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This is a specialized hematopoietic stem cell transplantation (HSCT) protocol developed by the University of Illinois at Chicago (UIC) for the treatment of adult patients with clinically aggressive sickle cell disease. The procedure utilizes HLA-haploidentical (half-matched) peripheral blood stem cells from family donors, significantly expanding the donor pool compared to traditional matched-sibling transplants. The protocol features a non-myeloablative conditioning regimen designed to minimize toxicity, including rabbit antithymocyte globulin (ATG), fludarabine, cyclophosphamide, and low-dose total body irradiation (3 Gy). Post-transplant, a combination of cyclophosphamide, mycophenolate mofetil, and sirolimus is administered for graft-versus-host disease (GVHD) prophylaxis. Clinical studies have demonstrated high rates of stable donor engraftment and reversal of the sickle cell phenotype in the majority of treated patients.
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