Mini Busulfan

Mini Busulfan is a reduced intensity conditioning (RIC) regimen developed by the Children's Hospital of Philadelphia (CHOP) for pediatric and young adult patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT) for non-malignant disorders. The regimen is designed to achieve stable mixed chimerism sufficient for cure while minimizing transplant-related toxicity compared to standard myeloablative conditioning. It specifically consists of a combination of alemtuzumab (Campath), fludarabine, busulfan, cyclosporine, and mycophenolate mofetil (Cellcept). This protocol is utilized for a variety of conditions including IPEX syndrome, sickle cell disease, thalassemia major, and various primary immunodeficiencies. By combining lymphodepletion (alemtuzumab), myelosuppression (busulfan, fludarabine), and post-transplant immunosuppression (cyclosporine, mycophenolate mofetil), the regimen facilitates donor engraftment with a lower risk of organ toxicity and transplant-related mortality.

Other names

RIC: Mini BusulfanCHOP RIC Mini Busulfan regimenAlemtuzumab + Fludarabine + Busulfan + Cyclosporine + Mycophenolate mofetil