Core binding factor acute myeloid leukemia (CBF-AML): Is high-dose Ara-C (HDAC) consolidation as effective as you think?

Current Opinion in Hematology

Volumn 16, Issue 2, 2009, Pages 92-97

  Dombret, Hervé ^a   Preudhomme, Claude ^b   Boissel, Nicolas ^a  

^a SAINT LOUIS HOSPITAL   (France)

^b LILLE UNIVERSITY HOSPITAL   (France)

Author keywords

Acute myeloid leukemia; Core binding factor; Gene expression profiling; Kit mutations; Minimal residual disease

Indexed keywords

ANTHRACYCLINE; CORE BINDING FACTOR; CYTARABINE; DASATINIB; DAUNORUBICIN; ETOPOSIDE; GEMTUZUMAB OZOGAMICIN; IDARUBICIN; MITOXANTRONE; TIOGUANINE;

ACUTE GRANULOCYTIC LEUKEMIA; ALLOGENEIC STEM CELL TRANSPLANTATION; CANCER REGRESSION; CHEMOTHERAPY; CHROMOSOME TRANSLOCATION; CLINICAL FEATURE; CLINICAL TRIAL; DISEASE FREE SURVIVAL; DRUG DOSE COMPARISON; DRUG EFFICACY; DRUG MEGADOSE; GENE EXPRESSION; GENE EXPRESSION PROFILING; GENE MUTATION; GENETIC HETEROGENEITY; HUMAN; MULTIPLE CYCLE TREATMENT; PRIORITY JOURNAL; PROGNOSIS; REVIEW; TREATMENT OUTCOME;

CORE BINDING FACTORS; CYTARABINE; DOSE-RESPONSE RELATIONSHIP, DRUG; GENE EXPRESSION PROFILING; HUMANS; LEUKEMIA, MYELOID, ACUTE; MUTATION; PROGNOSIS;

EID: 62349107715     PISSN: 10656251     EISSN: 15317048     Source Type: Journal    
DOI: 10.1097/MOH.0b013e3283257b18     Document Type: Review

References (74)

1. Erickson P, Gao J, Chang KS, et al. Identification of breakpoints in t(8;21) acute myelogenous leukemia and isolation of a fusion transcript, AML1/ETO, with similarity to Drosophila segmentation gene, runt. Blood 1992; 80:1825-1831.
2. Liu P, Tarle SA, Hajra A, et al. Fusion between transcription factor CBF beta/PEBP2 beta and a myosin heavy chain in acute myeloid leukemia. Science 1993; 261:1041-1044.
3. Speck NA, Gilliland DG. Core-binding factors in haematopoiesis and leukaemias. Nat Rev Cancer 2002; 2:502-513.
4. Downing JR. The core-binding factor leukemias: lessons learned from murine models. Curr Opin Gen Dev 2003; 13:48-54.
5. Ravindranath Y, Chang M, Steuber CP, et al. Pediatric Oncology Group (POG) studies of acute myeloid leukemia (AML): a review of four consecutive childhood AML trials conducted between 1981 and 2000. Leukemia 2005; 19:2101-2116.
6. Grimwade D, Walker H, Harrison G, et al. The predictive value of hierarchical cytogenetic classification in older adults with acute myeloid leukemia (AML): analysis of 1065 patients entered into the United Kingdom Medical Research Council AML11 trial. Blood 2001; 98:1312-1320.
7. Grimwade D, Walker H, Oliver F, et al. The importance of diagnostic cytogenetics on outcome in AML: analysis of 1,612 patients entered into the MRC AML 10 trial. The Medical Research Council Adult and Children's Leukaemia Working Parties. Blood 1998; 92:2322-2333.
8. Slovak ML, Kopecky KJ, Cassileth PA, et al. Karyotypic analysis predicts outcome of preremission and postremission therapy in adult acute myeloid leukemia: a Southwest Oncology Group/Eastern Cooperative Oncology Group Study. Blood 2000; 96:4075-4083.
9. Byrd JC, Mrózek K, Dodge RK, et al. Pretreatment cytogenetic abnormalities are predictive of induction success, cumulative incidence of relapse, and overall survival in adult patients with de novo acute myeloid leukemia: results from Cancer and Leukemia Group B (CALGB 8461). Blood 2002; 100:4325-4336.
10. Marcucci G, Mrózek K, Ruppert AS, et al. Prognostic factors and outcome of core binding factor acute myeloid leukemia patients with t(8;21) differ from those of patients with inv(16): a Cancer and Leukemia Group B study. J Clin Oncol 2005; 23:5705-5717.
11. Appelbaum FR, Kopecky KJ, Tallman MS, et al. The clinical spectrum of adult acute myeloid leukaemia associated with core binding factor translocations. Br J Haematol 2006; 135:165-173.
12. Quesnel B, Kantarjian H, Bjergaard JP, et al. Therapy-related acute myeloid leukemia with t(8;21), inv(16), and t(8;16): a report on 25 cases and review of the literature. J Clin Oncol 1993; 11:2370-2379.
13. Andersen MK, Johansson B, Larsen SO, Pedersen-Bjergaard J. Chromosomal abnormalities in secondary MDS and AML. Relationship to drugs and radiation with specific emphasis on the balanced rearrangements. Haematologica 1998; 83:483-488.
14. Merzianu M, Medeiros LJ, Cortes J, et al. inv(16)(p13q22) in chronic myelogenous leukemia in blast phase: a clinicopathologic, cytogenetic, and molecular study of five cases. Am J Clin Pathol 2005; 124:807-814.
15. Wu Y, Slovak ML, Snyder DS, Arber DA. Coexistence of inversion 16 and the Philadelphia chromosome in acute and chronic myeloid leukemias: report of six cases and review of literature. Am J Clin Pathol 2006; 125:260-266.
16. Yanada M, Garcia-Manero G, Borthakur G, et al. Potential cure of acute myeloid leukemia: analysis of 1069 consecutive patients in first complete remission. Cancer 2007; 110:2756-2760.
17. Schlenk RF, Benner A, Krauter J, et al. Individual patient data-based meta-analysis of patients aged 16 to 60 years with core binding factor acute myeloid leukemia: a survey of the German Acute Myeloid Leukemia Intergroup. J Clin Oncol 2004; 22:3741-3750.
18. Bishop JF, Matthews JP, Young GA, et al. A randomized study of high-dose cytarabine in induction in acute myeloid leukemia. Blood 1996; 87:1710-2177.
19. Weick JK, Kopecky KJ, Appelbaum FR, et al. A randomized investigation of high-dose versus standard-dose cytosine arabinoside with daunorubicin in patients with previously untreated acute myeloid leukemia: a Southwest Oncology Group study. Blood 1996; 88:2841-2851.
20. Büchner T, Hiddemann W, Wörmann B, et al. Double induction strategy for acute myeloid leukemia: the effect of high-dose cytarabine with mitoxantrone instead of standard-dose cytarabine with daunorubicin and 6-thioguanine: a randomized trial by the German AML Cooperative Group. Blood 1999; 93:4116-4124.
