIA; ANEMIA; CHRONIC MYELOID LEUKEMIA; CLINICAL RESEARCH; COMORBIDITY; DIARRHEA; DRUG APPROVAL; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERABILITY; DRUG TREATMENT FAILURE; EDEMA; EYELID EDEMA; FACE EDEMA; FATIGUE; GENE MUTATION; HEADACHE; HUMAN; METHODOLOGY; MUSCLE SPASM; MYALGIA; NAUSEA; NEUTROPENIA; PERIPHERAL EDEMA; PHASE 3 CLINICAL TRIAL (TOPIC); PRACTICE GUIDELINE; PRIORITY JOURNAL; PRURITUS; RANDOMIZED CONTROLLED TRIAL (TOPIC); RASH; REVIEW; RISK FACTOR; STUDY DESIGN; SYSTEMATIC REVIEW; THROMBOCYTOPENIA; TREATMENT OUTCOME; TREATMENT RESPONSE; VOMITING;

CLINICAL TRIALS AS TOPIC; HUMANS; LEUKEMIA, MYELOGENOUS, CHRONIC, BCR-ABL POSITIVE; PIPERAZINES; PROTEIN KINASE INHIBITORS; PROTEIN-TYROSINE KINASES; PYRIMIDINES; RESEARCH DESIGN; THIAZOLES; TREATMENT OUTCOME;

EID: 84865177656     PISSN: 00142972     EISSN: 13652362     Source Type: Journal    
DOI: 10.1111/j.1365-2362.2012.02675.x     Document Type: Review

References (53)

1. Rosti G, Castagnetti F, Palandri F, Poerio A, Breccia M, Levato L et al. Nilotinib 400mg BID in early chronic phase Ph+ chronic myeloid leukemia: results at 2years of a phase II trial. Haematologica 2010;95:459. [abstract 1114].
2. Kantarjian HM, Giles FJ, Bhalla KN, Pinilla-Ibarz JA, Larson RA, Gattermann N et al. Nilotinib is effective in patients with chronic myeloid leukemia in chronic phase following imatinib resistance or intolerance: 24-month follow-up results. Blood 2011;117:1141-5.
3. Cortes JE, Jones D, O'Brien S, Jabbour E, Konopleva M, Ferrajoli A et al. Nilotinib as front-line treatment for patients with chronic myeloid leukemia in early chronic phase. J Clin Oncol 2010;28:392-7.
4. Cortes JE, Jones D, O'Brien S, Jabbour E, Ravandi F, Koller C et al. Results of dasatinib therapy in patients with early chronic-phase chronic myeloid leukemia. J Clin Oncol 2010;28:398-404.
5. Shah NP, Cortes JE, Schiffer CA, Guilhot F, Brummendorf TH, Chen AC et al. Five-year follow-up of patients with imatinib-resistant or -intolerant chronic-phase chronic myeloid leukemia (CML-CP) receiving dasatinib. J Clin Oncol 2011;29:114. [abstract 6512].
6. Tasigna [package insert]. East Hanover, NJ, USA: Novartis Pharmaceuticals Corporation; 2010.
7. Weisberg E, Manley P, Mestan J, Cowan-Jacob S, Ray A, Griffin JD. AMN107 (nilotinib): a novel and selective inhibitor of BCR-ABL. Br J Cancer 2006;94:1765-9.
8. Weisberg E, Manley PW, Breitenstein W, Bruggen J, Cowan-Jacob SW, Ray A et al. Characterization of AMN107, a selective inhibitor of native and mutant Bcr-Abl. Cancer Cell 2005;7:129-41.
9. O'Hare T, Walters DK, Deininger MW, Druker BJ. AMN107: tightening the grip of imatinib. Cancer Cell 2005;7:117-9.
10. O'Hare T, Walters DK, Stoffregen EP, Jia T, Manley PW, Mestan J et al. In vitro activity of Bcr-Abl inhibitors AMN107 and BMS-354825 against clinically relevant imatinib-resistant Abl kinase domain mutants. Cancer Res 2005;65:4500-5.
