2016 Volume 5 Issue 2 Pages 35-40
An allogeneic stem cell transplantation (allo-SCT) from a human leukocyte antigen (HLA) -identical sibling donor is the first treatment of choice for young patients with aplastic anemia (AA) while an allo-SCT from an alternative donors, such as an HLA-identical unrelated donor, and umbilical cord blood transplantation (CBT) need to be considered for AA patients whose disease is refractory to immunosuppressive therapy. The use of reduced-intensity conditioning regimens that ensure the engraftment of donor stem cells, and the development of more effective methods for preventing graft-versus-host disease (GVHD) would help to improve the outcomes of allo-SCT from alternative donors for AA. A reduced-dose cyclophosphamide (CY) regimen combined with fludarabine was developed to avoid cardiotoxicity due to CY and has since become the standard conditioning regimen for allo-SCT from alternative donors for AA. Recent reports have shown that alemtuzumab is more effective than anti-thymocyte globulin at preventing GVHD. Intensive immunosuppression based on strong conditioning regimens might be useful for reducing the risk of engraftment failure associated with CBT. The combined use of an HLA-haploidentical SCT from a related donor and the post-transplant administration of the CY regimen is currently attracting attention as a useful approach to SCT for AA due to its low transplant-related mortality rate and very low incidence of chronic GVHD.
