Allogeneic hematopoietic cell transplantation for myelofibrosis in the era of JAK inhibitors

Blood

Volumn 120, Issue 7, 2012, Pages 1367-1379

  Gupta, Vikas ^a   Hari, Parameswaran ^b   Hoffman, Ronald ^c  

^a UNIVERSITY OF TORONTO   (Canada)

^b MEDICAL COLLEGE OF WISCONSIN   (United States)

^c MOUNT SINAI SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AB 0024; BUSULFAN; CYCLOPHOSPHAMIDE; FLUDARABINE; GIVINOSTAT; HYDROXYUREA; JANUS KINASE; JANUS KINASE INHIBITOR; LENALIDOMIDE; MELPHALAN; MONOCLONAL ANTIBODY; PANOBINOSTAT; PLACEBO; POMALIDOMIDE; RUXOLITINIB; SARIDEGIB; STAT PROTEIN; THALIDOMIDE; THIOTEPA; UNCLASSIFIED DRUG;

ACUTE GRANULOCYTIC LEUKEMIA; AGE; ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; CHROMOSOME 13Q; CHROMOSOME 20Q; CHROMOSOME TRANSLOCATION; CYTOGENETICS; GENE MUTATION; GENETIC RISK; GRAFT FAILURE; GRAFT VERSUS HOST REACTION; HUMAN; HYPERBILIRUBINEMIA; KARYOTYPE; LEUKOCYTOSIS; LIFE EXPECTANCY; LIVER TOXICITY; LONG TERM SURVIVAL; MORTALITY; MYELOABLATIVE CONDITIONING; MYELOFIBROSIS; PHASE 3 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; PROGNOSIS; QUALITY OF LIFE; RANDOMIZED CONTROLLED TRIAL (TOPIC); REDUCED INTENSITY CONDITIONING; REVIEW; RISK ASSESSMENT; RISK BENEFIT ANALYSIS; SIGNAL TRANSDUCTION; SPLENECTOMY; SPLENOMEGALY; THROMBOCYTOPENIA; WHOLE BODY RADIATION;

EID: 84865174824     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2012-05-399048     Document Type: Review

References (92)