21. Büchner T, Hiddemann W, Berdel WE, et al. 6-Thioguanine, cytarabine, and daunorubicin (TAD) and high-dose cytarabine and mitoxantrone (HAM) for induction, TAD for consolidation, and either prolonged maintenance by reduced monthly TAD or TAD-HAM-TAD and one course of intensive consolidation by sequential HAM in adult patients at all ages with de novo acute myeloid leukemia (AML): a randomized trial of the German AML Cooperative Group. J Clin Oncol 2003; 21:4496-4504.
22. Büchner T, Berdel WE, Schoch C, et al. Double induction containing either two courses or one course of high-dose cytarabine plus mitoxantrone and postremission therapy by either autologous stem-cell transplantation or by prolonged maintenance for acute myeloid leukemia. J Clin Oncol 2006; 24:2480-2489.
23. Woods WG, Kobrinsky N, Buckley JD, et al. Timed-sequential induction therapy improves postremission outcome in acute myeloid leukemia: a report from the Children's Cancer Group. Blood 1996; 87:4979-4989.
24. Castaigne S, Chevret S, Archimbaud E, et al. Randomized comparison of double induction and timed-sequential induction to a '3 + 7' induction in adults with AML: long-term analysis of the Acute Leukemia French Associa-tion (ALFA) 9000 study. Blood 2004; 104:2467-2474.
25. Burnett AK, Kell WJ, Goldstone AH, et al. The addition of gemtuzumab ozogamicin to induction chemotherapy for AML improves disease free survival without extra toxicity: preliminary analysis of 1115 patients in the MRC AML15 trial. Blood 2006; 108:13a.
26. Burnett AK, Wheatley K, Goldstone AH, et al. The value of allogeneic bone marrow transplant in patients with acute myeloid leukaemia at differing risk of relapse: results of the UK MRC AML 10 trial. Br J Haematol 2002; 118:385-400.
27. Suciu S, Mandelli F, De Witte T, et al. Allogeneic compared with autologous stem cell transplantation in the treatment of patients younger than 46 years with acute myeloid leukemia (AML) in first complete remission (CR1): an intention-to-treat analysis of the EORTC/GIMEMA AML-10 trial. Blood 2003; 102:1232-1240.
28. Jourdan E, Boiron J-M, Dastugue N, et al. Early allogeneic stem-cell transplantation for young adults with acute myeloblastic leukemia in first complete remission: an intent-to-treat long-term analysis of the BGMT experience. J Clin Oncol 2005; 23:7676-7684.
29. Cornelissen JJ, van Putten WLJ, Verdonck LF, et al. Results of a HOVON/ SAKK donor versus no-donor analysis of myeloablative HLA-identical sibling stem cell transplantation in first remission acute myeloid leukemia in young and middle-aged adults: benefits for whom? Blood 2007; 109:3658-3666. The above article shows the results of HOVON donor versus no-donor study, including a meia-analysis with the three other British Medical Research Council (MRC), European Organisation for Research and Treatment of Cancer/Gruppo Italiano Malattie Ematologiche dell'Adulto (EORTC/GIMEMA), and Bordeaux Grenoble Marseille Toulouse (BGMT)] donor versus no-donor studies.
30. Schlenk RF, Pasquini MC, Pérez WS, et al. HLA-identical sibling allogeneic transplants versus chemotherapy in acute myelogenous leukemia with t(8;21) in first complete remission: collaborative study between the German AML Intergroup and CIBMTR. Biol Blood Marrow Transplant 2008; 14:187-196.
31. de Labarthe A, Pautas C, Thomas X, et al. Allogeneic stem cell transplantation in second rather than first complete remission in selected patients with goodrisk acute myeloid leukemia. Bone Marrow Transplant 2005; 35:767-773.
32. Gorin N-C, Labopin M, Frassoni F, et al. Identical outcome after autologous or allogeneic genoidentical hematopoietic stem-cell transplantation in first remission of acute myelocytic leukemia carrying inversion 16 or t(8;21): a retrospective study from the European Cooperative Group for Blood and Marrow Transplantation. J Clin Oncol 2008; 26:3183-3188. The largest report on autologous transplantation in CBF-AML patients.
33. Mayer RJ, Davis RB, Schiffer CA, et al. Intensive postremission chemotherapy in adults with acute myeloid leukemia. N Engl J Med 1994; 331:896-903.
