Management recommendations for chronic myelomonocytic leukemia: Consensus statements from the SIE, SIES, GITMO groups

Haematologica

Volumn 98, Issue 9, 2013, Pages 1344-1352

  Onida, Francesco ^a   Barosi, Giovanni ^b   Leone, Giuseppe ^c   Malcovati, Luca ^d   Morra, Enrica ^e   Santini, Valeria ^f   Specchia, Giorgina ^g   Tura, Sante ^h  

^a UNIVERSITY OF MILAN   (Italy)

^b FONDAZIONE IRCCS POLICLINICO SAN MATTEO   (Italy)

^c CATHOLIC UNIVERSITY   (Italy)

^d UNIVERSITY OF PAVIA   (Italy)

^e OSPEDALE NIGUARDA CA GRANDA   (Italy)

^f UNIVERSITY OF FLORENCE   (Italy)

^g UNIVERSITY OF BARI   (Italy)

^h UNIVERSITY OF BOLOGNA   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

5 AZA 2' DEOXYCYTIDINE; AZACITIDINE; BENDAMUSTINE; CYTARABINE; ETOPOSIDE; HYDROXYUREA; LONAFARNIB; PROTEIN C JUN; PROTEIN C MYB; TIPIFARNIB; TOPOTECAN;

ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; CANCER PROGNOSIS; CELL PROLIFERATION; CHRONIC MYELOMONOCYTIC LEUKEMIA; CYTOGENETICS; DELPHI STUDY; DISEASE COURSE; FLOW CYTOMETRY; FOLLOW UP; GENE EXPRESSION; GENE MUTATION; HUMAN; IMMUNOPHENOTYPING; LEUKEMIA REMISSION; LIFE EXPECTANCY; OVERALL SURVIVAL; PATIENT MONITORING; PHASE 2 CLINICAL TRIAL (TOPIC); PROTEIN EXPRESSION; QUESTIONNAIRE; RANDOMIZED CONTROLLED TRIAL (TOPIC); REVIEW; RISK ASSESSMENT; SCORING SYSTEM; SPLENOMEGALY;

CONSENSUS; DISEASE MANAGEMENT; HUMANS; LEUKEMIA, MYELOMONOCYTIC, CHRONIC; PRACTICE GUIDELINES AS TOPIC; SOCIETIES, MEDICAL; WORLD HEALTH ORGANIZATION;

EID: 84883619210     PISSN: 03906078     EISSN: 15928721     Source Type: Journal    
DOI: 10.3324/haematol.2013.084020     Document Type: Review

References (71)

