How I treat atypical chronic myeloid leukemia

Blood

Volumn 129, Issue 7, 2017, Pages 838-845

  Gotlib, Jason ^a  

^a STANFORD CANCER INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AZACITIDINE; DECITABINE; LEDIPASVIR PLUS SOFOSBUVIR; RUXOLITINIB; ANTINEOPLASTIC AGENT; JANUS KINASE; PROTEIN KINASE INHIBITOR; PYRAZOLE DERIVATIVE;

ADULT; ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ARTICLE; ATYPICAL CHRONIC MYELOID LEUKEMIA; CANCER PROGNOSIS; CASE REPORT; CHROMOSOME ANALYSIS; DIAGNOSTIC APPROACH ROUTE; DISEASE COURSE; FEMALE; GENE MUTATION; HEPATITIS C; HUMAN; MALE; MIDDLE AGED; MULTIPLE CYCLE TREATMENT; PRIORITY JOURNAL; TREATMENT OUTCOME; TREATMENT RESPONSE; AGED; ANTAGONISTS AND INHIBITORS; GENETICS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; LEUKEMIA, MYELOID, CHRONIC, ATYPICAL, BCR-ABL NEGATIVE; PROCEDURES; PROGNOSIS;

AGED; ANTINEOPLASTIC AGENTS; CYTOGENETIC ANALYSIS; FEMALE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; JANUS KINASES; LEUKEMIA, MYELOID, CHRONIC, ATYPICAL, BCR-ABL NEGATIVE; MALE; MIDDLE AGED; PROGNOSIS; PROTEIN KINASE INHIBITORS; PYRAZOLES;

EID: 85014917374     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2016-08-693630     Document Type: Article

References (63)

