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)

Hematopoietic cell transplantation ( HCT) is an effective treatment for myelodysplasia ( MDS) and secondary acute myeloid leukemia ( sAML). In this study, outcome of 593 patients with MDS/ sAML after autologous and allogeneic HCT from a matched unrelated donor ( MUD) were compared. A total of 167 ( 28%) patients received HCT from MUD without prior chemotherapy ( MUD- U). The rest received HCT in first complete remission ( CR1) ( Autologous ( Auto- CR1), n 290 ( 49%), HCT from MUD ( MUD- CR1), n 136 ( 23%)). Survival at 3 years was best in MUD- CR1 ( 50%) compared to Auto- CR1 ( 41%) and MUDU ( 40%) ( P 0.01). Similarly, disease- free survival was 44% for MUD- CR1 compared to Auto- CR1 ( 28%) and MUD- U ( 34%) ( P 0.03). Treatment- related mortality was 17% in Auto- CR1 compared to MUD- CR1 ( 38%) and MUD- U ( 49%) ( Po0.001). Relapse for Auto- CR1 was 62% compared to 24 and 30% for MUD- CR1 and MUD- U, respectively ( Po0.001). Outcome was best for patients with low tumor burden transplanted 6 - 12 months after diagnosis. Factors influencing outcome at 3 years were mainly significant in the first 6 months. Only, relapse after autologous HCT remained constant over time. Outcomes after allogeneic HCT in patients of 20 - 40 and440 years were similar. Autologous and Allogeneic HCT from MUD offer the possibility of long- term survival to patients with MDS/ sAML.