Recent advances and current challenges in allogeneic stem cell transplantation in patients with acquired severe aplastic anemia

Authors: Sung-Eun Lee and Jong Wook Lee

first published: August, 28 2018

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Vol.1 Issue 1 No.2 2018 / BCT-2018-001


Allogeneic stem cell transplantation (SCT) from a human leukocyte antigen (HLA)-matched sibling donor (MSD-SCT) is the preferred first-line treatment option for younger patients with severe aplastic anemia (SAA). However, only 25% of patients have a chance of finding an HLA-MSD, and for SAA patients who lack a suitable MSD and fail first-line immunosuppressive therapy, SCT from an unrelated donor (URD-SCT) can be considered as a treatment option. The results of haplo-identical stem cell transplantation from a related mismatched donor (Haplo-SCT) have improved due to recent advances in controlling graft failure and graft-versus-host disease (GVHD), and the use of Haplo-SCT has recently extended to SAA. However, the conditioning regimen used to ensure engraftment with reduced toxicity such as infectious complications and GVHD is an important issue.

This review summarizes recent advances in allogeneic SCT for patients with acquired SAA. Special issues including the age of the patient and the effects of donor age, stem cell source, and iron overload on transplant outcomes are also discussed.

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Sung-Eun Lee1, Jong Wook Lee1

1. Department of Hematology, Catholic Blood and Marrow Transplantation Center, Seoul St. Mary’s Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea

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