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This myeloablative preparative regimen is a combination conditioning therapy used prior to allogeneic hematopoietic stem cell transplantation (HSCT). Developed and evaluated by the Masonic Cancer Center at the University of Minnesota, specifically in the MT2014-10C clinical trial, the regimen consists of the monoclonal antibody alemtuzumab and the alkylating agents cyclophosphamide and busulfan. It is designed for patients with high-risk hemoglobinopathies and other non-malignant red cell transfusion-dependent disorders, including sickle cell disease, thalassemia, and Diamond-Blackfan anemia. Alemtuzumab serves as a potent lymphodepleting agent to prevent graft rejection and graft-versus-host disease (GVHD), while cyclophosphamide and busulfan provide intensive myeloablation to clear the recipient's bone marrow, facilitating the engraftment of donor stem cells.
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