1. Arber DA, Orazi A, Hasserjian R, et al. The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia. Blood. 2016;127(20):2391-2405.
2. Vardiman JW, Bennett JM, Bain BJ, Brunning RD, Thiele J. Atypical chronic myeloid leukaemia, BCR-ABL1 negative. In: Swerdlow S, Harris NL, Stein H, Jaffe ES, Theile J, Vardiman JW, eds. World Health Organization Classification of Tumours. Pathology and Genetics of Tumours of Haematopoietic and Lymphoid Tissues. Lyon, France: IARC Press; 2008:80-81.
3. Dao KH, Tyner JW. What's different about atypical CML and chronic neutrophilic leukemia? Hematology Am Soc Hematol Educ Program. 2015;2015:264-271.
4. Mughal TI, Cross NCP, Padron E, et al. An International MDS/MPN Working Group's perspective and recommendations on molecular pathogenesis, diagnosis and clinical characterization of myelodysplastic/myeloproliferative neoplasms. Haematologica. 2015;100(9):1117-1130.
5. Tiu RV, Sekeres MA. Making sense of the myelodysplastic/myeloproliferative neoplasms overlap syndromes. Curr Opin Hematol. 2014;21(2):131-140.
6. Gotlib J, Maxson JE, George TI, Tyner JW. The new genetics of chronic neutrophilic leukemia and atypical CML: implications for diagnosis and treatment. Blood. 2013;122(10):1707-1711.
7. Wang SA, Hasserjian RP, Fox PS, et al. Atypical chronic myeloid leukemia is clinically distinct from unclassifiable myelodysplastic/myeloproliferative neoplasms. Blood. 2014;123(17):2645-2651.
8. Breccia M, Biondo F, Latagliata R, Carmosino I, Mandelli F, Alimena G. Identification of risk factors in atypical chronic myeloid leukemia. Haematologica. 2006;91(11):1566-1568.
9. Kurzrock R, Bueso-Ramos CE, Kantarjian H, et al. BCR rearrangement-negative chronic myelogenous leukemia revisited. J Clin Oncol. 2001;19(11):2915-2926.
10. Martiat P, Michaux JL, Rodhain J. Philadelphia-negative (Ph-) chronic myeloid leukemia (CML): comparison with Ph1 CML and chronic myelomonocytic leukemia. The Groupe Français de Cytogénétique Hématologique. Blood. 1991;78(1):205-211.
11. Hernández JM, del Cañizo MC, Cuneo A, et al. Clinical, hematological and cytogenetic characteristics of atypical chronic myeloid leukemia. Ann Oncol. 2000;11(4):441-444.
12. Zoi K, Cross NCP. Molecular pathogenesis of atypical CML, CMML and MDS/MPN-unclassifiable. Int J Hematol. 2015;101(3):229-242.
13. Piazza R, Valletta S, Winkelmann N, et al. Recurrent SETBP1 mutations in atypical chronic myeloid leukemia. Nat Genet. 2013;45(1):18-24.
14. Meggendorfer M, Bacher U, Alpermann T, et al. SETBP1 mutations occur in 9% of MDS/MPN and in 4% of MPN cases and are strongly associated with atypical CML, monosomy 7, isochromosome i(17)(q10), ASXL1 and CBL mutations. Leukemia. 2013;27(9):1852-1860.
15. Meggendorfer M, Haferlach T, Alpermann T, et al. Specific molecular mutation patterns delineate chronic neutrophilic leukemia, atypical chronic myeloid leukemia, and chronic myelomonocytic leukemia. Haematologica. 2014;99(12):e244-e246.
16. Minakuchi M, Kakazu N, Gorrin-Rivas MJ, et al. Identification and characterization of SEB, a novel protein that binds to the acute undifferentiated leukemia-associated protein SET. Eur J Biochem. 2001;268(5):1340-1351.
17. Cristóbal I, Blanco FJ, Garcia-Orti L, et al. SETBP1 overexpression is a novel leukemogenic mechanism that predicts adverse outcome in elderly patients with acute myeloid leukemia. Blood. 2010;115(3):615-625.
18. Hoischen A, van Bon BW, Gilissen C, et al. De novo mutations of SETBP1 cause Schinzel-Giedion syndrome. Nat Genet. 2010;42(6):483-485.
19. Makishima H, Yoshida K, Nguyen N, et al. Somatic SETBP1 mutations in myeloid malignancies. Nat Genet. 2013;45(8):942-946.
20. Maxson JE, Gotlib J, Pollyea DA, et al. Oncogenic CSF3R mutations in chronic neutrophilic leukemia and atypical CML. N Engl J Med. 2013;368(19):1781-1790.
21. Pardanani A, Lasho TL, Laborde RR, et al. CSF3R T618I is a highly prevalent and specific mutation in chronic neutrophilic leukemia. Leukemia. 2013;27(9):1870-1873.
22. Maxson JE, Luty SB, MacManiman JD, et al. The colony-stimulating factor 3 receptor T640N mutation Is oncogenic, sensitive to JAK inhibition, and mimics T618I. Clin Cancer Res. 2016;22(3):757-764.
