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This therapeutic protocol refers to an allogeneic hematopoietic stem cell transplantation (HSCT) procedure using bone marrow from a matched unrelated donor (MUD) for the treatment of newly diagnosed severe aplastic anemia (SAA). Investigated by the Medical College of Wisconsin in the BMT CTN 2207 (CureAA) trial, the protocol employs a non-myeloablative conditioning regimen designed to reduce toxicity while ensuring engraftment. The conditioning regimen includes antithymocyte globulin (ATG), fludarabine, cyclophosphamide, and low-dose total body irradiation (TBI). To prevent graft-versus-host disease (GVHD), the protocol utilizes post-transplant cyclophosphamide (PTCy), tacrolimus, and mycophenolate mofetil (MMF). This approach aims to provide a curative option for patients who lack a matched sibling donor, potentially establishing unrelated donor transplant as a viable first-line therapy for SAA.
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