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Fludarabine and Busulfan
Fludarabine and busulfan work together as a myeloablative conditioning regimen that depletes bone marrow and suppresses the immune system to prepare patients for hematopoietic stem cell transplantation.
Fludarabine and busulfan work together as a myeloablative conditioning regimen that depletes bone marrow and suppresses the immune system to prepare patients for hematopoietic stem cell transplantation. Used for Conditioning regimen prior to hematopoietic stem cell transplantation for hematologic malignancies, Acute myeloid leukemia (AML), Acute lymphoblastic leukemia (ALL).
At a glance
| Generic name | Fludarabine and Busulfan |
|---|---|
| Also known as | Fludara and Busulfex |
| Sponsor | Union Hospital, Tongji Medical College, Huazhong University of Science and Technology |
| Drug class | Myeloablative conditioning regimen (combination chemotherapy) |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 3 |
Mechanism of action
Fludarabine is a purine analog nucleoside that inhibits DNA synthesis and repair, leading to apoptosis of lymphocytes and other rapidly dividing cells. Busulfan is an alkylating agent that causes DNA cross-linking and cell death. Together, they provide intensive immunosuppression and myeloablation to eliminate malignant cells and create space for donor stem cell engraftment in transplant recipients.
Approved indications
- Conditioning regimen prior to hematopoietic stem cell transplantation for hematologic malignancies
- Acute myeloid leukemia (AML)
- Acute lymphoblastic leukemia (ALL)
- Chronic myeloid leukemia (CML)
Common side effects
- Myelosuppression/neutropenia
- Thrombocytopenia
- Anemia
- Mucositis
- Nausea and vomiting
- Hepatotoxicity
- Graft-versus-host disease (GVHD)
- Infection
Key clinical trials
- Venetoclax or Placebo in Combination With Reduced-Intensity Conditioning Hematopoietic Cell (Bone Marrow/Blood Stem Cell) Transplant and as Maintenance Therapy After Transplant in Patients With Acute Myeloid Leukemia (A MyeloMATCH Treatment Trial) (PHASE2)
- MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial) (PHASE2)
- Pilot Study of Reduced-Intensity Hematopoietic Stem Cell Transplant of DOCK8 Deficiency (PHASE2)
- US Study of ECT-001-CB in Pediatric and Young Adult Patients With High-Risk Myeloid Malignancies (PHASE1, PHASE2)
- 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)
- A Platform Protocol to Investigate Post-Transplant Cyclophosphamide-Based Graft-Versus-Host Disease Prophylaxis in Patients With Hematologic Malignancies Undergoing Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation (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 |
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