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Busulphan plus Cyclophosphamide
Busulphan and cyclophosphamide are alkylating agents that damage DNA in cancer cells and bone marrow cells, used together as a myeloablative conditioning regimen prior to hematopoietic stem cell transplantation.
Busulphan and cyclophosphamide are alkylating agents that damage DNA in cancer cells and bone marrow cells, used together as a myeloablative conditioning regimen prior to hematopoietic stem cell transplantation. Used for Conditioning regimen for allogeneic hematopoietic stem cell transplantation in hematologic malignancies, Acute myeloid leukemia, Acute lymphoblastic leukemia.
At a glance
| Generic name | Busulphan plus Cyclophosphamide |
|---|---|
| Also known as | I.V. BuCy2 |
| Sponsor | Gruppo Italiano Trapianto di Midollo Osseo |
| Drug class | Alkylating agents |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 3 |
Mechanism of action
Busulphan is a bifunctional alkylating agent that cross-links DNA strands, while cyclophosphamide is a nitrogen mustard derivative that also alkylates DNA. When combined, they provide intensive myeloablation to eliminate malignant hematopoietic cells and suppress the immune system to reduce graft rejection risk in bone marrow transplant recipients. This combination is a standard preparative regimen for allogeneic hematopoietic stem cell transplantation.
Approved indications
- Conditioning regimen for allogeneic hematopoietic stem cell transplantation in hematologic malignancies
- Acute myeloid leukemia
- Acute lymphoblastic leukemia
- Chronic myeloid leukemia
Common side effects
- Myelosuppression
- Mucositis
- Nausea and vomiting
- Hepatotoxicity
- Hemorrhagic cystitis
- Graft-versus-host disease
- Infections
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)
- HLA-Mismatched Unrelated Donor Hematopoietic Cell Transplantation With Post-Transplantation Cyclophosphamide (PHASE2)
Primary sources
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| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |