Pathogenesis and management of acute myeloid leukemia that has evolved from a myeloproliferative neoplasm

Current Opinion in Hematology

Volumn 21, Issue 2, 2014, Pages 65-71

  Rampal, Raajit ^a   Mascarenhas, John ^b  

^a MEMORIAL SLOAN KETTERING CANCER CENTER   (United States)

^b MOUNT SINAI SCHOOL OF MEDICINE   (United States)

Author keywords

Acute myeloid leukemia; JAK2; Leukemic transformation; Myeloproliferative neoplasm; Ruxolitinib

Indexed keywords

CELL TRANSFORMATION, NEOPLASTIC; DISEASE PROGRESSION; HUMANS; LEUKEMIA, MYELOID, ACUTE; MYELOPROLIFERATIVE DISORDERS; NEOPLASM STAGING; RISK FACTORS;

EID: 84894045284     PISSN: 10656251     EISSN: 15317048     Source Type: Journal    
DOI: 10.1097/MOH.0000000000000017     Document Type: Review

References (52)

1. Mesa RA, Vestovsek S, Cervantes F, et al. Primary myelofibrosis (PMF), post polycythemia vera myelofibrosis (post-PV MF), post essential thrombocythemia myelofibrosis (post-ET MF), blast phase PMF (PMF-BP): consensus on terminology by the international working group for myelofibrosis research and treatment (IWG-MRT). Leuk Res 2007; 31:737-740.
2. Cervantes F, Tassies D, Salgado C, et al. Acute transformation in nonleukemic chronic myeloproliferative disorders: actuarial probability and main characteristics in a series of 218 patients. Acta Haematol 1991; 85:124-127.
3. Cervantes F, Dupriez B, Pereira A, et al. New prognostic scoring system for primary myelofibrosis based on a study of the International Working Group for Myelofibrosis Research and Treatment. Blood 2009; 113:2895-2901.
4. Cervantes F, Dupriez B, Passamonti F, et al. Improving survival trends in primary myelofibrosis: an international study. J Clin Oncol 2012; 30:2981-2987.
5. Mesa RA, Li CY, Ketterling RP, et al. Leukemic transformation in myelofibrosis with myeloid metaplasia: a single-institution experience with 91 cases. Blood 2005; 105:973-977.
6. Tam CS, Nussenzveig RM, Popat U, et al. The natural history and treatment outcome of blast phase BCR-ABL: myeloproliferative neoplasms. Blood 2008; 112:1628-1637.
7. Helbig G, Wieczorkiewicz-Kabut A, Pajak J, et al. Very poor outcome of leukemic transformation in myelofibrosis: a single center experience with 13 patients. Leuk Lymphoma 2012; 53:1236-1238.
8. Abdulkarim K, Girodon F, Johansson P, et al. AML transformation in 56 patients with Ph-MPD in two well defined populations. Eur J Haematol 2009; 82:106-111.
9. ennedy JA, Atenafu EG, Messner HA, et al. Treatment outcomes following leukemic transformation in Philadelphia-negative myeloproliferative neoplasms. Blood 2013; 121:2725-2733.
10. Passamonti F, Rumi E, Arcaini L, et al. Leukemic transformation of polycythemia vera: a single center study of 23 patients. Cancer 2005; 104:1032-1036.
11. Noor SJ, Tan W, Wilding GE, et al. Myeloid blastic transformation of myeloproliferative neoplasms - a review of 112 cases. Leuk Res 2011; 35:608-613.
12. Gangat N, Wolanskyj AP, McClure RF, et al. Risk stratification for survival and leukemic transformation in essential thrombocythemia: a single institutional study of 605 patients. Leukemia 2007; 21:270-276.
13. Finazzi G, Caruso V, Marchioli R, et al. Acute leukemia in polycythemia vera: an analysis of 1638 patients enrolled in a prospective observational study. Blood 2005; 105:2664-2670.
14. Dupriez B, Morel P, Demory JL, et al. Prognostic factors in agnogenic myeloid metaplasia: a report on 195 cases with a new scoring system. Blood 1996; 88:1013-1018.
15. Huang J, Li CY, Mesa RA, et al. Risk factors for leukemic transformation in patients with primary myelofibrosis. Cancer 2008; 112:2726-2732.
