Allogeneic hematopoietic cell transplantation for patients with myelofibrosis

Current Opinion in Hematology

Volumn 16, Issue 2, 2009, Pages 140-146

  Zang, Dae Young ^a   Deeg, H Joachim ^b,c  

^a Hallym University Medical Center   (South Korea)

^b Fred Hutchinson Cancer Research Center   (United States)

^c UNIVERSITY OF WASHINGTON   (United States)

Author keywords

Allogeneic hematopoietic cell transplantation; Myelofibrosis; Nonrelapse mortality; Relapse

Indexed keywords

BUSULFAN; CYCLOPHOSPHAMIDE; FLUDARABINE; JANUS KINASE 2; MELPHALAN;

ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; MEDICAL DECISION MAKING; MORTALITY; MYELOFIBROSIS; PRIORITY JOURNAL; PROGNOSIS; RELAPSE; REVIEW; SURVIVAL RATE; TREATMENT OUTCOME; TREATMENT RESPONSE;

HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; JANUS KINASE 2; MUTATION; PRIMARY MYELOFIBROSIS; PROGNOSIS; TRANSPLANTATION, HOMOLOGOUS; TUMOR MARKERS, BIOLOGICAL;

EID: 62249138491     PISSN: 10656251     EISSN: 15317048     Source Type: Journal    
DOI: 10.1097/MOH.0b013e3283257ab2     Document Type: Review

References (47)