34. Bloomfield CD, Lawrence D, Byrd JC, et al. Frequency of prolonged remission duration after high-dose cytarabine intensification in acute myeloid leukemia varies by cytogenetic subtype. Cancer Res 1998; 58:4173-4179.
35. Bloomfield CD, Ruppert AS, Mrozek K, et al. Core binding factor acute myeloid leukemia. Cancer and Leukemia Group B (CALGB) Study 8461. Ann Hematol 2004; 83 (Suppl 1):S84-S85.
36. Byrd JC, Dodge RK, Carroll A, et al. Patients with t(8;21 )(q22;q22) and acute myeloid leukemia have superior failure-free and overall survival when repetitive cycles of high-dose cytarabine are administered. J Clin Oncol 1999; 17:3767-3775.
37. Byrd JC, Ruppert AS, Mrózek K, et al. Repetitive cycles of high-dose cytarabine benefit patients with acute myeloid leukemia and inv(16)(p13q22) or t(16;16)(p13;q22): results from CALGB 8461. J Clin Oncol 2004; 22:1087-1094.
38. Bradstock KF, Matthews JP, Lowenthal RM, et al. A randomized trial of high-versus conventional-dose cytarabine in consolidation chemotherapy for adult de novo acute myeloid leukemia in first remission after induction therapy containing high-dose cytarabine. Blood 2005; 105:481-488.
39. O'Brien S, Kantarjian HM, Keating M, et al. Association of granulocytosis with poor prognosis in patients with acute myelogenous leukemia and translocation of chromosomes 8 and 21. J Clin Oncol 1989; 7:1081-1086:
40. Tallman MS, Hakimian D, Shaw JM, et al. Granulocytic sarcoma is associated with the 8;21 translocation in acute myeloid leukemia. J Clin Oncol 1993; 11:690-697.
41. Baer MR, Stewart CC, Lawrence D, et al. Expression of the neural cell adhesion molecule CD56 is associated with short remission duration and survival in acute myeloid leukemia with t(8;21)(q22;q22). Blood 1997; 90:1643-1648.
42. Byrd JC, Weiss RB, Arthur DC, et al. Extramedullary leukemia adversely affects hematologic complete remission rate and overall survival in patients with t(8;21)(q22;q22): results from Cancer and Leukemia Group B 8461. J Clin Oncol 1997; 15:466-475.
43. Daniels JT, Davis BJ, Houde-McGrail L, Byrd JC. Clonal selection of CD56+ t(8;21) AML blasts: further suggestion of the adverse clinical significance of this biological marker? Br J Haematol 1999; 107:381-383.
44. Nguyen S, Leblanc T, Fenaux P, et al. A white blood cell index as the main prognostic factor in t(8;21) acute myeloid leukemia (AML): a survey of 161 cases from the French AML Intergroup. Blood 2002; 99:3517-3523.
45. Delaunay J, Vey N, Leblanc T, et al. Prognosis of inv(16)/t(16;16) acute myeloid leukemia (AML): a survey of 110 cases from the French AML Intergroup, Blood 2003; 102:462-469:
46. Narimatsu H, Yokozawa T, Iida H, et al. Clinical characteristics and outcomes in patients with t(8;21) acute myeloid leukemia in Japan. Leukemia 2008; 22:428-432.
47. Schoch C, Haase D, Haferlach T, et al. Fifty-one patients with acute myeloid leukemia and translocation t(8;21 )(q22;q22): an additional deletion in 9q is an adverse prognostic factor. Leukemia 1996; 10:1288-1295.
48. Beghini A, Peterlongo P, Ripamonti CB, et al. C-kit mutations in core binding factor leukemias. Blood 2000; 95:726-727.
49. Care RS, Valk PJM, Goodeve AC, et al. Incidence and prognosis of c-KIT and FLT3 mutations in core binding factor (CBF) acute myeloid leukaemias. Br J Haematol 2003; 121:775-777.
50. Goemans BF, Zwaan CM, Miller M, et al. Mutations in KIT and RAS are frequent events in pediatric core-binding factor acute myeloid leukemia. Leukemia 2005; 19:1536-1542.
51. Boissel N, Laroy H, Brethon B, et al. Incidence and prognostic impact of e-Kit, FLT3, and Ras gene mutations in core binding factor acute myeloid leukemia (CBF-AML). Leukemia 2006; 20:965-970.
52. Schnittger S, Kohl TM, Haferlach T, et al. KIT-D816 mutations in AML1 -ETO-positive AML are associated with impaired event-free and overall survival. Blood 2006; 107:1791-1799.
53. Cairoli R, Beghini A, Grillo G, et al. Prognostic impact of c-KIT mutations in core binding factor leukemias: an Italian retrospective study. Blood 2006; 107:3463-3468.
54. Cairoli R, Beghini A, Ripamonti CB, et al. Prevalence and prognostic impact of KIT mutations in acute myeloid leukaemia with inv(16). A retrospective study. Blood 2007; 110:1021a-1022a.
55. Shimada A, Taki T, Tabuchi K, et al. KIT mutations, and not FLT3 internal tandem duplication, are strongly associated with a poor prognosis in pediatric acute myeloid leukemia with t(8;21): a study of the Japanese Childhood AML Cooperative Study Group. Blood 2006; 107:1806-1809.
