2022 Volume 11 Issue 1 Pages 36-42
The purpose of pre-transplant conditioning in allogeneic hematopoietic stem cell transplantation for patients with aplastic anemia is to avoid graft failure and prevent graft-versus-host disease (GVHD). Although the combination of high-dose cyclophosphamide (CY) and anti-thymocyte globulin (ATG) was developed as an effective conditioning regimen, cardiac toxicity due to high-dose CY has been a major problem. Reduction of the CY dose with the addition of fludarabine (Flu) has recently been considered to decrease toxicity. In the Kanto Study Group for Cell Therapy (KSGCT), a prospective study using Flu, reduced-dose CY and low-dose thymoglobulin as a conditioning regimen for aplastic anemia was performed to determine the appropriate regimen for Japanese patients. This study showed an excellent overall survival rate of 96.3% at 1 year. However, the use of Flu may affect the occurrence of secondary graft failure. Recently, outcomes in cord blood transplantation and transplantation from a haploidentical donor are improving with the appropriate preconditioning regimens. In addition, alemtuzumab may be a promising drug for pre-transplant conditioning for aplastic anemia.
