Allogeneic hematopoietic cell transplantation for myelodysplastic syndrome: The past decade

Expert Review of Clinical Immunology

Volumn 8, Issue 4, 2012, Pages 373-381

  Karoopongse, Ekapun ^a   Deeg, H Joachim ^a  

^a UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

Author keywords

conditioning regimen; cytogenetics; disease stage and transplant outcome; donor selection; hematopoietic cell transplantation; MDS; patient age

Indexed keywords

HLA ANTIGEN;

ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; CANCER CLASSIFICATION; CANCER PROGNOSIS; CANCER STAGING; CYTOGENETICS; DONOR; ENGRAFTMENT; FLOW CYTOMETRY; GENE EXPRESSION PROFILING; HUMAN; MYELODYSPLASTIC SYNDROME; QUALITY OF LIFE; REVIEW; RISK FACTOR; TREATMENT OUTCOME; UMBILICAL CORD BLOOD;

ADULT; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; MIDDLE AGED; MYELODYSPLASTIC SYNDROMES; TRANSPLANTATION CONDITIONING; TRANSPLANTATION, HOMOLOGOUS;

EID: 84861394168     PISSN: 1744666X     EISSN: 17448409     Source Type: Journal    
DOI: 10.1586/eci.12.18     Document Type: Review

References (84)

