Abstract
Hematopoietic stem cell transplantation offers a curative therapy for patients with myelofibrosis. Due to toxicity, allografting following myeloablative regimens is mainly applicable to young patients. With the introduction of dose-reduced conditioning using busulfan or melphalan with fludarabine, transplantation became tolerable also in older patients. Improvement in management of transplant-related complications as well as increasing numbers of volunteer stem cell donors lead to an increased use of alternative donors. Through the discovery of new disease-specific mutations, close monitoring of residual disease became feasible in many patients, and the outcome of posttransplant strategies improved. Still challenging is achieving of new transplant-derived models to estimate risk status and possible outcome in every individual patient and help in therapy decision and determine optimal timing of stem cell transplantation. Such a tool may optimally include not only clinicomorphological characteristics but also other potentially relevant factors such as cytogenetics and novel molecular markers.
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References
Alchalby H, Badbaran A, Bock O et al (2010a) Screening and monitoring of MPL W515L mutation with real-time PCR in patients with myelofibrosis undergoing allogeneic stem cell transplantation. Bone Marrow Transplant 45:1404–1407
Alchalby H, Badbaran A, Zabelina T et al (2010b) Impact of JAK2V617F mutation status, allele burden, and clearance after allogeneic stem cell transplantation for myelofibrosis. Blood 116:3572–3581
Bacigalupo A, Dominetto A, Pozzi S et al (2007) Allogeneic hematopoietic stem transplant for patients with idiopathic myelofibrosis using a reduced intensity thiotepa based conditioning regimen. Blood 110: Abstract no. 684
Bacigalupo A, Soraru M, Dominietto A et al (2010) Allogeneic hematopoietic SCT for patients with primary myelofibrosis: a predictive transplant score based on transfusion requirement, spleen size and donor type. Bone Marrow Transplant 45:458–463
Ballen K, Zhang M, Arora M, the Chronic Leukemia Working Committee of the Center for International Blood Marrow Transplant Research (2005) Outcome of bone marrow transplantation for myelofibrosis. Blood 106: Abstract no. 170
Ballen KK, Shrestha S, Sobocinski KA et al (2010) Outcome of transplantation for myelofibrosis. Biol Blood Marrow Transplant 16(3):358–367. Epub 2009 Oct 30
Baurmann H, Burlakova I, Jedlickova Z et al (2009) Allogeneic haematopoietic cell transplantation for myelofibrosis – close post-transplant surveillance is mandatory [abstract no. V59]. Presented the Corporate Annual Conference ’09 of the German, Austrian and Swiss Haematology and Oncology Societies. Mannheim/Heidelberg, Germany; 2–6 Oct 2009
Benjamini O, Koren-Michowitz M, Amariglio N et al (2008) Relapse of postpolycythemia myelofibrosis after allogeneic stem cell transplantation in a polycythemic phase: successful treatment with donor lymphocyte infusion directed by quantitative PCR test for V617F-JAK2 mutation. Leukemia 22:1961–1963
Byrne JL, Beshti H, Clark D et al (2000) 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 108:430–433
Cervantes F, Barosi G, Demory JL et al (1998) Myelofibrosis with myeloid metaplasia in young individuals: disease characteristics, prognostic factors and identification of risk groups. Br J Haematol 102:684–690
Cervantes F, Rovira M, Urbano-Ispizua A (2000) Complete remission of idiopathic myelofibrosis following donor lymphocyte infusion after failure of allogeneic transplantation: demonstration of a graft-versus-myelofibrosis effect. Bone Marrow Transplant 26:697–699
Cervantes F, Passamonti F, Barosi G (2008) Life expectancy and prognostic factors in the classic BCR/ABL-negative myeloproliferative disorders. Leukemia 22:905–914
Cervantes F, Dupriez B, Pereira A et al (2009) New prognostic scoring system for primary myelofibrosis based on a study of the International Working Group for Myelofibrosis Research and Treatment. Blood 113:2895–2901
Ciurea SO, Sadegi B, Wilbur A et al (2008) Effects of extensive splenomegaly in patients with myelofibrosis undergoing a reduced intensity allogeneic stem cell transplantation. Br J Haematol 141:80–83
Creemers GJ, Lowenberg B, Hagenbeek A (1992) Allogeneic bone marrow transplantation for primary myelofibrosis. Br J Haematol 82:772–773