1. Mesa RA, Verstovsek 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. This document summarized the proceedings of the second meeting of IWG-MRT, in which the group discussed and agreed to standardize the nomenclature referring to chronic idiopatbic myelofibrosis.
2. Tefferi A, Thiele J, Orazi A, et al. Proposals and rationale for revision of the World Health Organization diagnostic criteria for polycythemia vera, essential thrombocythemia, and primary myelofibrosis: recommendations from an ad hoc international expert panel. Blood 2007; 110:1092-1097.
3. Tefferi A, Vardiman JW. Classification and diagnosis of myeloproliferative neoplasms: the 2008 World Health Organization criteria and point-of-care diagnostic algorithms. Leukemia 2008; 22:14-22. An up-to-date overview of classification.
4. Cervantes F, Passamonti F, Barosi G. Life expectancy and prognostic factors in the classic BCR/ABL-negative myeloproliferative disorders. Leukemia 2008; 22:905-914.
5. Rajantie J, Sale GE, Deeg HJ, et al. Adverse effect of severe marrow fibrosis on hematological recovery after chemoradiotherapy and allogeneic bone marrow transplantation. Blood 1986; 67:1693-1697.
6. Soll E, Massumoto C, Clift RA, et al. Relevance of marrow fibrosis in bone marrow transplantation: a retrospective analysis of engraftment. Blood 1995; 86:4667-4673.
7. Dokal I, Jones L, Deenmamode M, et al. Allogeneic bone marrow transplantation for primary myelofibrosis. Br J Haematol 1989; 71:158-160.
8. Creemers GJ, Lowenberg B, Hagenbeek A. Allogeneic bone marrow transplantation for primary myelofibrosis. Br J Haematol 1992; 82:772-773.
9. Guardiola P, Anderson JE, Bandini G, et al. Allogeneic stem cell transplantation for agnogenic myeloid metaplasia: a European Group for Blood and Marrow Transplantation, Société Française de Greffe de Moelle, Gruppo Italiano per il Trapianto del Midollo Osseo, and Fred Hutchinson Cancer Research Center collaborative study. Blood 1999; 93:2831-2838.
10. Deeg HJ, Gooley TA, Flowers ME, et al. Allogeneic hematopoietic stem cell transplantation for myelofibrosis. Blood 2003; 102:3912-3918.
11. Kerbauy DM, Gooley TA, Sale GE, et al. Hematopoietic cell transplantation as curative therapy for idiopathic myelofibrosis, advanced polycythemia vera, and essential thrombocythemia. Biol Blood Marrow Transplant 2007; 13:355-365. This report showed an excellent long-term survival after allogeneic transplantation and improved survival for patients conditioned with targeted busulfan and cyclophosphamide.
12. Sorror ML, Maris MB, Storb R, et al. Hematopoietic cell transplantation (HCT)-specific comorbidity index: a new tool for risk assessment before allogeneic HCT. Blood 2005; 106:2912-2919.
13. Daly A, Song K, Nevill T, et al. Stem cell transplantation for myelofibrosis: a report from two Canadian centers. Bone Marrow Transplant 2003; 32:35-40.
14. Ditschkowski M, Beelen DW, Trenschel R, et al. Outcome of allogeneic stem cell transplantation in patients with myelofibrosis. Bone Marrow Transplant 2004; 34:807-813.
15. Devine SM, Hoffman R, Verma A, et al. Allogeneic blood cell transplantation following reduced-intensity conditioning is effective therapy for older patients with myelofibrosis with myeloid metaplasia. Blood 2002; 99:2255-2258.
16. Hessling J, Kroger N, Werner M, et al. Dose-reduced conditioning regimen followed by allogeneic stem cell transplantation in patients with myelofibrosis with myeloid metaplasia. Br J Haematol 2002; 119:769-772.
17. Rondelli D, Barosi G, Bacigalupo A, et al. Allogeneic hematopoietic stem-cell transplantation with reduced-intensity conditioning in intermediate-risk or high-risk patients with myelofibrosis with myeloid metaplasia. Blood 2005; 105:4115-4119.
18. Kroger N, Zabelina T, Schieder H, et al. Pilot study of reduced-intensity conditioning followed by allogeneic stem cell transplantation from related and unrelated donors in patients with myelofibrosis. Br J Haematol 2005; 128:690-697.
19. Merup M, Lazarevic V, Nahi H, et al. Different outcome of allogeneic transplantation in myelofibrosis using conventional or reduced-intensity conditioning regimens. Br J Haematol 2006; 135:367-373.
20. Snyder DS, Palmer J, Stein AS, et al. Allogeneic hematopoietic cell transplantation following reduced intensity conditioning for treatment of myelofibrosis. Biol Blood Marrow Transplant 2006; 12:1161-1168.
21. Byrne JL, Beshti H, Clark D, et al. Induction of remission after donor leucocyte infusion for the treatment of relapsed chronic idiopathic myelofibrosis following allogeneic transplantation: evidence for a 'graft vs. myelofibrosis' effect. Br J Haematol 2000; 108:430-433.
22. Cervantes F, Rovira M, Urbano-Ispizua A, et al. Complete remission of idiopathic myelofibrosis following donor lymphocyte infusion after failure of allogeneic transplantation: demonstration of a graft-versus-myelofibrosis effect. Bone Marrow Transplant 2000; 26:697-699.
23. 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.
