busulfan + treosulfan

Busulfan and treosulfan are bifunctional alkylating agents widely utilized as key components of conditioning regimens prior to allogeneic or autologous hematopoietic stem cell transplantation (HSCT). While busulfan has historically served as a standard myeloablative conditioning agent, its use is often limited by significant extramedullary toxicities, such as sinusoidal obstruction syndrome (SOS), neurotoxicity, and pulmonary complications. Treosulfan, a water-soluble analog of busulfan, acts as a prodrug that non-enzymatically converts into active epoxide derivatives to alkylate DNA. Treosulfan has emerged as a reduced-toxicity myeloablative alternative, offering comparable stem-cell-depleting and immunosuppressive efficacy with a more favorable safety profile, particularly in elderly, pediatric, or comorbid patients.

Other names

BU/TreoBU/Treo regimenbusulfan and treosulfanbusulfan/treosulfantreosulfan + busulfantreosulfan/busulfan