11. Hughes T, Saglio G, Branford S, Soverini S, Kim DW, Muller MC et al. Impact of baseline BCR-ABL mutations on response to nilotinib in patients with chronic myeloid leukemia in chronic phase. J Clin Oncol 2009;27:4204-10.
12. Melnick JS, Janes J, Kim S, Chang JY, Sipes DG, Gunderson D et al. An efficient rapid system for profiling the cellular activities of molecular libraries. Proc Natl Acad Sci USA 2006;103:3153-8.
13. Das J, Chen P, Norris D, Padmanabha R, Lin J, Moquin RV et al. 2-aminothiazole as a novel kinase inhibitor template. Structure-activity relationship studies toward the discovery of N-(2-chloro-6-methylphenyl)-2-[[6-[4-(2-hydroxyethyl)-1- piperazinyl)]-2-methyl-4-pyrimidinyl]amino)]-1, 3-thiazole-5-carboxamide (dasatinib, BMS-354825) as a potent pan-Src kinase inhibitor. J Med Chem 2006;49:6819-32.
14. Müller MC, Cortes J, Kim DW. Dasatinib efficacy in patients with chronic myeloid leukemia in chronic phase (CML-CP) and pre-existing BCR-ABL mutations. Blood 2008;112:87. [abstract 449].
15. Kantarjian HM, Hochhaus A, Saglio G, De Souza C, Flinn IW, Stenke L et al. 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 2011;12:841-51.
16. Hochhaus A, Shah N, Cortes J, Baccarani M, Bradley-Garelik B, Zhu C et al. Efficacy and safety of dasatinib compared with imatinib in patients with newly diagnosed chronic myeloid leukemia in chronic phase (CML-CP): minimum 24-month follow-up from the DASISION trial. Haematologica 2011;96:422. [abstract 1011].
17. Kantarjian H, Shah NP, Cortes JE, Baccarani M, Bradley-Garelik M, Zhu C et al. 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;29:421s. [abstract 6510].
18. Hasford J, Pfirrmann M, Hehlmann R, Allan NC, Baccarani M, Kluin-Nelemans JC et al. A new prognostic score for survival of patients with chronic myeloid leukemia treated with interferon alfa. Writing Committee for the Collaborative CML Prognostic Factors Project Group. J Natl Cancer Inst 1998;90:850-8.
19. Sokal JE, Baccarani M, Russo D, Tura S. Staging and prognosis in chronic myelogenous leukemia. Semin Hematol 1988;25:49-61.
20. Kantarjian H, Shah NP, Hochhaus A, Cortes J, Shah S, Ayala M et al. Dasatinib versus imatinib in newly diagnosed chronic-phase chronic myeloid leukemia. N Engl J Med 2010;362:2260-70.
21. Saglio G, Kim DW, Issaragrisil S, le Coutre P, Etienne G, Lobo C et al. Nilotinib versus imatinib for newly diagnosed chronic myeloid leukemia. N Engl J Med 2010;362:2251-9.
22. Baccarani M, Saglio G, Goldman J, Hochhaus A, Simonsson B, Appelbaum F et al. Evolving concepts in the management of chronic myeloid leukemia: recommendations from an expert panel on behalf of the European LeukemiaNet. Blood 2006;108:1809-20.
23. Hochhaus A, Saglio G, le Coutre PD, Kim DW, Etienne G, Zanichelli MA et al. Superior efficacy of nilotinib compared with imatinib in newly-diagnosed patients with chronic myeloid leukemia in chronic (CML-CP): ENESTND minimum 24-month follow-up. Haematologica 2011;96:203-4. [abstract 0484].
24. Hughes TP, Hochhaus A, Saglio G, Kim DW, Jootar S, le Coutre PD et al. ENESTnd update: continued superiority of nilotinib versus imatinib in patients with newly diagnosed chronic myeloid leukemia in chronic phase (CML-CP). Blood 2010;116:94-5. [abstract 0207].
25. Saglio G, Hochhaus A, Guilhot F, Gatterman N, Wang MC, De Souza C et al. Nilotinib is associated with fewer treatment failures and suboptimal responses vs imatinib in patients with newly diagnosed chronic myeloid leukemia in chronic phase (CML-CP): results from ENESTND. Haematologica 2011;96:58-9. [abstract 0145].
