Current status of allogeneic hematopoietic cell transplantation for MDS

Current Pharmaceutical Design

Volumn 18, Issue 22, 2012, Pages 3215-3221

  Xu, Feng ^a   Deeg, H Joachim ^a,b  

^a USA   (United States)

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

Author keywords

Allogeneic hematopoietic cell transplantation; Comorbid conditions; Conditioning regimens; Disease stage and prognosis; Donors and sources of stem cells; Myelodysplastic syndrome; Patient age; Relapse

Indexed keywords

5 AZA 2' DEOXYCYTIDINE; AZACITIDINE; BUSULFAN; CYCLOPHOSPHAMIDE; FLUDARABINE; MELPHALAN; THIOTEPA; THYMOCYTE ANTIBODY;

ADJUVANT THERAPY; ADOLESCENT; ADULT; ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ARTICLE; BONE MARROW TRANSPLANTATION; CHROMOSOME ABERRATION; COMORBIDITY; CYTOGENETICS; DONOR; GENOTYPE; GRAFT VERSUS HOST REACTION; HAPLOIDY; HLA SYSTEM; HUMAN; MAJOR CLINICAL STUDY; MULTIPLE CYCLE TREATMENT; MYELODYSPLASTIC SYNDROME; PRIORITY JOURNAL; RELAPSE; RISK FACTOR; SIBLING; SURVIVAL; UMBILICAL CORD BLOOD;

EID: 84862532694     PISSN: 13816128     EISSN: 18734286     Source Type: Journal    
DOI: 10.2174/1381612811209023215     Document Type: Article

References (75)

