treosulfan + fludarabine + rabbit antithymocyte globulin

This treosulfan-based conditioning regimen is a combination therapy being evaluated by the Fred Hutchinson Cancer Center for patients with various inherited bone marrow failure syndromes (IBMFS) undergoing hematopoietic cell transplantation. The regimen consists of treosulfan, a bifunctional alkylating agent that induces DNA cross-linking; fludarabine phosphate, a purine antimetabolite that inhibits DNA synthesis; and rabbit antithymocyte globulin (rATG), which provides potent immunosuppression through T-cell depletion. This specific combination is designed to achieve stable donor engraftment with reduced toxicity compared to traditional myeloablative regimens, such as those using busulfan or total body irradiation. It is currently being studied in a Phase II clinical trial (BMT CTN 1904) for conditions including Diamond-Blackfan anemia, Shwachman-Diamond syndrome, GATA2 deficiency, and SAMD9/SAMD9L-related disorders, where patients often have increased sensitivity to conventional chemotherapy.

Other names

treosulfan-based conditioning regimentreosulfan + fludarabine + rATGTreosulfan/Fludarabine/rATG