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Busulfan/Cyclophosphamide
Busulfan and cyclophosphamide are alkylating agents that damage DNA and suppress bone marrow function, used together as a myeloablative conditioning regimen prior to hematopoietic stem cell transplantation.
Busulfan and cyclophosphamide are alkylating agents that damage DNA and suppress bone marrow function, used together as a myeloablative conditioning regimen prior to hematopoietic stem cell transplantation. Used for Conditioning regimen prior to hematopoietic stem cell transplantation for hematologic malignancies, Conditioning regimen prior to hematopoietic stem cell transplantation for severe aplastic anemia.
At a glance
| Generic name | Busulfan/Cyclophosphamide |
|---|---|
| Also known as | Cytoxan |
| Sponsor | Tel-Aviv Sourasky Medical Center |
| Drug class | Alkylating agents |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | FDA-approved |
Mechanism of action
Busulfan is a bifunctional alkylating agent that cross-links DNA strands, while cyclophosphamide is a nitrogen mustard derivative that also alkylates DNA. Together, they create a highly immunosuppressive and myeloablative conditioning regimen that destroys existing bone marrow and immune cells, allowing engraftment of donor hematopoietic stem cells in transplant recipients. This combination is used to treat hematologic malignancies and severe aplastic anemia.
Approved indications
- Conditioning regimen prior to hematopoietic stem cell transplantation for hematologic malignancies
- Conditioning regimen prior to hematopoietic stem cell transplantation for severe aplastic anemia
Common side effects
- Myelosuppression/neutropenia
- Mucositis
- Nausea and vomiting
- Hepatotoxicity/veno-occlusive disease
- Hemorrhagic cystitis
- Alopecia
- Infertility/gonadal dysfunction
Key clinical trials
- Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary Immunodeficiencies (PHASE2)
- Pilot Study of Reduced-Intensity Hematopoietic Stem Cell Transplant of DOCK8 Deficiency (PHASE2)
- Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +/- IFN-gamma) Antagonists (PHASE1, PHASE2)
- US Study of ECT-001-CB in Pediatric and Young Adult Patients With High-Risk Myeloid Malignancies (PHASE1, PHASE2)
- Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide (EARLY_PHASE1)
- Allogeneic Hematopoietic Stem Cell Transplant for GATA2 Mutations (PHASE2)
- Ruxolitinib-Decitabine Intensified Conditioning Regimen for AML: A Randomized Trial (PHASE4)
- Study on the Efficacy and Safety of the TmBU Conditioning Regimen in High-risk or Relapsed/Refractory Acute Leukemia (PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Busulfan/Cyclophosphamide CI brief — competitive landscape report
- Busulfan/Cyclophosphamide updates RSS · CI watch RSS
- Tel-Aviv Sourasky Medical Center portfolio CI