56. Shimada A, Ichikawa H, Taki T, et al. Low frequency of KIT gene mutation in pediatric acute myeloid leukemia with inv(16)(p13q22): a study of the Japanese Childhood AML Cooperative Study Group, Int J Hematol 2007; 86:289-290.
57. Paschka P, Marcucci G, Ruppert AS, et al. Adverse prognostic significance of KIT mutations in adult acute myeloid leukemia with inv(16) and t(8;21): a Cancer and Leukemia Group B Study. J Clin Oncol 2006; 24:3904-3911.
58. Shih LY, Liang DC, Huang CF, et al. Cooperating mutations of receptor tyrosine kinases and Ras genes in childhood core-binding factor acute myeloid leukemia and a comparative analysis on paired diagnosis and relapse samples. Leukemia 2008; 22:303-307.
59. Bacher U, Haferlach T, Schoch C, et al. Implications of NRAS mutations in AML: a study of 2502 patients. Blood 2006; 107:3847-3853.
60. Schoch C, Kohlmann A, Schnittger S, et al. Acute myeloid leukemias with reciprocal rearrangements can be distinguished by specific gene expression profiles. Proc Natl Acad Sci U S A 2002; 99:10008-10013.
61. Bullinger L, Dohner K, Bair E, et al. Use of gene-expression profiling to identify prognostic subclasses in adult acute myeloid leukemia. N Engl J Med 2004; 350:1605-1616.
62. Valk PJ, Verhaak RG, Beijen MA, et al. Prognostically useful gene-expression profiles in acute myeloid leukemia. N Engl J Med 2004; 350:1617-1628.
63. Haferlach T, Kohlmann A, Schnittger S, et al. Global approach to the diagnosis of leukemia using gene expression profiling. Blood 2005; 106:1189-1198.
64. Bullinger L, Rücker FG, Kurz S, et al. Gene-expression profiling identifies distinct subclasses of core binding factor acute myeloid leukemia. Blood 2007; 110:1291-1300. A study which shows CBF-AML GEP heterogeneity.
65. Paschka P, Radmacher MD, Marcucci G, et al. Gene expression profiling improves outcome prediction in adult core binding factor (CBF) acute myeloid leukemia (AML): a Cancer and Leukemia Group B (CALGB) study. J Clin Oncol 2007; 25:359a. A very interesting study using gene expression profiling as a prognostic tool in CBF-AML patients.
66. Jongen-Lavrencic M, Sun SM, Dijkstra MK, et al. MicroRNA expression profiling in relation to the genetic heterogeneity of acute myeloid leukemia. Blood 2008; 111:5078-5085. The first large study of microRNA expression profiling in AML.
67. Morschhauser F, Cayuela JM, Martini S, et al. Evaluation of minimal residual disease using reverse-transcription polymerase chain reaction in t(8;21) acute myeloid leukemia: a multicenter study of 51 patients. J Clin Oncol 2000; 18:788-794.
68. Guerrasio A, Pilatrino C, De Micheli D, et al. Assessment of minimal residual disease (MRD) in CBFbeta/MYH11-positive acute myeloid leukemias by qualitative and quantitative RT-PCR amplification of fusion transcripts. Leukemia 2002; 16:1176-1181.
69. Krauter J, Gorlich K, Ottmann O, et al. Prognostic value of minimal residual disease quantification by real-time reverse transcriptase polymerase chain reaction in patients with core binding factor leukemias. J Clin Oncol 2003; 21:4413-4422.
70. Schnittger S, Weisser M, Schoch C, et al. New score predicting for prognosis in PML-RARA+, AML1-ETO+, or CBFBMYH11+ acute myeloid leukemia based on quantification of fusion transcripts. Blood 2003; 102:2746-2755.
71. Leroy H, de Botton S, Grardel-Duflos N, et al. Prognostic value of real-time quantitative PCR (RQ-PCR) in AML with t(8;21). Leukemia 2005; 19:367-372.
72. Perea G, Lasa A, Aventín A, et al. Prognostic value of minimal residual disease (MRD) in acute myeloid leukemia (AML) with favorable cytogenetics [t(8;21) and inv(16)]. Leukemia 2006; 20:87-94.
73. Weisser M, Haferlach C, Hiddemann W, Schnittger S. The quality of molecular response to chemotherapy is predictive for the outcome of AML1 -ETO-positive AML and is independent of pretreatment risk factors. Leukemia 2007 ; 21:1177-1182. A recent report on the impact of MRD level in t(8;21) AML patients.
74. Narimatsu H, Iino M, Ichihashi T, et al. Clinical significance of minimal residual disease in patients with t(8;21) acute myeloid leukemia in Japan, Int J Hematol 2008; 88:154-158. doi: 10.1007/s12185-008-0108-1. Another recent report on the impact of MRD level in t(8;21) AML patients.