The objective of this study was to compare how cyclophosphamide (CY) and oral busulfan (BU) therapies, each in combination with total body irradiation (TBI), affect the outcome of stem cell transplantation in patients with advanced myelodysplastic syndrome. This study was conducted with 31 patients who received bone marrow from an HLA-matched sibling and underwent treatment with BU-TBI (n = 18) or CY-TBI (n = 13). The incidence of acute graft-versus-host disease (GVHD) grades II to IV was higher, but not significantly, in the BU-TBI group (58.8%) than in the CY-TBI group (30.8%). However, the incidences of chronic extensive GVHD were significantly different (41.7% [CY-TBI] versus 80.0% [BU-TBI],P =.04). Trends after BU-TBI conditioning indicated a lower 3-year probability of disease-free survival (DFS) (38.1% versus 53.9%,P =.4), lower relapse rates (28.5% versus 36.4%,P =.8), and higher nonrelapse mortality rates (46.7% versus 15.4%,P =.2). After adjusting for the International Prognostic Scoring System risk group and the cytogenetic risk group, DFS in the CY-TBI group (relative risk, 9.0; 95% confidence interval, 1.5–52.5;P =.015) was found to be significantly increased compared with the BU-TBI group. Approaches to minimize transplantation-related toxicity should be pursued, and efforts other than the potentiation of the conditioning regimen should be made to reduce the likelihood of posttransplantation relapse.
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Kouides PA, Bennett JM. Morphology and classification of the myelodysplastic syndromes and their pathologic variants.Semin Hematol. 1996;33:95–110.
Jacobs A. Genetic lesions in preleukaemia.Leukemia. 1991;5: 277–282.
Mufti GJ, Galton DA. Myelodysplastic syndromes: natural history and features of prognostic importance.Clin Haematol. 1986;15: 953–971.
Bennett JM, Catovsky D, Daniel MT, et al. Proposals for the classification of the myelodysplastic syndromes.Br J Haematol. 1982;51: 189–199.
Greenberg P, Cox C, LeBeau MM, et al. International scoring system for evaluating prognosis in myelodysplastic syndromes.Blood. 1997;89:2079–2088.
Pfeilstocker M, Reisner R, Nosslinger T, et al. Cross-validation of prognostic scores in myelodysplastic syndromes on 386 patients from a single institution confirms importance of cytogenetics.Br J Haematol. 1999;106:455–463.
Appelbaum FR, Anderson J. Allogeneic bone marrow transplantation for myelodysplastic syndrome: outcomes analysis according to IPSS score.Leukemia. 1998;12(suppl 1):S25-S29.
Deeg HJ, Shulman HM, Anderson JE, et al. Allogeneic and syngeneic marrow transplantation for myelodysplastic syndrome in patients 55 to 66 years of age.Blood. 2000;95:1188–1194.
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; 110:620–630.
Sutton L, Chastang C, Ribaud P, et al. Factors influencing outcome in de novo myelodysplastic syndromes treated by allogeneic bone marrow transplantation: a long-term study of 71 patients Societe Francaise de Greffe de Moelle.Blood. 1996;88:358–365.
Nevill TJ, Fung HC, Shepherd JD, et al. Cytogenetic abnormalities in primary myelodysplastic syndrome are highly predictive of outcome after allogeneic bone marrow transplantation.Blood. 1998; 92:1910–1917.
Copelan EA, Penza SL, Elder PJ, et al. Analysis of prognostic factors for allogeneic marrow transplantation following busulfan and cyclophosphamide in myelodysplastic syndrome and after leukemic transformation.Bone Marrow Transplant. 2000;25:1219–1222.
Anderson JE, Gooley TA, Schoch G, et al. Stem cell transplantation for secondary acute myeloid leukemia: evaluation of transplantation as initial therapy or following induction chemotherapy.Blood. 1997;89:2578–2585.
De Witte T, Zwaan F, Hermans J, et al. Allogeneic bone marrow transplantation for secondary leukemia and myelodysplastic syndrome: a survey by the Leukaemia Working Party of the European Bone Marrow Transplantation Group (EBMTG).Br J Haematol. 1990;74:151–155.
