bone marrow transplant-Medical College of Wisconsin-severe aplastic anemia

This therapeutic protocol, developed and investigated by the Medical College of Wisconsin in the BMT CTN 2207 (CureAA) trial, is a specialized treatment for newly diagnosed patients with severe aplastic anemia (SAA). The protocol utilizes an allogeneic bone marrow transplant (BMT) from haploidentical related, partially HLA-mismatched, or matched unrelated donors. To facilitate engraftment and minimize toxicity, the procedure employs a non-myeloablative conditioning regimen consisting of antithymocyte globulin (ATG), fludarabine, cyclophosphamide, and low-dose total body irradiation (TBI). Post-transplant management includes a specific graft-versus-host disease (GVHD) prophylaxis regimen featuring post-transplant cyclophosphamide (PTCy), tacrolimus, and mycophenolate mofetil (MMF). This approach aims to provide a curative option for SAA patients who lack a matched sibling donor by expanding the donor pool and utilizing a reduced-intensity conditioning strategy.

Other names

Bone Marrow Transplant for Severe Aplastic AnemiaBMT CTN CureAANon-Myeloablative Conditioning and Bone Marrow Transplantation for SAA