Outcome of peripheral blood stem cell transplantation from HLA-identical sibling donors for adult patients with aplastic anemia.

Although bone marrow transplantation is the recommended form of allogeneic hematopoietic stem cell transplantation for aplastic anemia, some patients undergo peripheral blood stem cell transplantation (PBSCT). Therefore, there is critical demand to identify factors affecting transplantation outcomes. Using the Japanese registry database, we retrospectively analyzed outcomes of 94 adult patients with aplastic anemia who underwent PBSCT from HLA-identical sibling donors. The cumulative incidence of neutrophil engraftment was 94% (95% confidence interval [CI] 86-97%), and was significantly higher in patients who received anti-thymocyte globulin (ATG) in conditioning. The cumulative incidence rate was 26% (95% CI 17-35%) in grades II-IV acute graft-versus-host disease (GVHD) and 20% (95% CI 13-29%) in extensive chronic GVHD, and tended to be lower in patients with chronic GVHD who received ATG-based conditioning. The 5-year overall survival (OS) rate was 70% (95% CI 59-78%). In multivariate analysis, patient age < 40 years, shorter period from diagnosis to transplantation, better performance status, and ATG-based conditioning were significantly correlated with favorable OS. In conclusion, PBSCT from HLA-identical sibling donors for aplastic anemia would result in acceptable outcomes. Several risk factors identified in our study should be considered when selecting a stem cell source.