16. Morel P, Duhamel A, Hivert B, et al. Identification during the follow-up of timedependent prognostic factors for the competing risks of death and blast phase in primary myelofibrosis: a study of 172 patients. Blood 2010; 115:4350-4355.
17. Quintas-Cardama A, Kantarjian H, Pierce S, et al. Prognostic model to identify patients with myelofibrosis at the highest risk of transformation to acute myeloid leukemia. Clin Lymphoma Myeloma Leuk 2013; 13:315-318; e2.
18. Barosi G, Ambrosetti A, Centra A, et al. Splenectomy and risk of blast transformation in myelofibrosis with myeloid metaplasia. Italian Cooperative Study Group on Myeloid with Myeloid Metaplasia. Blood 1998; 91:3630-3636.
19. Vaidya R, Caramazza D, Begna KH, et al. Monosomal karyotype in primary myelofibrosis is detrimental to both overall and leukemia-free survival. Blood 2011; 117:5612-5615.
20. Barosi G, Ambrosetti A, Buratti A, et al. Splenectomy for patients with myelofibrosis with myeloid metaplasia: pretreatment variables and outcome prediction. Leukemia 1993; 7:200-206.
21. Lafaye F, Rain JD, Clot P, Najean Y. Risks and benefits of splenectomy in myelofibrosis: an analysis of 39 cases. Nouv Rev Fr Hematol 1994; 36:359-362.
22. Tefferi A, Mesa R, Nagorney DM, et al. Splenectomy in myelofibrosis with myeloid metaplasia: a single-institution experience with 223 patients. Blood 2000; 95:2226-2233.
23. Tam CS, Kantarjian H, Cortes J, et al. Dynamic model for predicting death within 12 months in patients with primary or postpolycythemia vera/ essential thrombocythemia myelofibrosis. J Clin Oncol 2009; 27:5587-5593.
24. Bjorkholm M, Derolf AR, Hultcrantz M, et al. Treatment-related risk factors for transformation to acute myeloid leukemia and myelodysplastic syndromes in myeloproliferative neoplasms. J Clin Oncol 2011; 29:2410-2415.
25. Fruchtman SM, Mack K, Kaplan ME, et al. From efficacy to safety: a Polycythemia Vera Study group report on hydroxyurea in patients with polycythemia vera. Semin Hematol 1997; 34:17-23.
26. Barbui T. The leukemia controversy in myeloproliferative disorders: Is it a natural progression of disease, a secondary sequela of therapy, or a combination of both? Semin Hematol 2004; 41 (2 Suppl 3):15-17.
27. Barbui T, Finazzi G. Special issues in myeloproliferative neoplasms. Curr Hematol Malig Rep 2011; 6:28-35.
28. Gangat N, Caramazza D, Vaidya R, et al. DIPSS plus: a refined Dynamic International Prognostic Scoring System for primary myelofibrosis that incorporates prognostic information from karyotype, platelet count, and transfusion status. J Clin Oncol 2011; 29:392-397.
29. Passamonti F, Cervantes F, Vanucchi AM, et al. A dynamic prognostic model to predict survival in primary myelofibrosis: a study by the IWG-MRT (International Working Group for Myeloproliferative Neoplasms Research and Treatment). Blood 2010; 115:1703-1708.
30. Tefferi A, Jimma T, Gangat N, et al. Predictors of greater than 80% 2-year mortality in primary myelofibrosis: a Mayo Clinic study of 884 karyotypically annotated patients. Blood 2011; 118:4595-4598.
31. Jager R, Gisslinger H, Passamonti F, et al. Deletions of the transcription factor Ikaros in myeloproliferative neoplasms. Leukemia 2010; 24:1290-1298.
32. Thoennissen NH, Krug UO, Lee DH, et al. Prevalence and prognostic impact of allelic imbalances associated with leukemic transformation of Philadelphia chromosome-negative myeloproliferative neoplasms. Blood 2010; 115:2882-2890.
33. Abdel-Wahab O, Manshouri T, Patel J, et al. Genetic analysis of transforming events that convert chronic myeloproliferative neoplasms to leukemias. Cancer Res 2010; 70:447-452.
