Alternative donor marrow transplantation in children with aplastic anemia using low-dose irradiation and fludarabine-based conditioning.

摘要

Allogeneic BMT is a curative treatment for patients with severe aplastic anemia (SAA). In particular, BMT from a HLA-identical sibling is an established treatment for children with acquired SAA, but the results of alternative donor (AD) transplantation have been less favorable because of the high rates of graft failure and severe acute GVHD. However, recent data have shown improvements in the survival of patients receiving unrelated donor (UD) transplantation possibly due to better donor matching attributable to the use of high-resolution HLA typing data. Two major studies have been performed in the setting of UD transplantation for SAA. The first is a Japanese study that reported 154 SAA patients undergoing UD transplantation using CY/TBI/anti-thymocyte globulin (ATG) and also CY/limited field irradiation/ ATG: 11% showed rejection whereas 56% survived. The second involves a group in the USA, which reported a study that tested the deescalating doses of TBI (from 6 to 2 Gy) in combination with CY and ATG in UD transplantation for SAA, and showed the best outcomes in patients receiving 2Gy, with 8 of 13 patients surviving.