1. Tefferi A. Myelofibrosis with myeloid metaplasia. N Engl J Med. 2000;342(17):1255-1265. (Pubitemid 30235956)
2. Mesa RA, Silverstein MN, Jacobsen SJ, et al. Population-based incidence and survival figures in essential thrombocythemia and agnogenic myeloid metaplasia: an Olmsted County Study, 1976-1995. Am J Hematol. 1999;61(1):10-15. (Pubitemid 29218171)
3. Tefferi A, Lasho TL, Jimma T, et al. One thousand patients with primary myelofibrosis: the Mayo Clinic experience. Mayo Clin Proc. 2012;87(1): 25-33.
4. 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(13):2895-2901.
5. 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(4):392-397.
6. Passamonti F, Cervantes F, Vannucchi 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(9):1703-1708.
7. Deeg HJ, Appelbaum FR. Indications for and current results with allogeneic hematopoietic cell transplantation in patients with myelofibrosis. Blood. 2011;117(26):7185.
8. Tefferi A. How I treat myelofibrosis. Blood. 2011; 117(13):3494-3504.
9. Daly A, Song K, Nevill T, et al. Stem cell transplantation for myelofibrosis: a report from two Canadian centers. Bone Marrow Transplant. 2003; 32(1):35-40. (Pubitemid 36850278)
10. Deeg HJ, Gooley TA, Flowers ME, et al. Allogeneic hematopoietic stem cell transplantation for myelofibrosis. Blood. 2003;102(12):3912-3918. (Pubitemid 37486970)
11. Guardiola P, Anderson JE, Bandini G, et al. 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. 1999;93(9):2831-2838. (Pubitemid 29200773)
12. Horan JT, Logan BR, Agovi-Johnson MA, et al. Reducing the risk for transplantation-related mortality after allogeneic hematopoietic cell transplantation: how much progress has been made? J Clin Oncol. 2011;29(7):805-813.
13. Samuelson S, Sandmaier BM, Heslop HE, et al. Allogeneic haematopoietic cell transplantation for myelofibrosis in 30 patients 60-78 years of age. Br J Haematol. 2011;153(1):76-82.
14. Verstovsek S, Mesa RA, Gotlib J, et al. A doubleblind, placebo-controlled trial of ruxolitinib for myelofibrosis. N Engl J Med. 2012;366(9):799-807.
15. 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(9):787-798.
16. Verstovsek S, Kantarjian HM, Estrov Z, et al. Comparison of outcomes of advanced myelofibrosis patients treated with ruxolitinib (INCB018424) to those of a historical control group: survival advantage of ruxolitinib therapy [abstract]. Blood (ASH Annual Meeting Abstracts). 2011;118:793.
17. Tefferi A, Litzow MR, Pardanani A. Long-term outcome of treatment with ruxolitinib in myelofibrosis. N Engl J Med. 2011;365(15):1455-1457.
18. Tefferi A. JAK inhibitors for myeloproliferative neoplasms: clarifying facts from myths. Blood. 2012;119(12):2721-2730.
19. 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(3):1013-1018. (Pubitemid 26333326)
20. Mascarenhas J, Navada S, Malone A, et al. Therapeutic options for patients with myelofibrosis in blast phase. Leuk Res. 2010;34(9):1246-1249.
21. 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(3):973-977. (Pubitemid 40170863)
22. Tam CS, Nussenzveig RM, Popat U, et al. The natural history and treatment outcome of blast phase BCR-ABL- myeloproliferative neoplasms. Blood. 2008;112(5):1628-1637.
23. Passamonti F, Cervantes F, Vannucchi AM, et al. Dynamic International Prognostic Scoring System (DIPSS) predicts progression to acute myeloid leukemia in primary myelofibrosis. Blood. 2010; 116(15):2857-2858.
24. Passamonti F, Rumi E, Elena C, et al. Incidence of leukaemia in patients with primary myelofibrosis and RBC-transfusion-dependence. Br J Haematol. 2010;150(6):719-721.
25. Tefferi A, Pardanani A, Gangat N, et al. Leukemia risk models in primary myelofibrosis: an International Working Group study. Leukemia. 2012; 26(6):1439-1441.
26. Dingli D, Schwager SM, Mesa RA, et al. Presence of unfavorable cytogenetic abnormalities is the strongest predictor of poor survival in secondary myelofibrosis. Cancer. 2006;106(9):1985-1989. (Pubitemid 43673214)
27. Passamonti F, Rumi E, Caramella M, et al. A dynamic prognostic model to predict survival in post-polycythemia vera myelofibrosis. Blood. 2008;111(7):3383-3387.