1. Anderson JE, Appelbaum FR, Schoch G et al. Allogeneic marrow transplantation for myelodysplastic syndrome with advanced disease morphology: A Phase II study of busulfan, cyclophosphamide, and total-body irradiation and analysis of prognostic factors. J. Clin. Oncol. 14, 220-226 (1996).
2. Prébet T, Gore SD, Esterni B et al. Outcome of high-risk myelodysplastic syndrome after azacitidine treatment failure. J. Clin. Oncol. 29, 3322-3327 (2011).
3. Aoudjhane M, Labopin M, Gorin NC et al. Comparative outcome of reduced intensity and myeloablative conditioning regimen in HLA identical sibling allogeneic haematopoietic stem cell transplantation for patients older than 50 years of age with acute myeloblastic leukaemia: A retrospective survey from the Acute Leukemia Working Party (ALWP) of the European group for Blood and Marrow Transplantation (EBMT). Leukemia 19, 2304-2312 (2005).
4. De Roos AJ, Deeg HJ, Scott D. A population-based study of survival in patients with secondary myelodysplastic syndromes (MDS): Impact of type and treatment of primary cancers. Cancer Causes Control 18, 1199-1208 (2007).
5. Bennett JM, Catovsky D, Daniel MT et al. Proposals for the classification of the myelodysplastic syndromes. Br. J. Haematol. 51, 189-199 (1982).
6. Greenberg P, Cox C, LeBeau MM et al. International scoring system for evaluating prognosis in myelodysplastic syndromes. Blood 89, 2079-2088 (1997) [Erratum: Blood 91(3), 1100 (1998)].
7. Vardiman JW, Thiele J, Arber DA et al. The 2008 revision of the World Health Organization (WHO) classification of myeloid neoplasms and acute leukemia: Rationale and important changes. Blood 114, 937-951 (2009).
8. Schanz J, Steidl C, Fonatsch C et al. Coalesced multicentric analysis of 2,351 patients with myelodysplastic syndromes indicates an underestimation of poor-risk cytogenetics of myelodysplastic syndromes in the international prognostic scoring system. J. Clin. Oncol. 29, 1963-1970 (2011).
9. Bejar R, LeVine R, Ebert BL. Unraveling the molecular pathophysiology of myelodysplastic syndromes (review). J. Clin. Oncol. 29, 504-515 (2011).
10. Bejar R, Stevenson K, Abdel-Wahab O et al. Clinical effect of point mutations in myelodysplastic syndromes. N. Engl. J. Med. 364, 2496-2506 (2011).
11. Scott BL, Storer BE, Greene JE, Hackman RC, Appelbaum FR, Deeg HJ. Marrow fibrosis as a risk factor for post-transplant outcome in patients with advanced MDS or AML with multilineage dysplasia. Biol. Blood Marrow Transplant. 13, 345-354 (2007).
12. 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 114, 5264-5270 (2009).
13. Scott BL, Wells DA, Loken MR, Myerson D, Leisenring WM, Deeg HJ. Validation of a flow cytometric scoring system as a prognostic indicator for posttransplantation outcome in patients with myelodysplastic syndrome. Blood 112, 2681-2686 (2008).
14. Mills KI, Kohlmann A, Williams PM et al. Microarray-based classifiers and prognosis models identify subgroups with distinct clinical outcomes and high risk of AML transformation of myelodysplastic syndrome. Blood 114, 1063-1072 (2009).
15. Battiwalla M, Hahn T, Radovic M et al. Human leukocyte antigen (HLA) DR15 is associated with reduced incidence of acute GVHD in HLA-matched allogeneic transplantation but does not impact chronic GVHD incidence. Blood 107, 1970-1973 (2006).
16. Stern M, Passweg J, Tiercy JM et al. Human leukocyte antigen DR15 is associated with reduced relapse rate and improved survival after human leukocyte antigen-identical sibling hematopoietic stem cell transplantation. Biol. Blood Marrow Transplant. 12, 1169-1175 (2006).
17. Newell LF, Gooley T, Hansen JA, Stirewalt DL, Petersdorf EW, Deeg HJ. Tumor necrosis factor polymorphism affects transplantation outcome in patients with myelodysplastic syndrome but not in those with chronic myelogenous leukemia, independent of the presence of HLA-DR15. Biol. Blood Marrow Transplant. 16, 1700-1706 (2010).
18. Greenberg PL. Myelodysplastic syndromes: dissecting the heterogeneity. J. Clin. Oncol. 29, 1937-1938 (2011).
19. Sorror M, Storer B, Sandmaier BM et al. Hematopoietic cell transplantation- comorbidity index and Karnofsky performance status are independent predictors of morbidity and mortality after allogeneic nonmyeloablative hematopoietic cell transplantation. Cancer 112, 1992-2001 (2008).
20. Boehm A, Sperr WR, Leitner G et al. Comorbidity predicts survival in myelodysplastic syndromes or secondary acute myeloid leukaemia after allogeneic stem cell transplantation. Eur. J. Clin. Invest. 38, 945-952 (2008).
21. Parimon T, Au DH, Martin PJ, Chien JW. A risk score for mortality after allogeneic hematopoietic cell transplantation. Ann. Intern. Med. 144, 407-414 (2006).
22. Guilfoyle R, Demers A, Bredeson C et al. Performance status, but not the hematopoietic cell transplantation comorbidity index (HCT-CI), predicts mortality at a Canadian transplant center. Bone Marrow Transplant. 43, 133-139 (2009).
