alemtuzumab + fludarabine + busulfan + cyclosporine + mycophenolate mofetil

A reduced-intensity conditioning (RIC) regimen, specifically the "Mini Busulfan" protocol developed by the Children's Hospital of Philadelphia (CHOP). This combination therapy is used to prepare pediatric and young adult patients with non-malignant disorders for allogeneic hematopoietic stem cell transplantation (HSCT). The regimen consists of alemtuzumab (Campath) for lymphodepletion, fludarabine and a reduced dose of busulfan for myelosuppression, and a combination of cyclosporine and mycophenolate mofetil (Cellcept) for immunosuppression and graft-versus-host disease (GVHD) prophylaxis. By using lower doses of alkylating agents compared to standard myeloablative conditioning, this regimen aims to achieve donor engraftment and stable mixed chimerism while reducing the risk of severe transplant-related toxicities. It is being evaluated for various conditions including primary immunodeficiencies (e.g., SCID, WAS, IPEX), hemoglobinopathies (e.g., sickle cell disease, thalassemia), and histiocytic disorders (e.g., HLH).

Other names

Mini Busulfan regimenCHOP RIC Mini BusulfanMini Busulfan