1. Corey SJ, Minden MD, Barber DL, Kantarjian H, Wang JC, Schimmer AD. Myelodysplastic syndromes: the complexity of stem-cell diseases (Review). Nature Reviews Cancer. 2007; 7: 118-29.
2. Greenberg PL. Myelodysplastic syndrome. In: Hoffman R, Benz EJ, Shattil SJ, Furie B, Cohen HJ, Silberstein LE, McGlave P, eds. Hematology: Basic Principles and Practice. New York: Churchill Livingstone; 2000;1106-29.
3. Fenaux P, Mufti GJ, Hellstrom-Lindberg E, et al. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. Lancet Oncol. 2009; 10: 223-32.
4. Bartenstein M, Deeg HJ. Hematopoietic stem cell transplantation for MDS. Hematol Oncol Clin North Am. 2010; 24: 407-22.
5. Ingram W, Lim ZY, Mufti GJ. Allogeneic transplantation for myelodysplastic syndrome (MDS) (Review). Blood Rev. 2007; 21: 61-71.
6. Deeg HJ, Sandmaier BM. Who is fit for allogeneic transplantation? Blood. 2010; 116: 4762-70.
7. 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. 2004; 104: 579-85.
8. 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. 2007; 25: 3503-10.
9. 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. 2008; 113: 1351-61.
10. Kuendgen A, Strupp C, Aivado M, et al. Myelodysplastic syndromes in patients younger than age 50. J Clin Oncol. 2006; 24: 5358-65.
11. Lim Z, Brand R, Martino R, et al. Allogeneic hematopoietic stemcell transplantation for patients 50 years or older with myelodysplastic syndromes or secondary acute myeloid leukemia. J Clin Oncol. 2010; 28: 405-11.
12. McClune BL, Weisdorf DJ, Pedersen TL, et al. Effect of age on outcome of reduced-intensity hematopoietic cell transplantation for older patients with acute myeloid leukemia in first complete remission or with myelodysplastic syndrome. J Clin Oncol. 2010; 28: 1878-87.
13. Itzykson R, Thepot S, Quesnel B, et al. Prognostic factors for response and overall survival in 282 patients with higher-risk myelodysplastic syndromes treated with azacitidine. Blood. 2011; 117: 403-11.
14. 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. 2009; 15: 137-72.
15. 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. 2008; 112: 895-902.
16. 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. 2008; 112: 2681-6.
17. Runde V, de Witte T, Arnold R, et al. Bone marrow transplantation from HLA-identical siblings as first-line treatment in patients with myelodysplastic syndromes: early transplantation is associated with improved outcome. Chronic Leukemia Working Party of the European Group for Blood and Marrow Transplantation. Bone Marrow Transplant. 1998; 21: 255-61.
18. Woodard P, Carpenter PA, Davies SM, et al. Unrelated donor bone marrow transplantation for myelodysplastic syndrome in children. Biol Blood Marrow Transplant. 2011; 17: 723-8.
19. 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. 2009; 146: 627-36.
20. 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. 2002; 100: 1201-7.
21. 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. 2007; 13: 655-64.
22. 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. 2010; 45: 877-85.
23. Onida F, Brand R, van Biezen A, et al. Impact of cytogenetics on outcome of patients with MDS or secondary AML undergoing allogeneic HSCT from HLA-identical siblings: a retrospective analysis of the EBMT-CLWP. Blood. 2006; 108: 750aq, #2653.
24. Upton A, McCune JS, Kirby KA, et al. Fluconazole coadministration concurrent with cyclophosphamide conditioning may reduce regimen-related toxicity postmyeloablative hematopoietic cell transplantation. Biol Blood Marrow Transplant. 2007; 13: 760-4.
25. Mittelman M, Oster HS, Hoffman M, Neumann D. The lower risk MDS patient at risk of rapid progression (Review). Leuk Res. 2010; 34: 1551-5.
26. 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. 2008; 112: 1992-2001.
27. 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. 2008; 38: 945-52.
28. Parimon T, Au DH, Martin PJ, Chien JW. A risk score for mortality after allogeneic hematopoietic cell transplantation. Ann Intern Med. 2006; 144: 407-14.
29. Eissa H, Gooley TA, Sorror ML, et al. Allogeneic hematopoietic cell transplantation for chronic myelomonocytic leukemia: relapsefree survival is determined by karyotype and comorbidities. Biol Blood Marrow Transplant. 2011; 17: 908-15.
30. Lee J-H, Lee J-H, Lim S-N, et al. Allogeneic hematopoietic cell transplantion for myelodysplastic syndrome: prognostic significance of pre-transplant IPSS score and comorbidity. Bone Marrow Transplant. 2010; 45: 450-7.
31. Armand P, Kim HT, Cutler CS, et al. Prognostic impact of elevated pretransplantation serum ferritin in patients undergoing myeloablative stem cell transplantation. Blood. 2007; 109: 4586-8.
32. Mahindra A, Bolwell B, Sobecks R, et al. Elevated pretransplant ferritin is associated with a lower incidence of chronic graft-versushost disease and inferior survival after myeloablative allogeneic haematopoietic stem cell transplantation. Br J Haematol. 2009; 146: 310-6.
33. 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. 2008; 14: 1217-25.
34. 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. 2009; 114: 1270-5.
35. 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. 2007; 110: 1303-6.
36. Majhail NS, DeFor T, Lazarus HM, Burns LJ. High prevalence of iron overload in adult allogeneic hematopoietic cell transplant survivors. Biol Blood Marrow Transplant. 2008; 14: 790-4.
37. Pullarkat V. Objectives of iron chelation therapy in myelodysplastic syndromes: more than meets the eye? (Review). Blood. 2009; 114: 5251-5.
38. 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. 2007; 13: 345-54.
39. 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: 5264-70.
40. 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. 2010; 16: 1700-6.
41. Al Ali HK, Brand R, van Biezen A, et al. A retrospective comparison of autologous and unrelated donor hematopoietic cell transplantation in myelodysplastic syndrome and secondary acute myeloid leukemia: a report on behalf of the Chronic Leukemia Working Party of the European Group for Blood and Marrow Transplantation (EBMT). Leukemia. 2007; 21: 1945-51.