23. Bartels S, Lehmann U, Büsche G, et al. De novo CSF3R mutation associated with transformation of myeloproliferative neoplasm to atypical CML. Ann Hematol. 2015;94(7):1255-1256.
24. Gambacorti-Passerini CB, Donadoni C, Parmiani A, et al. Recurrent ETNK1 mutations in atypical chronic myeloid leukemia. Blood. 2015;125(3):499-503.
25. Lasho TL, Finke CM, Zblewski D, et al. Novel recurrent mutations in ethanolamine kinase 1 (ETNK1) gene in systemic mastocytosis with eosinophilia and chronic myelomonocytic leukemia. Blood Cancer J. 2015;5:e275.
26. Bousquet M, Quelen C, De Mas V, et al. The t(8;9)(p22;p24) translocation in atypical chronic myeloid leukaemia yields a new PCM1-JAK2 fusion gene. Oncogene. 2005;24(48):7248-7252.
27. Reiter A, Walz C, Watmore A, et al. The t(8;9) (p22;p24) is a recurrent abnormality in chronic and acute leukemia that fuses PCM1 to JAK2. Cancer Res. 2005;65(7):2662-2667.
28. Baxter EJ, Hochhaus A, Bolufer P, et al. The t(4;22)(q12;q11) in atypical chronic myeloid leukaemia fuses BCR to PDGFRA. Hum Mol Genet. 2002;11(12):1391-1397.
29. Safley AM, Sebastian S, Collins TS, et al. Molecular and cytogenetic characterization of a novel translocation t(4;22) involving the breakpoint cluster region and platelet-derived growth factor receptor-alpha genes in a patient with atypical chronic myeloid leukemia. Genes Chromosomes Cancer. 2004;40(1):44-50.
30. Wittman B, Horan J, Baxter J, et al. A 2-year-old with atypical CML with a t(5;12)(q33;p13) treated successfully with imatinib mesylate. Leuk Res. 2004;28(suppl 1):65-69.
31. Grand FH, Iqbal S, Zhang L, Russell NH, Chase A, Cross NC. A constitutively active SPTBN1-FLT3 fusion in atypical chronic myeloid leukemia is sensitive to tyrosine kinase inhibitors and immunotherapy. Exp Hematol. 2007;35(11):1723-1727.
32. Fu Y, Schroeder T, Zabelina T, et al. Postallogeneic monitoring with molecular markers detected by pretransplant next-generation or Sanger sequencing predicts clinical relapse in patients with myelodysplastic/myeloproliferative neoplasms. Eur J Haematol. 2014;92(3):189-194.
33. Lim SN, Lee JH, Lee JH, et al. Allogeneic hematopoietic cell transplantation in adult patients with myelodysplastic/myeloproliferative neoplasms. Blood Res. 2013;48(3):178-184.
34. Koldehoff M, Beelen DW, Trenschel R, et al. Outcome of hematopoietic stem cell transplantation in patients with atypical chronic myeloid leukemia. Bone Marrow Transplant. 2004;34(12):1047-1050.
35. Koldehoff M, Steckel NK, Hegerfeldt Y, Ditschkowski M, Beelen DW, Elmaagacli AH. Clinical course and molecular features in 21 patients with atypical chronic myeloid leukemia. Int J Lab Hematol. 2012;34(1):e3-e5.
36. Mittal P, Saliba RM, Giralt SA, et al. Allogeneic transplantation: a therapeutic option for myelofibrosis, chronic myelomonocytic leukemia and Philadelphia-negative/BCR-ABL-negative chronic myelogenous leukemia. Bone Marrow Transplant. 2004;33(10):1005-1009.
37. Langabeer SE, McCarron SL, Haslam K, O'Donovan MT, Conneally E. The CSF3R T618I mutation as a disease-specific marker of atypical CML post allo-SCT. Bone Marrow Transplant. 2014;49(6):843-844.
38. Patnaik MM, Tefferi A. Chronic myelomonocytic leukemia: 2016 update on diagnosis, risk stratification, and management. Am J Hematol. 2016;91(6):631-642.
39. Adès L, Sekeres MA, Wolfromm A, et al. Predictive factors of response and survival among chronic myelomonocytic leukemia patients treated with azacitidine. Leuk Res. 2013;37(6):609-613.
40. Mao L, You L, Yang M, et al. The first case of decitabine successfully in treatment of atypical chronic myeloid leukemia with CEPBPA double mutation. Chemotherapy. 2013;2:114.
41. Tong X, Li J, Zhou Z, Zheng D, Liu J, Su C. Efficacy and side-effects of decitabine in treatment of atypical chronic myeloid leukemia. Leuk Lymphoma. 2015;56(6):1911-1913.
42. Hausmann H, Bhatt VR, Yuan J, Maness LJ, Ganti AK. Activity of single-agent decitabine in atypical chronic myeloid leukemia. J Oncol Pharm Pract. 2016;22(6):790-794.
43. Jiang H, Wu Z, Ren LI, Tao D, Tong H. Decitabine for the treatment of atypical chronic myeloid leukemia: a report of two cases. Oncol Lett. 2016; 11(1):689-692.