1. Swerdlow SH, Campo E, Harris NL, Jaffe ES, Pileri SA, Stein H, et al. (eds.): WHO Classification of Tumours of Haematopoietic and Lymphoid Tissues. IARC: Lyon, 2008
2. Parikh SA, Tefferi A. Chronic myelomonocytic leukemia: 2012 update on diagnosis, risk stratification, and management. Am J Hematol. 2012;87(6):610-9.
3. Beran M. Chronic myelomonocytic leukemia. Cancer Treat Res. 2008;142:107-32.
4. Williams PL, Webb C. The Delphi technique: a methodological discussion. J Adv Nurs. 1994;19(1):180-6.
5. Delbecq AL, van de Ven AH, Gustafson DH. Group Techniques for Program Planning: A guide to nominal group and Delphi processes. Scott, Foresman and Co. Glenview, IL, USA 1975.
6. Bacher U, Haferlach T, Kern W, Hiddemann W, Schnittger S, Schoch C. Conventional cytogenetics of myeloproliferative diseases other than CML contribute valid information. Ann Hematol. 2005;84 (4):250-7.
7. Haase D, Germing U, Schanz J, Pfeilstöcker M, Nösslinger T, Hildebrandt B, et al. New insights into the prognostic impact of the karyotype in MDS and correlation with subtypes: Evidence from a core dataset of 2124 patients. Blood. 2007;110(13):4385-95.
8. Grossmann V, Kohlmann A, Eder C, Haferlach C, Kern W, Cross NC, et al. Molecular profiling of chronic myelomonocytic leukemia reveals diverse mutations in >80% of patients with TET2 and EZH2 being of high prognostic relevance. Leukemia. 2011;25(5):877-9.
9. Kohlmann A, Grossmann V, Klein HU, Schindela S, Weiss T, Kazak B, et al. Nextgeneration sequencing technology reveals a characteristic pattern of molecular mutations in 72.8% of chronic myelomonocytic leukemia by detecting frequent alterations in TET2, CBL, RAS, and RUNX1. J Clin Oncol. 2010;28(24):3858-65.
10. Jankowska AM, Makishima H, Tiu RV, Szpurka H, Huang Y, Traina F, et al. Mutational spectrum analysis of chronic myelomonocytic leukemia includes genes associated with epigenetic regulation: UTX, EZH2, and DNMT3A. Blood. 2011; 118(14):3932-41.
11. Asou N. The role of a Runt domain transcription factor AML1/RUNX1 in leukemogenesis and its clinical implications. Crit Rev Oncol Hematol. 2003;45(12):129-50.
12. Kuo MC, Liang DC, Huang CF, Shih YS, Wu JH, Lin TL, et al. RUNX1 mutations are frequent in chronic myelomonocytic leukemia and mutations at the C-terminal region might predict acute myeloid leukemia transformation. Leukemia. 2009; 23(8):1426-31.
13. Ward PS, Patel J, Wise DR, Abdel-Wahab O, Bennett BD, Coller HA, et al. The common feature of leukemia-associated IDH1 and IDH2 mutations is a neomorphic enzyme activity converting alpha-ketoglutarate to 2-hydroxyglutarate. Cancer Cell. 2010;17(3):225-34.
14. Gelsi-Boyer V, Trouplin V, Adélaïde J, Aceto N, Remy V, Pinson S, et al. Genome profiling of chronic myelomonocytic leukemia: Frequent alterations of RAS and RUNX1 genes. BMC Cancer. 2008;8:299.
15. Ricci C, Fermo E, Corti S, Molteni M, Faricciotti A, Cortelezzi A, et al. RAS mutations contribute to evolution of chronic myelomonocytic leukemia to the proliferative variant. Clin Cancer Res. 2010;16(18): 2246-56.
16. Pich A, Riera L, Sismondi F, Godio L, Davico Bonino L, Marmont F, et al. JAK2V617F activating mutation is associated with the myeloproliferative type of chronic myelomonocytic leukaemia. J Clin Pathol. 2009;62(9):798-801.
17. Grand FH, Hidalgo-Curtis CE, Ernst T, Zoi K, Zoi C, McGuire C, et al. Frequent CBL mutations associated with 11q acquired uniparental disomy in myeloproliferative neoplasms. Blood. 2009;113(24):6182-92.
18. Gelsi-Boyer V, Trouplin V, Adélaïde J, Bonansea J, Cervera N, Carbuccia N, et al. Mutations of polycomb-associated gene ASXL1 in myelodysplastic syndromes and chronic myelomonocytic leukaemia. Br J Haematol. 2009;145(6):788-800.
19. Abdel-Wahab O, Pardanani A, Patel J, Wadleigh M, Lasho T, Heguy A, et al. Concomitant analysis of EZH2 and ASXL1 mutations in myelofibrosis, chronic myelomonocytic leukemia and blast-phase myeloproliferative neoplasms. Leukemia 2011;25(7):1200-2.