Deeg HJ, Gooley TA, Flowers ME et al (2003) Allogeneic hematopoietic stem cell transplantation for myelofibrosis. Blood 102:3912–3918
Devine SM, Hoffman R, Verma A et al (2002) Allogeneic blood cell transplantation following reduced-intensity conditioning is effective therapy for older patients with myelofibrosis with myeloid metaplasia. Blood 99:2255–2258
Dokal I, Jones L, Deenmamode M et al (1989) Allogeneic bone marrow transplantation for primary myelofibrosis. Br J Haematol 71:158–160
Dupriez B, Morel P, Demory JL et al (1996) Prognostic factors in agnogenic myeloid metaplasia: a report on 195 cases with a new scoring system. Blood 88:1013–1018
Elliott MA, Verstovsek S, Dingli D et al (2007) Monocytosis is an adverse prognostic factor for survival in younger patients with primary myelofibrosis. Leuk Res 31:1503–1509
George B, Kerridge I, Gottlieb D et al (2008) A reduced intensity conditioning protocol associated with excellent survival in patients with myelofibrosis. Bone Marrow Transplant 42:567–568
Guardiola P, Anderson JE, Bandini GW et al (1999) Allogeneic stem cell transplantation for agnogenic myeloid metaplasia: a European Group for Blood and Marrow Transplantation, Societe Francaise de Greffe de Moelle, Gruppo Italiano per il Trapianto del Midollo Osseo, and Fred Hutchinson Cancer Research Center Collaborative Study. Blood 93:2831–2838
Gupta V, Kröger N, Aschan J et al (2009) A retrospective comparison of conventional intensity conditioning and reduced-intensity conditioning for allogeneic hematopoietic cell transplantation in myelofibrosis. Bone Marrow Transplant 44:317–320
Hertenstein B, Guardiola P, Finke J et al (2002) Non-myeloablative (NMA) stem cell transplantation (SCT) for myeloid metaplasia with myelofibrosis (MMM): a survey from the Chronic Leukemia Working Party of the EBMT. Blood 100:Abstract no. 70
Hessling J, Kroger N, Werner M et al (2002) Dose-reduced conditioning regimen followed by allogeneic stem cell transplantation in patients with myelofibrosis with myeloid metaplasia. Br J Haematol 119:769–772
Hussein K, Huang J, Lasho T et al (2009) Karyotype complements the International Prognostic Scoring System for primary myelofibrosis. Eur J Haematol 82:255–259
James C, Ugo V, Le Couedic JP et al (2005) A unique clonal JAK2 mutation leading to constitutive signalling causes polycythaemia vera. Nature 434:1144–1148
Kerbauy DM, Gooley TA et al (2007) Hematopoietic cell transplantation as curative therapy for idiopathic myelofibrosis, advanced polycythemia vera, and essential thrombocythemia. Biol Blood Marrow Transplant 13:355–365
Klyuchnikov E, Holler E, Bornhäuser M et al (2011) Donor lymphocyte infusions and/or second allogeneic stem cell transplantation as salvage treatment for relapsed myelofibrosis after reduced-intensity allografting. (manuscript submitted)
Kröger N, Mesa RA (2008) Choosing between stem cell therapy and drugs in myelofibrosis. Leukemia 22:474–486
Kröger N, Zabelina T, Schieder H et al (2005) Pilot study of reduced-intensity conditioning followed by allogeneic stem cell transplantation from related and unrelated donors in patients with myelofibrosis. Br J Haematol 128:690–697
Kröger N, Badbaran A, Holler E et al (2007a) Monitoring of the JAK2-V617F mutation by highly sensitive quantitative real-time PCR after allogeneic stem cell transplantation in patients with myelofibrosis. Blood 109:1316–1321
Kröger N, Holler E, Kobbe G et al (2007b) 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). Blood 110:Abstract no. 683
Kröger N, Thiele J, Zander A et al (2007c) Rapid regression of bone marrow fibrosis after dose-reduced allogeneic stem cell transplantation in patients with primary myelofibrosis. Exp Hematol 35:1719–1722
Kröger N, Alchalby H, Klyuchnikov E et al (2009a) JAK2-V617F-triggered preemptive and salvage adoptive immunotherapy with donor-lymphocyte infusion in patients with myelofibrosis after allogeneic stem cell transplantation. Blood 113:1866–1868
Kröger N, Holler E, Kobbe G et al (2009b) Allogeneic stem cell transplantation after reduced-intensity conditioning in patients with myelofibrosis: a prospective, multicenter study of the Chronic Leukemia Working Party of the European Group for Blood and Marrow Transplantation (EBMT). Blood 114:5264–5270
Li Z, Deeg HJ (2001) Pros and cons of splenectomy in patients with myelofibrosis undergoing stem cell transplantation. Leukemia 15:465–467
