Haematopoietic stem cell transplantation for patients with myelodysplasticsyndromes 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)

Allogeneic stem cell transplantation from an HLA-identical sibling donor is a curative treatment option for a young patient with myelodysplastic syndr ome, limited by age and lack of sibling donors. Alternative stem cell sourc es have been used more recently, such as unrelated donors, non-identical fa mily members or autologous transplants. This analysis of 1378 transplants r eported to the European Group for Blood and Marrow Transplantation (EBMT) a ddresses the outcome of the varying procedures according to the known risk factors. The estimated disease-free survival (DFS) and estimated relapse ri sk at 3 years were both 36% for 885 patients transplanted with stem cells f rom matched siblings. In the multivariate analysis, age and stage of diseas e had independent prognostic significance for DFS, survival and treatment-r elated mortality. Patients transplanted at an early stage of disease had a significantly lower risk of relapse than patients transplanted at more adva nced stages. The estimated DFS at 3 years was 25% for the 198 patients with voluntary unrelated donors, 28% for the 91 patients with alternative famil y donors and 33% for the 126 patients autografted in first complete remissi on. The non-relapse mortality was 58% for patients with unrelated donors, 6 6% for patients with non-identical family donors and 25% for autografted pa tients. The relapse rate of 18% was relatively low for patients with non-id entical family donors, 41% for patients with unrelated donors and 55% for p atients treated with autologous stem cell transplantation, Both allogeneic and autologous stem cell transplantation have emerged as treatment options for patients with myelodysplastic syndromes. Transplantation with an HLA-id entical sibling donor is the preferred treatment option. Patients without a n sibling donor may be treated with either autologous stem cell transplanta tion or an alternative donor transplantation. Patients: younger than 20 yea rs may be treated with an unrelated donor transplantation. Patients older t han 40 years, and probably also patients between 20 and 40 years, may benef it most from an autologous stem cell transplantation.