The Case for Upfront HLA-Matched Unrelated Donor Hematopoietic Stem Cell Transplantation as a Curative Option for Adult Acquired Severe Aplastic Anemia

Allogeneic HLA-matched unrelated donor (MUD) hematopoietic stem cell transplantation (HSCT) for adults with acquired severe aplastic anemia (SAA) is currently recommended after failure to respond to a course of immunosuppressive therapy (IST) [1,2]. This guidance reflects the historical developments in HSCT and IST, as well as the improving outcomes of MUD HSCT over time. Although patients with SAA are surviving long term after both HSCT and IST, the long-term complications of clonal transformation to myelodysplastic syndrome (MDS) and acute myelogenous leukemia (AML) and hemolytic paroxysmal nocturnal hemoglobinuria (PNH) after IST in up to 15% to 26% of patients at 10 years are notable [3-7].
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research