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This combination is a reduced toxicity myeloablative conditioning (RTC) regimen specifically designed for patients with Wiskott-Aldrich Syndrome (WAS) undergoing hematopoietic stem cell transplantation (HSCT). Developed by The Korean Society of Pediatric Hematology Oncology, the regimen aims to balance the need for robust donor cell engraftment with the necessity of minimizing treatment-related toxicities often seen with conventional high-dose conditioning. It consists of fludarabine (a purine antimetabolite), busulfan (a bifunctional alkylating agent), and Thymoglobulin (rabbit anti-thymocyte globulin). Fludarabine and busulfan work synergistically to provide myeloablation and lymphotoxicity, while Thymoglobulin provides potent T-cell depletion to prevent graft-versus-host disease (GVHD) and facilitate engraftment, particularly in mismatched or cord blood transplants.
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