44. Fleischman AG, Maxson JE, Luty SB, et al. The CSF3R T618I mutation causes a lethal neutrophilic neoplasia in mice that is responsive to therapeutic JAK inhibition. Blood. 2013;122(22):3628-3631.
45. Shanavas M, Popat U, Michaelis LC, et al. Outcomes of allogeneic hematopoietic cell transplantation in patients with myelofibrosis with prior exposure to Janus kinase 1/2 inhibitors. Biol Blood Marrow Transplant. 2016;22(3):432-440.
46. Dao KH, Solti MB, Maxson JE, et al. Significant clinical response to JAK1/2 inhibition in a patient with CSF3R-T618I-positive atypical chronic myeloid leukemia. Leuk Res Rep. 2014;3(2):67-69.
47. Freedman JL, Desai AV, Bailey LC, et al. Atypical chronic myeloid leukemia in two pediatric patients. Pediatr Blood Cancer. 2016;63(1):156-159.
48. Ammatuna E, Eefting M, van Lom K, Kavelaars FG, Valk PJ, Touw IP. Atypical chronic myeloid leukemia with concomitant CSF3R T618I and SETBP1 mutations unresponsive to the JAK inhibitor ruxolitinib [published correction appears in Ann Hematol. 2015;94(6):1079]. Ann Hematol. 2015;94(5):879-880.
49. Lierman E, Selleslag D, Smits S, Billiet J, Vandenberghe P. Ruxolitinib inhibits transforming JAK2 fusion proteins in vitro and induces complete cytogenetic remission in t(8;9)(p22; p24)/PCM1-JAK2-positive chronic eosinophilic leukemia. Blood. 2012;120(7):1529-1531.
50. Schwaab J, Knut M, Haferlach C, et al. Limited duration of complete remission on ruxolitinib in myeloid neoplasms with PCM1-JAK2 and BCR-JAK2 fusion genes. Ann Hematol. 2015;94(2):233-238.
51. Rumi E, Milosevic JD, Selleslag D, et al. Efficacy of ruxolitinib in myeloid neoplasms with PCM1-JAK2 fusion gene. Ann Hematol. 2015;94(11):1927-1928.
52. Kurzrock R, Kantarjian HM, Shtalrid M, Gutterman JU, Talpaz M. Philadelphia chromosome-negative chronic myelogenous leukemia without breakpoint cluster region rearrangement: a chronic myeloid leukemia with a distinct clinical course. Blood. 1990;75(2):445-452.
53. Montefusco E, Alimena G, Lo Coco F, et al. Ph-negative and bcr-negative atypical chronic myelogenous leukemia: biological features and clinical outcome. Ann Hematol. 1992;65(1):17-21.
54. Costello R, Lafage M, Toiron Y, et al. Philadelphia chromosome-negative chronic myeloid leukaemia: a report of 14 new cases. Br J Haematol. 1995;90(2):346-352.
55. Jabbour E, Kantarjian H, Cortes J, et al. PEG-IFN-alpha-2b therapy in BCR-ABL-negative myeloproliferative disorders: final result of a phase 2 study. Cancer. 2007;110(9):2012-2018.
56. Park S, Grabar S, Kelaidi C, et al; GFM group (Groupe Francophone des Myélodysplasies). Predictive factors of response and survival in myelodysplastic syndrome treated with erythropoietin and G-CSF: the GFM experience. Blood. 2008;111(2):574-582.
57. Khanna V, Pierce ST, Dao KH, et al. Durable disease control with MEK inhibition in a patient with NRAS-mutated atypical chronic myeloid leukemia. Cureus. 2015;7(12):e414.
58. Borthakur G, Popplewell L, Boyiadzis M, et al. Activity of the oral mitogen-activated protein kinase kinase inhibitor trametinib in RAS-mutant relapsed or refractory myeloid malignancies. Cancer. 2016;122(12):1871-1879.
59. Burgess MR, Hwang E, Firestone AJ, et al. Preclinical efficacy of MEK inhibition in Nras-mutant AML. Blood. 2014;124(26):3947-3955.
60. Cristóbal I, Garcia-Orti L, Cirauqui C, Alonso MM, Calasanz MJ, Odero MD. PP2A impaired activity is a common event in acute myeloid leukemia and its activation by forskolin has a potent antileukemic effect. Leukemia. 2011;25(4):606-614.
61. Perrotti D, Neviani P. Protein phosphatase 2A: a target for anticancer therapy. Lancet Oncol. 2013;14(6):e229-e238.
62. Lee SC, Dvinge H, Kim E, et al. Modulation of splicing catalysis for therapeutic targeting of leukemia with mutations in genes encoding spliceosomal proteins. Nat Med. 2016;22(6):672-678.
63. Savona MR, Malcovati L, Komrokji R, et al; MDS/MPN International Working Group. An international consortium proposal of uniform response criteria for myelodysplastic/myeloproliferative neoplasms (MDS/MPN) in adults. Blood. 2015;125(12):1857-1865.