20. Tefferi A, Lim KH, Abdel-Wahab O, Lasho TL, Patel J, Patnaik MM, et al. Detection of mutant TET2 in myeloid malignancies other than myeloproliferative neoplasms: CMML, MDS, MDS/MPN and AML. Leukemia. 2009;23(7):1343-5.
21. Jankowska AM, Szpurka H, Tiu RV, Makishima H, Afable M, Huh J, et al. Loss of heterozygosity 4q24 and TET2 mutations associated with myelodysplastic/myeloproliferative neoplasms. Blood. 2009;113(25):6403-10.
22. Meggendorfer M, Roller A, Haferlach T, Eder C, Dicker F, Grossmann V, et al. SRSF2 mutations in 275 cases with chronic myelomonocytic leukemia (CMML). Blood. 2012;120(15):3080-8.
23. Rashidi HH, Xu X, Wang HY, Shafi NQ, Rameshkumar K, Messer K, et al. Utility of peripheral blood flow cytometry in differentiating low grade versus high grade myelodysplastic syndromes (MDS) and in the evaluation of cytopenias. Int J Clin Exp Pathol. 2012;5(3):224-30.
24. Bellos F, Alpermann T, Gouberman E, Haferlach C, Schnittger S, Haferlach T, et al. Evaluation of flow cytometric assessment of myeloid nuclear differentiation antigen expression as a diagnostic marker for myelodysplastic syndromes in a series of 269 patients. Cytometry B Clin Cytom. Cytometry B Clin Cytom. 2012;82 (15):295-304.
25. Rollins-Raval MA, Roth CG. The value of immunohistochemistry for CD14, CD123, CD33, myeloperoxidase and CD68R in the diagnosis of acute and chronic myelomonocytic leukaemias. Histopathology. 2012;60 (6):933-42.
26. Bennett JM, Catovsky D, Daniel MT, Flandrin G, Galton DA, Gralnick HR, et al. Proposals for the classification of the myelodysplastic syndromes. Br J Haematol. 1982;51(2):189-99.
27. Bennett JM, Catovsky D, Daniel MT, Flandrin G, Galton DA, Gralnick H, et al. The chronic myeloid leukaemias: guidelines for distinguishing chronic granulocytic, atypical chronic myeloid, and chronic myelomonocytic leukaemia. Proposals by the French-American-British Cooperative Leukaemia Group. Br J Haematol. 1994;87 (4):746-54.
28. Germing U, Gattermann N, Minning H, Heyll A, Aul C. Problems in the classification of LMMC--dysplastic versus proliferative type. Leuk Res. 1998;22(10):871-8.
29. Voglová J, Chrobák L, Neuwirtová R, Malasková V, Straka L. Myelodysplastic and myeloproliferative type of chronic myelomonocytic leukemia-distinct subgroups or two stages of the same disease? Leuk Res. 2001;25(6):493-9.
30. Nösslinger T, Reisner R, Grüner H, Tüchler H, Nowotny H, Pittermann E, et al. Dysplastic versus proliferative LMMC-a retrospective analysis of 91 patients from a single institution. Leuk Res. 2001;25 (9):741-7.
31. Onida F, Kantarjian H, Ball G, Estey E, Keating MJ, Glassman A, et al. The dysplastic versus proliferative classification dilemma of chronic myelomonocytic leukemia: a retrospective single institution analysis of 273 patients. Blood. 2001, 98;11(1 Suppl): 622A-623A.
32. Cervera J, Sanz GF, Vallespi T, del Cañizo MC, Irriguible D, Löpez F, et al. Does WBC count really define two different subtypes of chronic myelomonocytic leukemia (LMMC)? Analysis of a series of 119 patients. Leuk Res. 1997;21(4):S7.
33. González-Medina I, Bueno J, Torre-quebrada A, López A, Vallespí T, Massagué I, et al. Two groups of chronic myelomonocytic leukaemia: myelodysplastic and myeloproliferative. Prognostic implications in a series of a single center. Leuk Res. 2002;26(9):821-4.
34. Breccia M, Cannella L, Frustaci A, Stefanizzi C, D'Elia GM, Alimena G, et al. Chronic myelomonocytic leukemia with antecedent refractory anemia with excess blasts: further evidence for the arbitrary nature of current classification systems. Leuk Lymphoma. 2008;49(7):1292-6.
35. Jaffe E, Harris NL, Stein HS, Vardiman JW (eds.). Pathology and genetics of tumours of haematopoietic and lymphoid tissue. World Health Organization classification of tumours. Lyon: IARC Press, 2001, pp. 35-38.
36. Greenberg P, Cox C, LeBeau MM, Fenaux P, Morel P, Sanz G, et al. International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood. 1997;89(6):2079-88.