24. Kroeger N, Holler E, Kobbe G, et al. Dose-reduced conditioning followed by allogeneic stem cell transplantation in patients with myelofibrosis. Results from a multicenter prospective trial of the Chronic Leukemia Working Party of the European Group for Blood and Marrow Transplantation (EBMT) [abstract #683]. Blood 2007; 110 (Part 1):210a. This abstract reports on an extensive analysis of 104 patients with PMF receiving dose-reduced conditioning allogeneic HCT.
25. Bacigalupo A, Dominietto A, Pozzi S, et al. Allogeneic hemopoietic stem transplant for patient with idiopathic myelofibrosis using a reduced intensity based conditioning regimen [abstract #684]. Blood 2007; 110 (Part 1):210a. A report of 39 patients with PMF showing that reduced-intensity thiotepa/cyclophosphamide regimens produced favorable outcome.
26. Li Z, Gooley T, Appelbaum FR, Deeg HJ. Splenectomy and hemopoietic stem cell transplantation for myelofibrosis. Blood 2001; 97:2180-2181.
27. Barosi G, Ambrosetti A, Centra A, et al. Splenectomy and risk of blast transformation in myelofibrosis with myeloid metaplasia. Blood 1998; 91:3630-3636.
28. Tefferi A, Mesa RA, Nagorney DM, et al. Splenectomy in myelofibrosis with myeloid metaplasia: a single-institution experience with 223 patients. Blood 2000; 95:2226-2233.
29. Li Z, Deeg HJ. Pros and cons of splenectomy in patients with myelofibrosis undergoing stem cell transplantation. Leukemia 2001; 15:465-467.
30. Baxter EJ, Scott LM, Campbell PJ, et al. Acquired mutation of the tyrosine kinase JAK2 in human myeloproliferative disorders. Lancet 2005; 365:1054-1061.
31. Levine RL, Wadleigh M, Cools J, et al. Activating mutation in the tyrosine kinase JAK2 in polycythemia vera, essential thrombocythemia, and myeloid metaplasia with myelofibrosis. Cancer Cell 2005; 7:387-397.
32. Barosi G, Bergamaschi G, Marchetti M, et al. JAK2 V617F mutational status predicts progression to large splenomegaly and leukemic transformation in primary myelofibrosis. Blood 2007; 110:4030-4036. The first paper reporting JAK2 mutation as the first biologic prognostic marker of PMF independent of conventional predictors.
33. Kralovics R, Passamonti F, Buser AS, et al. A gain-of-function mutation of JAK2 in myeloproliferative disorders. N Engl J Med 2005; 352:1779-1790.
34. Ditschkowski M, Elmaagacli AH, Trenschel R, et al. No influence of V617F mutation in JAK2 on outcome after allogeneic hematopoietic stem cell transplantation (HSCT) for myelofibrosis. Biol Blood Marrow Transplant 2006; 12:1350-1351.
35. Tefferi A, Barosi G, Mesa RA, 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; 108:1497-1503.
36. Kroger N, Badbaran A, Holler E, et al. Monitoring of the JAK2-V617F mutation by highly sensitive quantitative real-time PCR after allogeneic stem cell transplantation in patients with myelofibrosis. Blood 2007; 109:1316-1321. JAK2-V617F is useful to monitor minimal residual disease or relapse.
37. Druker BJ, Guilhot F, O'Brien SG, et al. Five-year follow-up of patients receiving imatinib for chronic myeloid leukemia. N Engl J Med 2006; 355:2408-2417.
38. Steckel NK, Koldehoff M, Ditschkowski M, et al. Use of the activating gene mutation of the tyrosine kinase (VAL617Phe) JAK2 as a minimal residual disease marker in patients with myelofibrosis and myeloid metaplasia after allogeneic stem cell transplantation. Transplantation 2007; 83:1518-1520.
39. Kroger N, Thiele J, Zander A, et al. Rapid regression of bone marrow fibrosis after dose-reduced allogeneic stem cell transplantation in patients with primary myelofibrosis. Exp Hematol 2007; 35:1719-1722. The reduced-intensity conditioning results in regression of fibrosis.
40. Sale GE, Deeg HJ, Porter BA. Regression of myelofibrosis and osteosclerosis following hematopoietic cell transplantation assessed by magnetic resonance imaging and histologic grading. Biol Blood Marrow Transplant 2006; 12:1285-1294.
41. Dingli D, Schwager SM, Mesa RA, et al. Prognosis in transplant-eligible patients with agnogenic myeloid metaplasia: a simple CBC-based scoring system. Cancer 2006; 106:623-630.
42. Cervantes F, Pereira A, Esteve J, et al. Identification of 'short-lived' and 'long-lived' patients at presentation of idiopathic myelofibrosis. Br J Haematol 1997; 97:635-640.
43. Elliott MA, Verstovsek S, Dingli D, et al. Monocytosis is an adverse prognostic factor for survival in younger patients with primary myelofibrosis. Leuk Res 2007; 31:1503-1509. This study added monocytes as a prognostic factor.
44. Tefferi A, Huang J, Schwager S, et al. Validation and comparison of contemporary prognostic models in primary myelofibrosis: analysis based on 334 patients from a single institution. Cancer 2007; 109: 2083-2088. An update on prognostic models.
45. Huang J, Li CY, Mesa RA, et al. Risk factors for leukemic transformation in patients with primary myelofibrosis. Cancer 2008; 112:2726-2732.
46. Vener C, Fracchiolla NS, Gianelli U, et al. Prognostic implications of the European consensus for grading of bone marrow fibrosis in chronic idiopathic myelofibrosis. Blood 2008; 111:1862-1865. This study showed marrow fibrosis as a major prognostic factor.
47. Cervantes F, Barosi G, Demory JL, et al. Myelofibrosis with myeloid metaplasia in young individuals: disease characteristics, prognostic factors and identification of risk groups. Br J Haematol 1998; 102:684-690.