26. Larson RA, Kim D, Rosti G, Stenke L, Pasquini R, Hoenekopp A et al. 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;29:421s. [abstract 6511].
27. Druker BJ, Guilhot F, O'Brien SG, Gathmann I, Kantarjian H, Gattermann N et al. Five-year follow-up of patients receiving imatinib for chronic myeloid leukemia. N Engl J Med 2006;355:2408-17.
28. Cortes JE, Baccarani M, Guilhot F, Druker BJ, Branford S, Kim DW et al. Phase III, randomized, open-label study of daily imatinib mesylate 400mg versus 800mg in patients with newly diagnosed, previously untreated chronic myeloid leukemia in chronic phase using molecular end points: tyrosine kinase inhibitor optimization and selectivity study. J Clin Oncol 2010;28:424-30.
29. O'Brien SG, Guilhot F, Larson RA, Gathmann I, Baccarani M, Cervantes F et al. Imatinib compared with interferon and low-dose cytarabine for newly diagnosed chronic-phase chronic myeloid leukemia. N Engl J Med 2003;348:994-1004.
30. Hochhaus A, Drucker BJ, Larson RA, O'Brien SG, Gathmann I, Guilhot F. IRIS 6-year follow-up: sustained survival and declining annual rate of transformation in patients with newly diagnosed chronic myeloid leukemia in chronic phase (CML-CP) treated with imatinib. Blood 2007;110:15a-6a. [abstract 25].
31. Gorre ME, Mohammed M, Ellwood K, Hsu N, Paquette R, Rao PN et al. Clinical resistance to STI-571 cancer therapy caused by BCR-ABL gene mutation or amplification. Science 2001;293:876-80.
32. Jabbour E, Kantarjian H, Jones D, Talpaz M, Bekele N, O'Brien S et al. Frequency and clinical significance of BCR-ABL mutations in patients with chronic myeloid leukemia treated with imatinib mesylate. Leukemia 2006;20:1767-73.
33. Hochhaus A, Kreil S, Corbin AS, La Rosée P, Müller MC, Lahaye T et al. Molecular and chromosomal mechanisms of resistance to imatinib (STI571) therapy. Leukemia 2002;16:2190-6.
34. Lahaye T, Riehm B, Berger U, Paschka P, Müller MC, Kreil S et al. Response and resistance in 300 patients with BCR-ABL-positive leukemias treated with imatinib in a single center: a 4.5-year follow-up. Cancer 2005;103:1659-69.
35. Soverini S, Gnani A, De Bendittis C, Gugliotta G, Breccia M, Abruzzese E et al. BCR-ABL kinase domain mutations in imatinib and in second-generation tyrosine kinase inhibitor eras: a review of seven years of mutation analysis by the GIMEMA CML working party. Haematologica 2011;96:204-5. [abstract 0486].
36. Soverini S, Hochhaus A, Nicolini FE, Gruber F, Lange T, Saglio G et al. Bcr-Abl kinase domain mutation analysis in chronic myeloid leukemia patients treated with tyrosine kinase inhibitors: recommendations from an expert panel on behalf of European LeukemiaNet. Blood 2011;118:1208-15.
37. Deininger M, O'Brien SG, Guilhot F, Goldman JM, Hochhaus A, Hughes TP et al. International randomized study of interferon vs 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 2009;114:462. [abstract 1126].
38. Saglio G, Kantarjian H, Reiffers J, Jootar S, Kalaycio ME, Shibayama H et al. The incidence of BCR-ABL mutations in patients (pts) with newly diagnosed chronic myeloid leukemia (CML) in chronic phase (CP) treated with nilotinib or imatinib in ENESTnd: 24-month follow-up. J Clin Oncol 2011;29:419s. [abstract 6502].
39. Shah NP, Skaggs BJ, Branford S, Hughes TP, Nicoll JM, Paquette RL et al. Sequential ABL kinase inhibitor therapy selects for compound drug-resistant BCR-ABL mutations with altered oncogenic potency. J Clin Invest 2007;117:2562-9.
