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busulfan + cyclophosphamide + melphalan + anti-thymocyte globulin followed by hematopoietic cell transplantation
- Development stage
- Unknown
- Lead developer
- Masonic Cancer Center
- Modality
- Small Molecules, Antibody-Based Therapeutics, Stem Cell Therapies → Cell Therapies
- Administration
- Intravenous
01
Overview
This treatment protocol, developed by the Masonic Cancer Center at the University of Minnesota, is a specialized hematopoietic cell transplantation (HCT) procedure for children with juvenile myelomonocytic leukemia (JMML). The protocol involves a multi-agent conditioning regimen consisting of busulfan, cyclophosphamide, and melphalan (BU/CY/L-PAM), often supplemented with mesna and anti-thymocyte globulin (ATG). This intensive chemotherapy aims to eradicate the patient's leukemic clones and provide sufficient immunosuppression to allow for the engraftment of healthy donor hematopoietic stem cells, typically sourced from bone marrow or umbilical cord blood. JMML is a rare and aggressive myelodysplastic/myeloproliferative neoplasm where allogeneic HCT remains the only curative option.
- Other names
- HCT for JMML protocol (University of Minnesota)BU/CY/L-PAM conditioning regimenHematopoietic Cell Transplantation in Children With Juvenile Myelomonocytic Leukemia
02
Targets
DNA
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