34. Green A, Beer P. Somatic mutations of IDH1 and IDH2 in the leukemic transformation of myeloproliferative neoplasms. N Engl J Med 2010; 362:369-370.
35. Harutyunyan A, Klampfl T, Cazzola M, Kralovics R, et al. p53 lesions in leukemic transformation. N Engl J Med 2011; 364:488-490.
36. Zhang SJ, Rampal R, Manshouri T, et al. Genetic analysis of patients with leukemic transformation of myeloproliferative neoplasms shows recurrent SRSF2 mutations that are associated with adverse outcome. Blood 2012; 119:4480-4485.
37. Tefferi A, Jimma T, Sulai NH, et al. IDH mutations in primary myelofibrosis predict leukemic transformation and shortened survival: clinical evidence for leukemogenic collaboration with JAK2V617F. Leukemia 2012; 26:475-480.
38. Vannucchi AM, Lasho TL, Guglielmelli P, et al. Mutations and prognosis in primary myelofibrosis. Leukemia 2013; 27:1861-1869.
39. Ding Y, Harada Y, Imagawa J, et al. AML1/RUNX1 point mutation possibly promotes leukemic transformation in myeloproliferative neoplasms. Blood 2009; 114:5201-5205.
40. Beer PA, Delhommeau F, LeCouedic JP, et al. Two routes to leukemic transformation after a JAK2 mutation-positive myeloproliferative neoplasm. Blood 2010; 115:2891-2900.
41. Cancer Genome Atlas Research Network. Genomic and epigenomic landscapes of adult de novo acute myeloid leukemia. N Engl J Med 2013; 368:2059-2074.
42. Patel JP, Gonen M, Figueroa M, et al. Prognostic relevance of integrated genetic profiling in acute myeloid leukemia. N Engl J Med 2012; 366:1079-1089.
43. Campbell PJ, Baxter EJ, Beer PA, et al. Mutation of JAK2 in the myeloproliferative disorders: timing, clonality studies, cytogenetic associations, and role in leukemic transformation. Blood 2006; 108:3548-3555.
44. Theocharides A, Boissinot M, Girodon F, et al. Leukemic blasts in transformed JAK2-V617F-positive myeloproliferative disorders are frequently negative for the JAK2-V617F mutation. Blood 2007; 110:375-379.
45. Mascarenhas J, Navada S, Malone A, et al. Therapeutic options for patients with myelofibrosis in blast phase. Leuk Res 2010; 34:1246-1249.
46. Polliack A, Prokocimer M, Matzner Y. Lymphoblastic leukemic transformation (lymphoblastic crisis) in myelofibrosis and myeloid metaplasia. Am J Hematol 1980; 9:211-220.
47. Thepot S, Itzykson R, Seegers V, et al. Treatment of progression of Philadelphia-negative myeloproliferative neoplasms to myelodysplastic syndrome or acute myeloid leukemia by azacitidine: a report on 54 cases on the behalf of the Groupe Francophone des Myelodysplasies (GFM). Blood 2010; 116:3735-3742.
48. Cherington C, Slack JL, Leis J, et al. Allogeneic stem cell transplantation for myeloproliferative neoplasm in blast phase. Leuk Res 2012; 36:1147-1151.
49. Verstovsek S, Mesa RA, Gotlib J, et al. A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis. N Engl J Med 2012; 366:799-807.
50. Harrison C, Kiladjian JJ, Al-Ali HK, et al. JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis. N Engl J Med 2012; 366:787-798.
51. Eghtedar A, Verstovsek S, Estrov Z, et al. Phase 2 study of the JAK kinase inhibitor ruxolitinib in patients with refractory leukemias, including postmyeloproliferative neoplasm acute myeloid leukemia. Blood 2012; 119:4614-4618. A study of the use of ruxolitinib to treat AML, including patients with MPN-BP.
52. Mascarenhas J, Heaney ML, Najfeld V, et al. Proposed criteria for response assessment in patients treated in clinical trials for myeloproliferative neoplasms in blast phase (MPN-BP): formal recommendations from the postmyeloproliferative neoplasm acute myeloid leukemia consortium. Leuk Res 2012; 36:1500-1504.