Li Z, Gooley T, Applebaum FR (2001) Splenectomy and hemopoietic stem cell transplantation for myelofibrosis. Blood 97:2180–2181
Lissandre A, Bay J-O, Cahn J-Y et al (2011) Retrospective study of allogeneic haematopoietic stem-cell transplantation for myelofibrosis. Bone Marrow Transplant 46:557–561
Merup M, Lazarevic V, Nahi H, Andreasson B, Malm C, Nilsson L et al (2006) Different outcome of allogeneic transplantation in myelofibrosis using conventional or reduced-intensity conditioning regimens. Br J Haematol 135:367–373
Mesa RA, Verstovsek S, Cervantes F et al (2007) 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 31:737–740
Patriarca F, Bacigalupo A, Sperotto A et al (2008) Allogeneic hematopoietic stem cell transplantation in myelofibrosis: the 20-year experience of the Gruppo Italiano Trapianto di Midollo Osseo (GITMO). Haematologica 93:1514–1522
Percy MJ, McMullin MF (2005) The V617F JAK2 mutation and the myeloproliferative disorders. Hematol Oncol 23:91–93
Rajantie J, Sale GE, Deeg HJ et al (1986) Adverse effect of severe marrow fibrosis on hematologic recovery after chemoradiotherapy and allogeneic bone marrow transplantation. Blood 67:1693–1697
Robin M, Tabrizi R, Mohty M et al (2010) Allogeneic haematopoietic stem cell transplantation for myelofibrosis: a report of the Société Française de Greffe de Moelle et de Thérapie Cellulaire (SFGM-TC). Br J Haematol 152:331–339
Rondelli D, Barosi G, Bacigalupo A et al (2005) Allogeneic hematopoietic stem-cell transplantation with reduced-intensity conditioning in intermediate- or high-risk patients with myelofibrosis with myeloid metaplasia. Blood 105:4115–4119
Rumi E, Passamonti F, Arcaini L et al (2010) Molecular remission after allo-SCT in a patient with post-essential thrombocythemia myelofibrosis carrying the MPL (W515A) mutation. Bone Marrow Transplant 45:798–800
Sale GE, Deeg HJ, Porter BA (2006) Regression of myelofibrosis and osteosclerosis following hematopoietic cell transplantation assessed by magnetic resonance imaging and histologic grading. Biol Blood Marrow Transplant 12:1285–1294
Samuelson S, Sandmaier BM, Heslop HE et al (2011) Allogeneic haematopoietic cell transplantation for myelofibrosis in 30 patients 60–78 years of age. Br J Haematol 153:76–82
Smith RE, Chelmowski MK, Szabo EJ (1990) Myelofibrosis: a review of clinical and pathologic features and treatment. Crit Rev Oncol Hematol 10:305–314
Soll E, Massumoto C, Clift RA et al (1995) Relevance of marrow fibrosis in bone marrow transplantation: a retrospective analysis of engraftment. Blood 86:4667–4673
Steckel NK, Koldehoff M, Ditschkowski M (2007) 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 83:1518–1520
Stewart WA, Pearce R, Kirkland KE et al on behalf of the British Society for Blood and Marrow Transplantation (2010) The role of allogeneic SCT in primary myelofibrosis: a British Society for Blood and Marrow Transplantation study. Bone Marrow Transplant 45:1587–1593
Tam CS, Abruzzo LV, Lin KI et al (2009) The role of cytogenetic abnormalities as a prognostic marker in primary myelofibrosis: applicability at the time of diagnosis and later during disease course. Blood 113:4171–4178
Tefferi A (2005) Pathogenesis of myelofibrosis with myeloid metaplasia. J Clin Oncol 23:8520–8530
Tefferi A, Mesa RA, Nagorney DM (2000) Splenectomy in myelofibrosis with myeloid metaplasia: a single-institution experience with 223 patients. Blood 95:2226–2233
Tefferi A, Barosi G, Mesa RA et al (2006) 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 108:1497–1503
Thiele J, Kvasnicka HM, Dietrich H et al (2005) Dynamics of bone marrow changes in patients with chronic idiopathic myelofibrosis following allogeneic stem cell transplantation. Histol Histopathol 20:879–889
Vannucchi AM, Antonioli E, Guglielmelli P (2008) Clinical correlates of JAK2V617F presence or allele burden in myeloproliferative neoplasms: a critical reappraisal. Leukemia 22:1299–1307
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Kröger, N. (2012). Allogeneic Hematopoietic Stem Cell Transplantation for Myelofibrosis. In: Barbui, T., Tefferi, A. (eds) Myeloproliferative Neoplasms. Hematologic Malignancies. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-642-24989-1_16
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DOI: https://doi.org/10.1007/978-3-642-24989-1_16
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