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Allogeneic hematopoietic stem cell transplant for severe aplastic anemia: Current state and future directions.

Abstract
Acquired severe aplastic anemia (SAA) is a rare and life-threatening bone marrow failure syndrome characterized by cytotoxic T-cells excessive activity, hematopoietic precursors decrease and peripheral blood (PB) pancytopenia. Patients with severe aplastic anemia (SAA) died 1 to 2 years after diagnosis due to fatal infections and/or hemorrhagic complications if they went without any effective treatment. Nowadays, Immunosuppressive therapy (IST) and allogeneic hematopoietic stem cell transplantation (HSCT) are still the standard treatment for SAA. For patients younger than 40 years old, allogeneic HSCT were often the best choice. Recently, outcomes of matched unrelated donor and haploidentical donor transplantation have significant improved, notably in some cases which are comparable to the result of matched related donor transplantation. Mixed chimerism status was more common in SAA post-transplantation patients, which was effected by conditioning regimen used in transplantation and closely relevant to donor cells rejection and secondary graft failure. In this article, we briefly review the current state and future directions for SAA HSCT, and share our SAA data and transplant experience in recently decade. We analyzed the impact of conditioning regimen on engraftment and chimerism status in SAA transplantation, and have compiled our findings in this report.

PMID: 28245775 [Pubmed - Publisher]

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