37. Onida F, Kantarjian HM, Smith TL, Ball G, Keating MJ, Estey EH, et al. Prognostic factors and scoring systems in chronic myelomonocytic leukemia: a retrospective analysis of 213 patients. Blood. 2002;99 (3):840-9.
38. Germing U, Strupp C, Alvado M, Gattermann N. New Prognostic parameters for myelomonocytic leukemia. Blood. 2002;100(2):731-3.
39. Beran M, Wen S, Shen Y, Onida F, Jelinek J, Cortes J, et al. Prognostic factors and risk assessment in chronic myelomonocytic leukemia: validation study of the M.D. Anderson Prognostic Scoring System. Leuk Lymphoma. 2007;48(6):1150-60.
40. Such E, Cervera J, Costa D, Solé F, Vallespí T, Luño E, et al. Cytogenetic risk stratification in chronic myelomonocytic leukemia. Haematologica. 2011;96(3):375-83.
41. Onida F, Gatto S, Scappini B, Ricci C, Ball G, Cortes J, et al. Significance of Ras point mutations for prognosis and response to treatment in chronic myelomonocytic leukemia: analysis of 112 patients. Proc Am Soc Clin Oncol. 2002;21:263a(1048).
42. Kosmider O, Gelsi-Boyer V, Ciudad M, Racoeur C, Jooste V, Vey N, et al. TET2 gene mutation is a frequent and adverse event in chronic myelomonocytic leu-kemia. Haematologica. 2009;94(12): 1676-81.
43. Braun T, Itzykson R, Renneville A, de Renzis B, Dreyfus F, Laribi K, et al. Molecular predictors of response to decitabine in advanced chronic myelo-monocytic leukemia: a phase 2 trial. Blood. 2011;118(14):3824-31.
44. Wattel E, Guerci A, Hecquet B, Econo-mopoulos T, Copplestone A, Mahé B, et al. A randomized trial of hydroxyurea versus VP16 in adult chronic myelomonocytic leukemia. Groupe Francais des Myelo-dysplasies and European CMML Group. Blood. 1996;88(17):2480-7.
45. Cheson BD, Greenberg PL, Bennett JM, Lowenberg B, Wijermans PW, Nimer SD, et al. Clinical application and proposal for modification of the International Working Group (IWG) response criteria in myelodysplasia. Blood. 2006;108(2):419-25.
46. Tefferi A, Barosi G, Mesa RA, Cervantes F, Deeg HJ, Reilly JT, et al. International Working Group (IWG) consensus criteria for treatment response in myelofibrosis with myeloid metaplasia, for the IWG for Myelofibrosis Research and Treatment (IWG-MRT). Blood. 2006;108(5):1497-503.
47. Barosi G, Birgegard G, Finazzi G, Griesshammer M, Harrison C, Hasselbalch H, et al. A unified definition of clinical resistance and intolerance to hydroxycarbamide in polycythaemia vera and primary myelofibrosis: results of a European LeukemiaNet (ELN) consensus process. Br J Haematol. 2010;148(6):961-3.
48. Silverman LR, Demakos EP, Peterson BL, Kornblith AB, Holland JC, Odchimar-Reissig R, et al. Randomized controlled trial of azacitidine inpatients with the myelodysplastic syndrome: a study of the cancer and leukemia group B. J Clin Oncol. 2002;20(10):2429-40.
49. Kantarjian H, Issa JP, Rosenfeld CS, Bennett JM, Albitar M, DiPersio J, et al. Decitabine improves patient outcomes in myelodysplastic syndromes: results of a phase III randomized study. Cancer. 2006;106(8):1794-803.
50. Costa R, Abdulhaq H, Haq B, Shadduck RK, Latsko J, Zenati M, et al. Activity of azacitidine in chronic myelomonocytic leukemia. Cancer. 2011;117(12):2690-6.
51. Fianchi L, Criscuolo M, Breccia M, Maurillo L, Salvi F, Musto P, et al. High rate of remissions in chronic myelomonocytic leukemia treated with 5-azacytidine: results of an Italian retrospective study. Leuk Lymphoma. 2013;54(3):658-61.
52. Tsai HC, Li H, Van Neste L, Cai Y, Robert C, Rassool FV, et al. Transient low doses of DNA-demethylating agents exert durable antitumor effects on hematological and epithelial tumor cells. Cancer Cell. 2012;21 (3):430-46.
53. Thorpe M, Montalvão A, Pierdomenico F, Moita F, Almeida A. Treatment of chronic myelomonocytic leukemia with 5-Azacitidine: A case series and literature review. Leuk Res. 2012;36 (8):1071-3.
54. Aribi A, Borthakur G, Ravandi F, Shan J, Davisson J, Cortes J, et al. Activity of decitabine, a hypomethylating agent, in chronic myelomonocytic leukemia. Cancer. 2007;109(4):713-7.
