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busulfan + cyclophosphamide + melphalan
- Development stage
- Unknown
- Lead developer
- GSK
- Modality
- Nucleic Acid-Directed Small Molecules → Small Molecules, Covalent Small Molecules → Small Molecules, Classical Binding Small Molecules → Small Molecules
- Administration
- Intravenous, Oral
01
Overview
Busulfan + cyclophosphamide + melphalan is a combination of three alkylating agents used as a myeloablative conditioning regimen prior to hematopoietic stem cell transplantation (HSCT), particularly in pediatric and adult patients with acute myeloid leukemia (AML), multiple myeloma (MM), chronic myelogenous leukemia (CML), acute lymphoblastic leukemia (ALL), and non-Hodgkin's lymphoma (NHL)[1][2]. Each component—busulfan, cyclophosphamide, and melphalan—acts by cross-linking DNA strands, thereby inhibiting DNA replication and leading to cell death. This regimen provides potent anti-leukemic effects but is associated with significant extramedullary toxicities such as veno-occlusive disease of the liver and hemorrhagic cystitis[1][2]. The combination is typically used in high-risk or relapsed/refractory settings where intensive cytoreduction is required before autologous or allogeneic stem cell transplantation.
- Other names
- BuCyMelBUCY-2 plus melphalanBUCY2 plus melphalanBUCY 2 plus melphalan
02
Targets
DNA
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