Umbilical cord blood transplantation for a child with very severe aplastic anemia refractory to immunosuppressive treatment

Abstract

It is a great challenge to treat patients with very severe aplastic anemia (VSAA), who lack an HLA-matched related donor, and who fail to respond to immunosuppressive treatment (IST). Although cord blood transplantation (CBT) is a treatment option for hematological malignancies in the absence of a suitable unrelated donor, there is no evidence so far of the effectivity of CBT for aplastic anemia (AA). We performed semiurgent CBT to control recurrent infections in a patient with VSAA, which was refractory to IST and complicated by infections. The conditioning regimen consisted of fludarabine, melphalan, and low-dose total body irradiation, and GVHD prophylaxis consisted of tacrolimus and mycophenolate mofetil. Neutrophil engraftment was achieved on day 16. Complications such as Grade I acute GVHD, hemophagocytic syndrome, and virus-associated hemorrhagic cystitis were observed; however, they were tolerable. CBT could be an alternative treatment option for VSAA in the absence of an unrelated donor.