28. Demory JL, Dupriez B, Fenaux P, et al. Cytogenetic studies and their prognostic significance in agnogenic myeloid metaplasia: a report on 47 cases. Blood. 1988;72(3):855-859.
29. Reilly JT, Snowden JA, Spearing RL, et al. Cytogenetic abnormalities and their prognostic significance in idiopathic myelofibrosis: a study of 106 cases. Br J Haematol. 1997;98(1):96-102. (Pubitemid 27348801)
30. 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(21):5612-5615.
31. Hidaka T, Shide K, Shimoda H, et al. The impact of cytogenetic abnormalities on the prognosis of primary myelofibrosis: a prospective survey of 202 cases in Japan. Eur J Haematol. 2009;83(4): 328-333.
32. Caramazza D, Begna KH, Gangat N, et al. Refined cytogenetic-risk categorization for overall and leukemia-free survival in primary myelofibrosis: a single center study of 433 patients. Leukemia. 2011;25(1):82-88.
33. Tam CS, Abruzzo LV, Lin KI, et al. The role of cytogenetic abnormalities as a prognostic marker in primary myelofibrosis: applicability at the time of diagnosis and later during disease course. Blood. 2009;113(18):4171-4178.
34. Baxter EJ, Scott LM, Campbell PJ, et al. Acquired mutation of the tyrosine kinase JAK2 in human myeloproliferative disorders. Lancet. 2005; 365(9464):1054-1061. (Pubitemid 40386783)
35. 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(4):387-397. (Pubitemid 40544655)
36. Kralovics R, Passamonti F, Buser AS, et al. A gain-of-function mutation of JAK2 in myeloproliferative disorders. N Engl J Med. 2005;352(17): 1779-1790. (Pubitemid 40570926)
37. James C, Ugo V, Le Couedic JP, et al. A unique clonal JAK2 mutation leading to constitutive signalling causes polycythaemia vera. Nature. 2005; 434(7037):1144-1148. (Pubitemid 40663494)
38. Scott LM, Tong W, Levine RL, et al. JAK2 exon 12 mutations in polycythemia vera and idiopathic erythrocytosis. N Engl J Med. 2007;356(5):459-468. (Pubitemid 46193073)
39. Oh ST, Simonds EF, Jones C, et al. Novel mutations in the inhibitory adaptor protein LNK drive JAK-STAT signaling in patients with myeloproliferative neoplasms. Blood. 2010;116(6):988-992.
40. Pikman Y, Lee BH, Mercher T, et al. MPLW515L is a novel somatic activating mutation in myelofibrosis with myeloid metaplasia. PLoS Med. 2006; 3(7):e270.
41. Campbell PJ, Griesshammer M, Dohner K, et al. V617F mutation in JAK2 is associated with poorer survival in idiopathic myelofibrosis. Blood. 2006;107(5):2098-2100. (Pubitemid 43289395)
42. Guglielmelli P, Barosi G, Specchia G, et al. Identification of patients with poorer survival in primary myelofibrosis based on the burden of JAK2V617F mutated allele. Blood. 2009;114(8):1477-1483.
43. Tefferi A, Lasho TL, Huang J, et al. Low JAK2V617F allele burden in primary myelofibrosis, compared to either a higher allele burden or unmutated status, is associated with inferior overall and leukemia-free survival. Leukemia. 2008; 22(4):756-761. (Pubitemid 351559282)
44. 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(12):4030-4036. (Pubitemid 350248459)
45. Pardanani A, Guglielmelli P, Lasho TL, et al. Primary myelofibrosis with or without mutant MPL: comparison of survival and clinical features involving 603 patients. Leukemia. 2011;25(12): 1834-1839.
46. 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(2):447-452.
47. Zhang SJ, Rampal R, Manshouri T, et al. Genetic analysis of patients with leukemic transformation of myeloproliferative neoplasms reveals recurrent SRSF2 mutations which are associated with adverse outcome. Blood. 2012;119(19):4480-4485.
48. Martínez-Trillos A, Gaya A, Maffioli M, et al. Efficacy and tolerability of hydroxyurea in the treatment of the hyperproliferative manifestations of myelofibrosis: results in 40 patients. Ann Hematol. 2010;89(12):1233-1237.
49. Mesa RA, Nagorney DS, Schwager S, et al. Palliative goals, patient selection, and perioperative platelet management: outcomes and lessons from 3 decades of splenectomy for myelofibrosis with myeloid metaplasia at the Mayo Clinic. Cancer. 2006;107(2):361-370. (Pubitemid 44036566)
50. Pardanani A, Gotlib J, Gupta V, et al. An expanded multicenter phase I/II study of CYT387, a JAK- 1/2 inhibitor for the treatment of myelofibrosis [abstract]. Blood (ASH Annual Meeting Abstracts). 2011;118:3849.