55. Wijermans PW, Rüter B, Baer MR, Slack JL, Saba HI, Lübbert M. Efficacy of decitabine in the treatment of patients with chronic myelomonocytic leukemia (CMML). Leuk Res. 2008;32(4):587-91.
56. Beran M, Estey E, O'Brien S, Cortes J, Koller CA, Giles FJ, et al. Topotecan and cytarabine is an active combination regimen in myelodysplastic syndromes and chronic myelomonocytic leukemia. J Clin Oncol. 1999;17(9):2819-30.
57. Beran M, Estey E, O'Brien SM, Giles FJ, Koller CA, Kornblau S, et al. Results of topotecan single-agent therapy in patients with myelodysplastic syndromes and chronic myelomonocytic leukemia. Leuk Lymphoma. 1998;31(5-6):521-31.
58. Grinblatt DL, Yu D, Hars V, Vardiman JW, Powell BL, Nattam S, et al. Treatment of myelodysplastic syndrome with 2 schedules and doses of oral topotecan: a randomized phase 2 trial by the Cancer and Leukemia Group B (CALGB 19803). Cancer. 2009;115(1):84-93.
59. Fenaux P, Raza A, Mufti GJ, Aul C, Germing U, Kantarjian H, et al. A multicenter phase 2 study of the farnesyltransferase inhibitor tipifarnib in intermediateto high-risk myelodysplastic syndrome. Blood. 2007;109(10):4158-63.
60. Feldman EJ, Cortes J, DeAngelo DJ, Holyoake T, Simonsson B, O'Brien SG, et al. On the use of lonafarnib in myelodysplastic syndrome and chronic myelomonocytic leukemia. Leukemia. 2008;22(9): 1707-11.
61. Strupp C, Knipp S, Hartmann J, Gattermann N, Haas R, Germing U. A pilot study of bendamustine in elderly patients with high-risk MDS and AML. Leuk Lymphoma. 2007;48(6):1161-6.
62. Cheng H, Kirtani VG, Gergis U. Current status of allogeneic HST for chronic myelomonocytic leukemia. Bone Marrow Transplant. 2012;47(4):535-41.
63. Elliott MA, Tefferi A, Hogan WJ, Letendre L, Gastineau DA, Ansell SM, et al. Allogeneic stem cell transplantation and donor lymphocyte infusions for chronic myelomonocytic leukemia. Bone Marrow Transplant. 2006;37(11):1003-8.
64. Kerbauy DM, Chyou F, Gooley T, Sorror ML, Scott B, Pagel JM, et al. Allogeneic hematopoietic cell transplantation for chronic myelomonocytic leukemia. Biol Blood Marrow Transplant. 2005;11(9):713-20.
65. Krishnamurthy P, Lim ZY, Nagi W, Kenyon M, Mijovic A, Ireland R, et al. Allogeneic haematopoietic SCT for chronic myelomonocytic leukaemia: a single-centre experience. Bone Marrow Transplant. 2010;45(10):1502-7.
66. Kroger N, Zabelina T, Guardiola P, Runde V, Sierra J, Van Biezen A, et al. Allogeneic stem cell transplantation of adult chronic myelomonocytic leukaemia. A report on behalf of the Chronic Leukaemia Working Party of the European Group for Blood and Marrow Transplantation (EBMT). Br J Haematol. 2002;118(1):67-73.
67. Laport GG, Sandmaier BM, Storer BE, Scott BL, Stuart MJ, Lange T, et al. Reducedintensity conditioning followed by allogeneic hematopoietic cell transplantation for adult patients with myelodysplastic syndrome and myeloproliferative disorders. Biol Blood Marrow Transplant. 2008;14(2):246-55.
68. Mittal P, Saliba RM, Giralt SA, Shahjahan M, Cohen AI, Karandish S, et al. Allogeneic transplantation: a therapeutic option for myelofibrosis, chronic myelomonocytic leukemia and Philadelphia-negative/BCRABL-negative chronic myelogenous leu-kemia. Bone Marrow Transplant. 2004; 33(10):1005-9.
69. Ocheni S, Kroger N, Zabelina T, Zander AR, Bacher U. Outcome of allo-SCT for chronic myelomonocytic leukemia. Bone Marrow Transplant. 2009;43(8):659-61.
70. Eissa H, Gooley TA, Sorror ML, Nguyen F, Scott BL, Doney K, et al. Allogeneic hematopoietic cell transplantation for chronic myelomonocytic leukemia: relapse-free survival is determined by karyotype and comorbidities. Biol Blood Marrow Transplant. 2010;17(6):908-15.
71. Such E, Germing U, Malcovati L, Cervera J, Kuendgen A, Della Porta MG, et al. Development and validation of a prognostic scoring system for patients with chronic myelomonocytic leukemia. Blood. 2013; 121(15):3005-15.