23. Eissa H, Gooley TA, Sorror ML et al. Allogeneic hematopoietic cell transplantation for chronic myelomonocytic leukemia: Relapse-free survival is determined by karyotype and comorbidities. Biol. Blood Marrow Transplant. 17, 908-915 (2011).
24. Lee JH, Lee JH, Lim SN et al. Allogeneic hematopoietic cell transplantion for myelodysplastic syndrome: prognostic significance of pre-transplant IPSS score and comorbidity. Bone Marrow Transplant. 45, 450-457 (2010).
25. Armand P, Kim HT, Cutler CS et al. Prognostic impact of elevated pretransplantation serum ferritin in patients undergoing myeloablative stem cell transplantation. Blood 109, 4586-4588 (2007).
26. Mahindra A, Bolwell B, Sobecks R et al. Elevated pretransplant ferritin is associated with a lower incidence of chronic graft-versus-host disease and inferior survival after myeloablative allogeneic haematopoietic stem cell transplantation. Br. J. Haematol. 146, 310-316 (2009).
27. Platzbecker U, Bornhäuser M, Germing U et al. Red blood cell transfusion dependence and outcome after allogeneic peripheral blood stem cell transplantation in patients with De novo myelodysplastic syndrome (MDS). Biol. Blood Marrow Transplant. 14, 1217-1225 (2008).
28. Maradei SC, Maiolino A, De Azevedo AM, Colares M, Bouzas LF, Nucci M. Serum ferritin as risk factor for sinusoidal obstruction syndrome of the liver in patients undergoing hematopoietic stem cell transplantation. Blood 114, 1270-1275 (2009).
29. Kontoyiannis DP, Chamilos G, Lewis RE et al. Increased bone marrow iron stores is an independent risk factor for invasive aspergillosis in patients with high-risk hematologic malignancies and recipients of allogeneic hematopoietic stem cell transplantation. Cancer 110, 1303-1306 (2007).
30. Pullarkat V. Objectives of iron chelation therapy in myelodysplastic syndromes: More than meets the eye? (Review). Blood 114, 5251-5255 (2009).
31. Oliansky DM, Antin JH, Bennett JM et al. The role of cytotoxic therapy with hematopoietic stem cell transplantation in the therapy of myelodysplastic syndromes: An evidence-based review. Biol. Blood Marrow Transplant. 15, 137-172 (2009).
32. Weiden PL, Flournoy N, Thomas ED et al. Antileukemic effect of graft-versus-host disease in human recipients of allogeneic-marrow grafts. N. Engl. J. Med. 300, 1068-1073 (1979).
33. Weiden PL, Sullivan KM, Flournoy N, Storb R, Thomas ED; the Seattle Marrow Transplant Team. Antileukemic effect of chronic graft-versus-host disease. Contribution to improved survival after allogeneic marrow transplantation. N. Engl. J. Med. 304, 1529-1533 (1981).
34. Slavin S, Or R, Nagler A et al. Allogeneic cell therapy (AlloCT) for prevention of relapse in high risk acute leukemia following allogeneic bone marrow transplantation: control of GVHD by graded increments of donor lymphocyte infusion. Blood 88, 10(Suppl. 1), 418A (1996) (Abstract 1661).
35. Schmid C, Labopin M, Nagler A et al. Donor lymphocyte infusion in the treatment of first hematological relapse after allogeneic stem-cell transplantaion in adults with acute myeloid leukemia: A retrospective risk factors analysis and comparison with other strategies by the EBMT acute leukemia working party. J. Clin. Oncol. 25, 4938-4945 (2007).
36. Storb R, Raff RF, Appelbaum FR et al. What radiation dose for DLA-identical canine marrow grafts? Blood 72, 1300-1304 (1988).
37. Slattery JT, Clift RA, Buckner CD et al. Marrow transplantation for chronic myeloid leukemia: the influence of plasma busulfan levels on the outcome of transplantation. Blood 89, 3055-3060 (1997).
38. Tutschka PJ, Copelan EA, Kapoor N. Replacing total body irradiation with busulfan as conditioning of patients with leukemia for allogeneic marrow transplantation. Trans. Proc. 21, 2952-2954 (1989).
39. De Lima M, Couriel D, Shahjahan M et al. Allogeneic transplantation for acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) using a low-toxicity combination of intravenous (IV) busulfan (Bu) and fludarabine (Flu) ± ATG. Blood 100(Part 1; 11), 853A (2002) (Abstract 3366).
40. Russell JA, Duan Q, Chaudhry MA et al. Transplantation from matched siblings using once-daily intravenous busulfan/fludarabine with thymoglobulin: A myeloablative regimen with low nonrelapse mortality in all but older patients with high-risk disease. Biol. Blood Marrow Transplant. 14, 888-895 (2008).
41. Remberger M, Storer B, Ringden O, Anasetti C. Association between pretransplant Thymogloblulin and reduced non-relapse mortality rate after marrow transplantation from unrelated donors. Bone Marrow Transplant. 29, 391-397 (2002).
42. Finke J, Bethge WA, Schmoor C et al. Standard graft-versus-host disease prophylaxis with or without anti-T-cell globulin in haematopoietic cell transplantation from matched unrelated donors: A randomised, open-label, multicentre Phase 3 trial. Lancet Oncol. 10, 855-864 (2009).