51. Pardanani A, Gotlib JR, Jamieson C, et al. Safety and efficacy of TG101348, a selective JAK2 inhibitor, in myelofibrosis. J Clin Oncol. 2011;29(7): 789-796.
52. Verstovsek S, Kantarjian H, Mesa RA, et al. Safety and efficacy of INCB018424, a JAK1 and JAK2 inhibitor, in myelofibrosis. N Engl J Med. 2010;363(12):1117-1127.
53. Tyner JW, Bumm TG, Deininger J, et al. CYT387, a novel JAK2 inhibitor, induces hematologic responses and normalizes inflammatory cytokines in murine myeloproliferative neoplasms. Blood. 2010;115(25):5232-5240.
54. Kröger N, Holler E, Kobbe G, et al. 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. Blood. 2009; 114(26):5264-5270.
55. Rondelli D, Goldberg JD, Marchioli R, et al. Results of phase II clinical trial MPD-RC 101: allogeneic hematopoietic stem cell transplantation conditioned with fludarabine/melphalan in patients with myelofibrosis [abstract]. Blood (ASH Annual Meeting Abstracts). 2011;118:1750.
56. Ballen KK, Shrestha S, Sobocinski KA, et al. Outcome of transplantation for myelofibrosis. Biol Blood Marrow Transplant. 2010;16(3):358-367.
57. Scott BL, Gooley TA, Sorror ML, et al. The Dynamic International Prognostic Scoring System for myelofibrosis predicts outcomes after hematopoietic cell transplantation. Blood. 2012;119(11): 2657-2664.
58. 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(3):355-365.
59. 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(6):697-699.
60. Devine SM, Hoffman R, Verma A, et al. Allogeneic blood cell transplantation following reducedintensity conditioning is effective therapy for older patients with myelofibrosis with myeloid metaplasia. Blood. 2002;99(6):2255-2258. (Pubitemid 34525517)
61. 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(2):430-433. (Pubitemid 30143663)
62. Bacigalupo A, Ballen K, Rizzo D, et al. Defining the intensity of conditioning regimens: working definitions. Biol Blood Marrow Transplant. 2009; 15(12):1628-1633.
63. Bacigalupo A, Soraru M, Dominietto A, et al. Allogeneic hemopoietic SCT for patients with primary myelofibrosis: a predictive transplant score based on transfusion requirement, spleen size and donor type. Bone Marrow Transplant. 2010;45(3): 458-463.
64. Gupta V, Kroger N, Aschan J, et al. A retrospective comparison of conventional intensity conditioning and reduced-intensity conditioning for allogeneic hematopoietic cell transplantation in myelofibrosis. Bone Marrow Transplant. 2009; 44(5):317-320.
65. Patriarca F, Bacigalupo A, Sperotto A, et al. Allogeneic hematopoietic stem cell transplantation in myelofibrosis: the 20-year experience of the Gruppo Italiano Trapianto di Midollo Osseo (GITMO). Haematologica. 2008;93(10):1514-1522.
66. Rondelli D, Barosi G, Bacigalupo A, et al. Allogeneic hematopoietic stem-cell transplantation with reduced-intensity conditioning in intermediate- or high-risk patients with myelofibrosis with myeloid metaplasia. Blood. 2005;105(10):4115-4119. (Pubitemid 40656163)
67. Abelsson J, Merup M, Birgegard G, et al. The outcome of allo-HSCT for 92 patients with myelofibrosis in the Nordic countries. Bone Marrow Transplant. 2012;47(3):380-386.
68. Alchalby H, Yunus DR, Zabelina T, et al. Risk models predicting survival after reduced-intensity transplantation for myelofibrosis [published online ahead of print January 27, 2012]. Br J Haematol. doi: 10.1111/j.1365-2141.2011. 09009.x.
69. Nivison-Smith I, Dodds AJ, Butler J, et al. Allogeneic hematopoietic cell transplantation for chronic myelofibrosis in Australia and New Zealand: older recipients receiving myeloablative conditioning at increased mortality risk. Biol Blood Marrow Transplant. 2012;18(2):302-308.
70. Ditschkowski M, Elmaagacli AH, Trenschel R, et al. DIPSS scores, pre-transplant therapy and chronic GVHD determine outcome after allogeneic hematopoietic stem cell transplantation for myelofibrosis [published online ahead of print April 4, 2012]. Haematologica. doi: 10.3324.haematol.2011.061168.
71. Alchalby H, Badbaran A, Zabelina T, et al. Impact of JAK2V617F mutation status, allele burden, and clearance after allogeneic stem cell transplantation for myelofibrosis. Blood. 2010;116(18):3572-3581.