40. Bradeen HA, Eide CA, O'Hare T, Johnson KJ, Willis SG, Lee FY et al. Comparison of imatinib mesylate, dasatinib (BMS-354825), and nilotinib (AMN107) in an N-ethyl-N-nitrosourea (ENU)-based mutagenesis screen: high efficacy of drug combinations. Blood 2006;108:2332-8.
41. Gruber FX, Ernst T, Porkka K, Engh RA, Mikkola I, Maier J et al. Dynamics of the emergence of dasatinib and nilotinib resistance in imatinib-resistant CML patients. Leukemia 2012;26:172-7.
42. Mueller MC, Erben P, Ernst T. Molecular response according to type of preexisting BCR-ABL mutations after second line dasatinib therapy in chronic phase CML patients. Blood 2007;110:100a-1a.
43. Soverini S, Colarossi S, Gnani A, Castagnetti F, Rosti G, Bosi C et al. Resistance to dasatinib in Philadelphia-positive leukemia patients and the presence or the selection of mutations at residues 315 and 317 in the BCR-ABL kinase domain. Haematologica 2007;92:401-4.
44. Khorashad JS, Milojkovic D, Mehta P, Anand M, Ghorashian S, Reid AG et al. In vivo kinetics of kinase domain mutations in CML patients treated with dasatinib after failing imatinib. Blood 2008;111:2378-81.
45. Rosti G, Palandri F, Castagnetti F, Breccia M, Levato L, Gugliotta G et al. Nilotinib for the frontline treatment of Ph(+) chronic myeloid leukemia. Blood 2009;114:4933-8.
46. Rosti G, Castagnetti F, Gugliotta G, Breccia M, Levato L, Capucci A et al. Excellent outcomes at 3years with nilotinib 800mg daily in early chronic phase, Ph+ chronic myeloid leukemia (CML): results of a phase 2 GIMEMA CML WP clinical trial. Blood 2010;116:162. [abstract 0359].
47. Quintás-Cardama A, Kantarjian H, O'Brien S, Borthakur G, Bruzzi J, Munden R et al. Pleural effusion in patients with chronic myelogenous leukemia treated with dasatinib after imatinib failure. J Clin Oncol 2007;25:3908-14.
48. Hochhaus A, Baccarani M, Deininger M, Apperley JF, Lipton JH, Goldberg SL et al. Dasatinib induces durable cytogenetic responses in patients with chronic myelogenous leukemia in chronic phase with resistance or intolerance to imatinib. Leukemia 2008;22:1200-6.
49. Cortes J, Borthakur G, O'Brien S, Jones D, Jabbour E, Ravandi F et al. Efficacy of dasatinib in patients (pts) with previously untreated chronic myelogenous leukemia (CML) in early chronic phase (CML-CP). Blood 2009;114:143. [abstract 338].
50. Porkka K, Baccarani M, Cortes J, Hochhaus A, Kantarjian H, Shah N et al. Pleural effusion in patients with chronic-phase chronic myeloid leukemia (CML-CP) who received first-line dasatinib in the DASISION trial: patient characteristics, management, and outcomes. Haematologica 2011;96:126. [abstract 0692].
51. Laneuville P, Baccarani M, Cortes JE, Hochhaus A, Kantarjian H, Shah NP et al. Analysis of patients (pts) with chronic phase chronic myeloid leukemia (CML-CP) who develop pleural effusion on first-line dasatinib: management and outcomes. J Clin Oncol 2011;29:488. [abstract 6605].
52. Kantarjian H, Shah NP, Hochhaus A, Cortes JE, Shah S, Ayala M et al. Dasatinib compared to imatinib (IM) in patients (pts) with newly diagnosed chronic-phase chronic myelogenous leukemia in chronic phase (CML-CP): twelve-month efficacy and safety from the phase III DASISION study. J Clin Oncol 2010;28:953s. [abstract LBA6500].
53. Hochhaus A, Saglio G, Larson RA, Kim DW, Flinn IW, Goh YT et al. Nilotinib lowers the incidence of BCR-ABL mutations and improves the molecular response kinetics compared with imatinib in patients (pts) with newly diagnosed chronic myeloid leukemia (CML). Blood 2010;116:1406. [abstract 3431].