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This preparative regimen is a multi-agent conditioning therapy developed by the Masonic Cancer Center at the University of Minnesota specifically for pediatric patients with juvenile myelomonocytic leukemia (JMML) undergoing hematopoietic cell transplantation (HCT). The regimen consists of the alkylating agents busulfan, cyclophosphamide, and melphalan, which serve to eradicate malignant cells and provide myeloablation. Mesna is included as a uroprotective agent to prevent cyclophosphamide-induced hemorrhagic cystitis, and anti-thymocyte globulin (ATG) is utilized for immunosuppression to prevent graft-versus-host disease and facilitate engraftment. The goal of this intensive combination is to achieve long-term disease-free survival in JMML, a rare and aggressive myeloproliferative neoplasm of childhood.
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