Objective: To evaluate the clinical outcome of all-ogeneic hematopoietic stem cell transplantation (all-HSCT) for myelodysplastic syndrome (MDS).
Methods: From March 2001 to February 2009, 60 patients with MDS underwent allo-HSCT in our hospital were enrolled in this study. The conditioning regimens were Myleran (BU)/Cyclophosphamide (Cy) or Flu for identical sibling HSCT, and BU/Cy or Flu plus anti-thymocyte globulin (ATG) for haploidentical and unrelated HSCT. Cyclosporine A and short-course MTX were used for graft-versus-host disease (GVHD) prophylaxis. Diseased free survival (DFS) was calculated by Kaplan-Meier analysis.
Results: Total DFS rate was 75.3%. The relapse rate was 20%. DFS rates in RA/RAS/5q-, RCMD, RAEB-I/RAEB-II and acute myelocytic leukemia (AML) subgroups were 84.6%, 80.0%, 81.0%, 56.2%, respectively (P > 0.05). DFS rates in IPSS low risk group, intermediate-I risk group, intermediate-I risk group and high risk group were 80.0%, 84.6%, 81.8% and 65.4%, respectively (P > 0.05). DFS rates for allo-HSCT from identical sibling, unrelated or haploidentical donors were 79.2%, 60.0%, 76.9%, respectively (P = 0.028). DFS rates for percentages of blasts in bone marrow pre-transplant were 87.0%, 65.5%, 75.0% in < 5% blasts, 5% - 20% blasts, > 20% blasts subgroups, respectively (P > 0.05).
Conclusions: Since favorable clinical outcomes have been seen in all kinds of MDS by allo-HSCT, HSCT should be the first-line therapy for MDS. No significant differences are found based on different stem cell donors and the percentages of bone marrow blasts pre-HSCT, unrelated or haploidentical donors should be important alternatives if there is no identical sibling available. Chemotherapy before transplantation is not necessary except overt acute leukemia. A larger clinical study is needed to evaluate the clinical outcomes of allo-HSCT in MDS.