42. de Lima M, Couriel D, Thall PF, et al. Once-daily intravenous buslfan and fludarabine: clinical and pharmacokinetic results of a myeloabltive, reduced-toxicity conditioning regimen for allogeneic stem cell transplantation in AML and MDS. Blood. 2004; 104: 857-64.
43. Nakai K, Kanda Y, Fukuhara S, et al. Value of chemotherapy before allogeneic hematopoietic stem cell transplantation from an HLA-identical sibling donor for myelodysplastic syndrome. Leukemia. 2005; 19: 396-401.
44. Scott BL, Storer B, Loken M, Storb R, Appelbaum FR, Deeg HJ. Pretransplantation induction chemotherapy and posttransplantation relapse in patients with advanced myelodysplastic syndrome. Biol Blood Marrow Transplant. 2005; 11: 65-73.
45. Warlick ED, Cioc A, DeFor T, Dolan M, Weisdorf D. Allogeneic stem cell transplantation for adults with myelodysplastic syndromes: importance of pretransplant disease burden. Biol Blood Marrow Transplant. 2009; 15: 30-8.
46. Oran B, Giralt S, Saliba R, et al. Allogeneic hematopoietic stem cell transplantation for the treatment of high-risk acute myelogenous leukemia and myelodysplastic syndrome using reducedintensity conditioning with fludarabine and melphalan. Biol Blood Marrow Transplant. 2007; 13: 454-62.
47. Castro-Malaspina H, Jabubowski AA, Papadopoulos EB, et al. Transplantation in remission improves the disease-free survival of patients with advanced myelodysplastic syndromes treated with myeloablative T cell-depleted stem cell transplants from HLAidentical siblings. Biol Blood Marrow Transplant. 2008; 14: 458-68.
48. Field T, Perkins J, Huang Y, et al. 5-azacitidine for myelodysplasia before allogeneic hematopoietic cell transplantation. Bone Marrow Transplant. 9999; prepublished online 22 June 2009; doi:10.1038/bmt.2009.134.
49. De Padua SL, de Lima M, Kantarjian H, et al. Feasibility of alloSCT after hypomethylating therapy with decitabine for myelodysplastic syndrome. Bone Marrow Transplant. 2009; 43: 839-43.
50. Kolb HJ, Schmid C, Tischer J, et al. Allogeneic stem cell transplantation for MDS and sAML following reduced intensity conditioning and preemptive donor lymphocyte transfusion. Blood. 2006; 108: 101a, #324.
51. Rezvani K, Yong AS, Mielke S, et al. Leukemia-associated antigen-specific T-cell responses following combined PR1 and WT1 peptide vaccination in patients with myeloid malignancies. Blood. 2008; 111: 236-42.
52. Warren EH. The human graft-versus-tumor response and how to exploit it. In: Appelbaum FR, Forman SJ, Negrin RS, Blume KG, eds. Thomas' Hematopoietic Cell Transplantation. Oxford, UK: Wiley-Blackwell; 2009;232-47.
53. 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. 2009; 114: 493, #1198.
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. 2002; 99: 1943-51.
55. Sato A, Ooi J, Takahashi S, et al. Unrelated cord blood transplantation after myeloablative conditioning in adults with advanced myelodysplastic syndromes. Bone Marrow Transplant. 2011; 46: 257-61.
56. 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. 2009; 15: 948-55.
57. 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. 2008; 14: 282-9.
58. 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. 2000; 110: 620-30.
59. 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. 2010; 45: 429-36.
60. Chen Y, Liu K, Xu L, et al. HLA-mismatched hematopoietic SCT without in vitro T-cell depletion for myelodysplastic syndrome. Bone Marrow Transplant. 2010; 45: 1333-9.
61. 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. 2003; 32: 987-92.
62. 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. 2002; 100: 1525-31.
63. Guardiola P, Runde V, Bacigalupo A, et al. Retrospective comparison of bone marrow and granulocyte colony-stimulating factormobilized peripheral blood progenitor cells for allogeneic stem cell transplantation using HLA identical sibling donors in myelodysplastic syndromes. Blood. 2002; 99: 4370-8.
64. 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. 2008; 14: 888-95.
65. Deeg HJ, Maris MB, Scott BL, Warren EH. Optimization of allogeneic transplant conditioning: not the time for dogma. Leukemia. 2006; 20: 1701-5.
66. Deeg HJ. Optimization of transplant regimens for patients with myelodysplastic syndromes (MDS). In: Berliner N, Lee SJ, Linenberger M, Vogelsang GB, eds. Hematology 2005: American Society of Hematology Education Program Book. Washington, DC: American Society of Hematology; 2005;167-73.
67. 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. 2006; 108: 836-46.
68. Parker JE, Shafi T, Pagliuca A, et al. Allogeneic stem cell transplantation in the myelodysplastic syndromes: interim results of outcome following reduced-intensity conditioning compared with standard preparative regimens. Br J Haematol. 2002; 119: 144-54.
69. Scott BL, Sandmaier BM, Storer B, et al. Myeloablative vs nonmyeloablative allogeneic transplantation for patients with myelodysplastic syndrome or acute myelogenous leukemia with multilineage dysplasia: a retrospective analysis. Leukemia. 2006; 20: 128-35.
70. Shimoni A, Hardan I, Shem-Tov N, Yerushalmi R, Nagler A. Allogeneic hematopoietic stem-cell transplantation in AML and MDS using myeloablative versus reduced-intensity conditioning: longterm follow-up. Leukemia. 2010; 24: 1050-2.
71. Khabori MA, El-Emary M, Xu W, et al. Impact of intensity of conditioning therapy in patients aged 40-60 years with AML/myelodysplastic sydrome undergoing allogeneic transplantation. Bone Marrow Transplant. 9999; prepublished online July 12, 2010; doi:10.1038/bmt.2010.164.
72. 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. 2011; 17: 341-50.
73. Kawase T, Matsuo K, Kashiwase K, et al. HLA mismatch combinations associated with decreased risk of relapse: implications for the molecular mechanism. Blood. 2009; 113: 2851-8.
74. Michalek J, Collins RH, Durrani HP, et al. Definitive separation of graft-versus-leukemiaand graft-versus-host-specific CD4+ T cells by virtue of their receptor beta loci sequences. PNAS. 2003; 100: 1180-4.
75. Greenberg P, Cox C, LeBeau MM, et al. International scoring system for evaluating prognosis in myelodysplastic syndromes [erratum appears in Blood 1998 Feb 1;91(3):1100]. Blood. 1997; 89: 2079-88.