43. Bacigalupo A, Lamparelli T, Bruzzi P et al. Antithymocyte globulin for graft-versus-host disease prophylaxis in transplants from unrelated donors: 2 randomized studies from Gruppo Italiano Trapianti Midollo Osseo (GITMO). Blood 98, 2942-2947 (2001).
44. Deeg HJ, Storer BE, Boeckh M et al. Reduced incidence of acute and chronic graft-versus-host disease with the addition of thymoglobulin to a targeted busulfan/ cyclophosphamide regimen. Biol. Blood Marrow Transplant. 12, 573-584 (2006).
45. Cutler CS, Lee SJ, Greenberg P et al. A decision analysis of allogeneic bone marrow transplantation for the myelodysplastic syndromes: delayed transplantation for low-risk myelodysplasia is associated with improved outcome. Blood 104, 579-585 (2004).
46. Kantarjian H, O'Brien S, Ravandi F et al. Proposal for a new risk model in myelodysplastic syndrome that accounts for events not considered in the original International Prognostic Scoring System. Cancer 113, 1351-1361 (2008).
47. Malcovati L, Germing U, Kuendgen A et al. Time-dependent Prognostic Scoring System for predicting survival and leukemic evolution in myelodysplastic syndromes. J. Clin. Oncol. 25, 3503-3510 (2007).
48. Alessandrino EP, Della Porta MG, Bacigalupo A et al. WHO classification and WPSS predict posttransplantation outcome in patients with myelodysplastic syndrome: A study from the Gruppo Italiano Trapianto di Midollo Osseo (GITMO). Blood 112, 895-902 (2008).
49. Robin M, Sanz G, Ionescu I et al. Unrelated cord blood transplantation (UCBT) in adults with MDS or secondary acute myeloblastic leukemia (sAML): A survey on behalf of Eurocord and CLWP of EBMT. Blood 114, 493 (2009) (Abstract 1198).
50. del Canizo MC, Martinez C, Conde E et al. Peripheral blood is safer than bone marrow as a source of hematopoietic progenitors in patients with myelodysplastic syndromes who receive an allogeneic transplantation. Results from the Spanish registry. Bone Marrow Transplant. 32, 987-992 (2003).
51. Couban S, Simpson DR, Barnett MJ et al. A randomized multicenter comparison of bone marrow and peripheral blood in recipients of matched sibling allogeneic transplants for myeloid malignancies. Blood 100, 1525-1531 (2002).
52. Guardiola P, Runde V, Bacigalupo A et al. Retrospective comparison of bone marrow and granulocyte colony-stimulating factor-mobilized peripheral blood progenitor cells for allogeneic stem cell transplantation using HLA identical sibling donors in myelodysplastic syndromes. Blood 99, 4370-4378 (2002).
53. Anasetti C, Logan BR, Lee SJ et al. Increased incidence of chronic graft-versus-host disease (GVHD) and no survival advantage with filgrastim-mobilized peripheral blood stem cells (PBSC) compared to bone marrow (BM) transplants from unrelated donors: Results of Blood and Marrow Transplant Clinical Trials Network (BMT CTN) protocol 0201, a Phase III, prospective, randomized trial. Blood 118(21), 1 (2011).
54. Castro-Malaspina H, Harris RE, Gajewski J et al. Unrelated donor marrow transplantation for myelodysplastic syndromes: outcome analysis in 510 transplants facilitated by the National Marrow Donor Program. Blood 99, 1943-1951 (2002).
55. Deeg HJ, Storer B, Slattery JT et al. Conditioning with targeted busulfan and cyclophosphamide for hemopoietic stem cell transplantation from related and unrelated donors in patients with myelodysplastic syndrome. Blood 100, 1201-1207 (2002).
56. De Witte T, Brand R, van Biezen A et al. Allogeneic stem cell transplantation for patients with refractory anaemia with matched related and unrelated donors: delay of the transplant is associated with inferior survival. Br. J. Haematol. 146, 627-636 (2009).
57. Woodard P, Carpenter PA, Davies SM et al. Unrelated donor bone marrow transplantation for myelodysplastic syndrome in children. Biol. Blood Marrow Transplant. 17, 723-728 (2011).
58. Anderson JE, Appelbaum FR, Schoch G et al. Allogeneic marrow transplantation for refractory anemia: A comparison of two preparative regimens and analysis of prognostic factors. Blood 87, 51-58 (1996).
59. Sierra J, Pérez WS, Rozman C et al. Bone marrow transplantation from HLA-identical siblings as treatment for myelodysplasia. Blood 100, 1997-2004 (2002).
60. Armand P, Kim HT, DeAngelo DJ et al. Impact of cytogenetics on outcome of De novo and therapy-related AML and MDS after allogeneic transplantation. Biol. Blood Marrow Transplant. 13, 655-664 (2007).
61. Armand P, Deeg HJ, Kim HT et al. Multicenter validation study of a transplantation-specific cytogenetics grouping scheme for patients with myelodysplastic syndromes. Bone Marrow Transplant. 45, 877-885 (2010).
62. Nemecek ER, Guthrie KA, Sorror ML et al. Conditioning with treosulfan and fludarabine followed by allogeneic hematopoietic cell transplantation for high-risk hematologic malignancies. Biol. Blood Marrow Transplant. 17, 341-350 (2011).