72. 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(12):1350-1351. (Pubitemid 44858260)
73. Alchalby H, Lioznov M, Fritzsche-Friedland U, et al. Circulating CD34(+) cells as prognostic and follow-up marker in patients with myelofibrosis undergoing allo-SCT. Bone Marrow Transplant. 2012;47(1):143-145.
74. Takagi S, Ota Y, Uchida N, et al. Successful engraftment after reduced-intensity umbilical cord blood transplantation for myelofibrosis. Blood. 2010;116(4):649-652.
75. Li Z, Gooley T, Applebaum FR, et al. Splenectomy and hemopoietic stem cell transplantation for myelofibrosis. Blood. 2001;97(7):2180-2181. (Pubitemid 32239105)
76. Stewart WA, Pearce R, Kirkland KE, et al. The role of allogeneic SCT in primary myelofibrosis: a British Society for Blood and Marrow Transplantation study. Bone Marrow Transplant. 2010;45(11): 1587-1593.
77. Robin M, Esperou H, de Latour RP, et al. Splenectomy after allogeneic haematopoietic stem cell transplantation in patients with primary myelofibrosis. Br J Haematol. 2010;150(6):721-724.
78. Kröger N, Alchalby H, Klyuchnikov E, et al. JAK2- V617F-triggered preemptive and salvage adoptive immunotherapy with donor-lymphocyte infusion in patients with myelofibrosis after allogeneic stem cell transplantation. Blood. 2009;113(8): 1866-1868.
79. Wong KM, Atenafu EG, Kim D, et al. Incidence, risk factors for early hepatotoxicity and its impact on survival in patients with myelofibrosis undergoing allogeneic hematopoietic cell transplantation [published online ahead of print April 21, 2012]. Biol Blood Marrow Transplant. doi: 10.1016/jbbmt.2012.04.011.
80. Bryder D, Ramsfjell V, Dybedal I, et al. Self-renewal of multipotent long-term repopulating hematopoietic stem cells is negatively regulated by Fas and tumor necrosis factor receptor activation. J Exp Med. 2001;194(7):941-952. (Pubitemid 32953263)
81. Dybedal I, Bryder D, Fossum A, et al. Tumor necrosis factor (TNF)-mediated activation of the p55 TNF receptor negatively regulates maintenance of cycling reconstituting human hematopoietic stem cells. Blood. 2001;98(6):1782-1791.
82. Fleischman AG, Aichberger KJ, Luty SB, et al. TNFalpha facilitates clonal expansion of JAK2V617F positive cells in myeloproliferative neoplasms. Blood. 2011;118(24):6392-6398.
83. Tefferi A, Vaidya R, Caramazza D, et al. Circulating interleukin (IL)-8, IL-2R, IL-12, and IL-15 levels are independently prognostic in primary myelofibrosis: a comprehensive cytokine profiling study. J Clin Oncol. 2011;29(10):1356-1363.
84. Ferrara JL, Levine JE, Reddy P, et al. Graft-versus- host disease. Lancet. 2009;373(9674): 1550-1561.
85. Barbui T, Barosi G, Birgegard G, et al. Philadelphia-negative classical myeloproliferative neoplasms: critical concepts and management recommendations from European LeukemiaNet. J Clin Oncol. 2011;29(6):761-770.
86. Tefferi A, Pardanani A, Lim KH, et al. TET2 mutations and their clinical correlates in polycythemia vera, essential thrombocythemia and myelofibrosis. Leukemia. 2009;23(5):905-911.
87. Abdel-Wahab O, Pardanani A, Rampal R, et al. DNMT3A mutational analysis in primary myelofibrosis, chronic myelomonocytic leukemia and advanced phases of myeloproliferative neoplasms. Leukemia. 2011;25(7):1219-1220.
88. 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(3):475-480.
89. Guglielmelli P, Biamonte F, Score J, et al. EZH2 mutational status predicts poor survival in myelofibrosis. Blood. 2011;118(19):5227-5234.
90. Score J, Hidalgo-Curtis C, Jones AV, et al. Inactivation of polycomb repressive complex 2 components in myeloproliferative and myelodysplastic/ myeloproliferative neoplasms. Blood. 2012; 119(5):1208-1213.
91. Puda A, Milosevic JD, Berg T, et al. Frequent deletions of JARID2 in leukemic transformation of chronic myeloid malignancies. Am J Hematol. 2012;87(3):245-250.
92. Robin M, Tabrizi R, Mohty M, et al. Allogeneic haematopoietic stem cell transplantation for myelofibrosis: a report of the Societe Francaise de Greffe de Moelle et de Therapie Cellulaire (SFGM-TC). Br J Haematol. 2011;152(3):331-339.