Successful unrelated cord blood transplantation in a child with very severe aplastic anemia

Abstract

Unrelated cord blood transplantation (UCBT) and mismatched related donor stem cell transplantation (MMRD-SCT) have been considered as alternative therapeutic options for patients with very severe aplastic anemia (VSAA) and severe infection when an HLA-matched donor is not available. However, a high rate of graft failure and severe graft-versus-host disease (GVHD) are the major problems in SCT from these alternative donors. Here, we report the case of a 2-year-old boy with VSAA complicated by repeated infection who was successfully treated by UCBT (HLA 5/6 antigen-matched). Cord blood with a sufficient number of total nucleated and CD34-positive cells was used, and a conditioning regimen including low-dose TBI was employed. Iron-chelating therapy was performed before UCBT. Engraftment was achieved on day 20 without severe acute GVHD. Although CMV antigenemia persisted for 5 months, which might have resulted from ATG use in the conditioning regimen, UCBT may become a good alternative therapeutic option for VSAA.