63. Casper J, Wolff D, Knauf W et al. Allogeneic hematopoietic stem-cell transplantation in patients with hematologic malignancies after dose-escalated treosulfan/ fludarabine conditioning. J. Clin. Oncol. 28, 3344-3351 (2010).
64. Chang CK, Storer BE, Scott BL et al. Hematopoietic cell transplantation in patients with myelodysplastic syndrome or acute myeloid leukemia arising from myelodysplastic syndrome: similar outcomes in patients with De novo disease and disease following prior therapy or antecedent hematologic disorders. Blood 110, 1379-1387 (2007).
65. Litzow MR, Tarima S, Perez WS et al. Allogeneic transplantation for therapy-related myelodysplastic syndrome and acute myeloid leukemia. Blood 115, 1850-1857 (2010).
66. Sato A, Ooi J, Takahashi S et al. Unrelated cord blood transplantation after myeloablative conditioning in adults with advanced myelodysplastic syndromes. Bone Marrow Transplant. 46, 257-261 (2011).
67. Parikh SH, Mendizabal A, Martin PL et al. Unrelated donor umbilical cord blood transplantation in pediatric myelodysplastic syndrome: A single-center experience. Biol. Blood Marrow Transplant. 15, 948-955 (2009).
68. Majhail NS, Brunstein CG, Tomblyn M et al. Reduced-intensity allogeneic transplant in patients older than 55 years: unrelated umbilical cord blood is safe and effective for patients without a matched related donor. Biol. Blood Marrow Transplant. 14, 282-289 (2008).
69. Henslee-Downey PJ. Allogeneic transplantation across major HLA barriers (review). Bailliere's Best Pract. Clin. Haematol. 14, 741-754 (2001).
70. De Witte T, Hermans J, Vossen J et al. Haematopoietic stem cell transplantation for patients with myelo-dysplastic syndromes and secondary acute myeloid leukaemias: A report on behalf of the Chronic Leukaemia Working Party of the European Group for Blood and Marrow Transplantation (EBMT). Br. J. Haematol. 110, 620-630 (2000).
71. Kasamon YL, Luznik L, Leffell MS et al. Nonmyeloablative HLA-haploidentical bone marrow transplantation with high-dose posttransplantation cyclophosphamide: effect of HLA disparity on outcome. Biol. Blood Marrow Trans. 16, 482-489 (2010).
72. Ciurea SO, Saliba R, Rondon G et al. Reduced-intensity conditioning using fludarabine, melphalan and thiotepa for adult patients undergoing haploidentical SCT. Bone Marrow Transplant. 45, 429-436 (2010).
73. Chen Y, Liu K, Xu L et al. HLA-mismatched hematopoietic SCT without in vitro T-cell depletion for myelodysplastic syndrome. Bone Marrow Transplant. 45, 1333-1339 (2010).
74. Field T, Perkins J, Huang Y et al. 5-azacitidine for myelodysplasia before allogeneic hematopoietic cell transplantation. Bone Marrow Transplant. 45, 255-260 (2010).
75. De Padua Silva L, De Lima M, Kantarjian H et al. Feasibility of allo-SCT after hypomethylating therapy with decitabine for myelodysplastic syndrome. Bone Marrow Transplant. 43, 839-843 (2009).
76. Lubbert M, Bertz H, Ruter B et al. Non-intensive treatment with low-dose 5-aza-2'-deoxycytidine (DAC) prior to allogeneic blood SCT of older MDS/AML patients. Bone Marrow Transplant. 44, 585-588 (2009).
77. Jabbour E, Garcia-Manero G, Shan J et al. Outcome of patients (pts) with myelodysplastic syndrome (MDS) and chronic myelomonocytic leukemia (CMML) post decitabine failure. Blood 112, 585-586 (2008) (Abstract 1659).
78. Cheson BD, Bennett JM, Kantarjian H et al. Report of an International Working Group to standardize response criteria for myelodysplastic syndromes. Blood 96, 3671-3674 (2000).
79. Smith AG, Wald J. Acute ventilatory failure in Lambert-Eaton myasthenic syndrome and its response to 3,4-diaminopyridine (review). Neurology 46, 1143-1145 (1996).
80. Martino R, Iacobelli S, Brand R et al. Retrospective comparison of reduced-intensity conditioning and conventional high-dose conditioning for allogeneic hematopoietic stem cell transplantation using HLA-identical sibling donors in myelodysplastic syndromes. Blood 108, 836-846 (2006).
81. Choi J, Ritchey J, Prior JL et al. In vivo administration of hypomethylating agents mitigate graft-versus-host disease without sacrificing graft-versus-leukemia. Blood 116, 129-139 (2010).
82. De Lima M, Giralt S, Thall PF et al. Maintenance therapy with low-dose azacitidine after allogeneic hematopoietic stem cell transplantation for recurrent acute myelogenous leukemia or myelodysplastic syndrome: A dose and schedule finding study. Cancer 116, 5420-5431 (2010).
83. Platzbecker U, Wermke M, Radke J et al. Azacitidine for treatment of imminent relapse in MDS or AML patients after allogeneic HSCT: Results of the RELAZA trial. Leukemia 26(3), 381-389 (2012).
84. Koreth J. A decision analysis of reduced-intensity conditioning allogeneic hematopoietic stem cell transplantation for older patients with de-novo myelodysplastic syndrome (MDS): early transplantation offers survival benefit in higher-risk MDS. Presented at: American Society of Hematology 53rd Annual Meeting. San Diego, CA, USA, 10-13 December 2011.