cyclophosphamide + fludarabine + anti-thymocyte globulin + total body irradiation + sirolimus + mycophenolate mofetil

This therapeutic regimen is a non-myeloablative (reduced-intensity) conditioning and transplantation protocol developed at the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins for the treatment of severe sickle cell anemia and other hemoglobinopathies. The protocol utilizes a multi-drug conditioning phase consisting of fludarabine, cyclophosphamide, and low-dose total body irradiation (TBI) to achieve lymphodepletion and myelosuppression, facilitating the engraftment of donor bone marrow (often from haploidentical or matched donors). A hallmark of this approach is the administration of high-dose post-transplantation cyclophosphamide (PTCy) on days +3 and +4, which selectively depletes alloreactive T cells to prevent graft-versus-host disease (GVHD) and graft rejection. Post-transplant immunosuppression is maintained using sirolimus and mycophenolate mofetil. This regimen is designed to establish stable donor chimerism and cure the underlying blood disorder while minimizing the severe toxicities typically associated with traditional myeloablative bone marrow transplantation.

Other names

Johns Hopkins non-myeloablative BMT regimenBaltimore ProtocolHopkins Protocol for Sickle Cell AnemiaPTCy-based BMT for hemoglobinopathies