Niemeyer C, Duffner U, Bender-Gotze C, et al. AML-type intensive chemotherapy prior to stem cell transplantation (SCT) does not improve survival in children and adolescents with primary myelodysplastic syndromes (MDS) [abstract].Blood. 2000; 96(suppl 1):521a.
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. 1996;14:220–226.
Anderson JE, Anasetti C, Appelbaum FR, et al. Unrelated donor marrow transplantation for myelodysplasia (MDS) and MDS-related acute myeloid leukaemia.Br J Haematol. 1996;93:59–67.
Jurado M, Deeg HJ, Storer B, et al. Hematopoietic stem cell transplantation for advanced myelodysplastic syndrome after conditioning with busulfan and fractionated total body irradiation is associated with low relapse rate but considerable nonrelapse mortality.Biol Blood Marrow Transplant. 2002;8:161–169.
Glucksberg H, Storb R, Fefer A, et al. Clinical manifestations of graft-versus-host disease in human recipients of marrow from HL-A-matched sibling donors.Transplantation. 1974;18:295–304.
Shulman HM, Sullivan KM, Weiden PL, et al. Chronic graft-versus-host syndrome in man: a long-term clinicopathologic study of 20 Seattle patients.Am J Med. 1980;69:204–217.
Karanes C, Abella E, Du W, et al. Allogeneic bone marrow transplantation (alloBMT) in myelodysplastic syndromes (MDS) [abstract].Blood. 1998;92(suppl 1):659a.
Horowitz MM. Results of allogeneic stem cell transplantation for malignant disorders. In: Hoffman R, Benz EJ, Shattil SJ, et al, eds.Hematology: Basic Principles and Practice. New York, NY: Churchill Livingstone; 2000:1573–1587.
Storb R, Prentice RL, Sullivan KM, et al. Predictive factors in chronic graft-versus-host disease in patients with aplastic anemia treated by marrow transplantation from HLA-identical siblings.Ann Intern Med. 1983;98:461–466.
Ringden O, Paulin T, Lonnqvist B, Nilsson B. An analysis of factors predisposing for chronic graft-versus-host disease.Exp Hematol. 1985;13:1062–1067.
Deeg HJ, Spitzer TR, Cottler-Fox M, Cahill R, Pickle LW. Conditioning-related toxicity and acute graft-versus-host disease in patients given methotrexate/cyclosporine prophylaxis.Bone Marrow Transplant. 1991;7:193–198.
Carlens S, Ringden O, Remberger M, et al. Risk factors for chronic graft-versus-host disease after bone marrow transplantation: a retrospective single centre analysis.Bone Marrow Transplant. 1998;22: 755–761.
Anderson JE, Appelbaum FR, Fisher LD, et al. Allogeneic bone marrow transplantation for 93 patients with myelodysplastic syndrome.Blood. 1993;82:677–681.
Parker JE, Shafi T, Mijovic A, et al. Allogeneic stem cell transplantation (SCT) in MDS: interim results of outcome following non-myeloablative conditioning compared to standard preparative regimens [abstract].Blood. 2000;96(suppl 1):554a.
Kroger N, Schetelig J, Zabelina T, et al. A fludarabine-based dose-reduced conditioning regimen followed by allogeneic stem cell transplantation from related or unrelated donors in patients with myelodysplastic syndrome.Bone Marrow Transplant. 2001;28: 643–647.
Martino R, Caballero MD, Simon JA, et al. Evidence for a graft-versus-leukemia effect after allogeneic peripheral blood stem cell transplantation with reduced-intensity conditioning in acute myelogenous leukemia and myelodysplastic syndromes.Blood. 2002; 100:2243–2245.
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;100:1201–1207.
Andersson BS, Kashyap A, Gian V, et al. Conditioning therapy with intravenous busulfan and cyclophosphamide (IV BuCy2) for hematologic malignancies prior to allogeneic stem cell transplantation: a phase II study.Biol Blood Marrow Transplant. 2002;8: 145–154.
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Kim, Y., Kim, D., Lee, S. et al. Comparison of 2 Preparative Regimens for Stem Cell Transplantation from HLA-Matched Sibling Donors in Patients with Advanced Myelodysplastic Syndrome. Int J Hematol 82, 66–71 (2005). https://doi.org/10.1532/IJH97.A30501
- Myelodysplastic syndrome
- Stem cell transplantation
- Total body irradiation