Last reviewed · How we verify
busulfex
At a glance
| Generic name | busulfex |
|---|---|
| Sponsor | Dana-Farber Cancer Institute |
| Target | Matrix metalloproteinase-9 |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | FDA-approved |
Approved indications
- Allogeneic bone marrow transplantation
- Autologous hematopoietic stem-cell transplantation for Ewing's sarcoma family of tumors
- Malignant lymphoma
- Neuroblastoma
- Philadelphia Chromosome Positive Chronic Myelocytic Leukemia
Common side effects
- Myelosuppression
- Nausea
- Stomatitis
- Vomiting
- Anorexia
- Diarrhea
- Insomnia
- Fever
- Hypomagnesemia
- Abdominal pain
- Anxiety
- Headache
Serious adverse events
- Graft-versus-host disease (GVHD)
- Alveolar hemorrhage
- Pneumonia
- Interstitial fibrosis
- Cerebral hemorrhage
- Hepatic veno-occlusive disease (HVOD)
- Hemorrhagic cystitis
- Thrombotic microangiopathy (TMA)
- Tumor lysis syndrome
- Sepsis
Key clinical trials
- Pilot Trial of Allogeneic Blood or Marrow Transplantation for Primary Immunodeficiencies (PHASE2)
- Part B- G1X-CGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease (PHASE1, PHASE2)
- Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID) (PHASE1, PHASE2)
- 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)
- Base Editing Hematopoietic Stem Cell and T Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study (PHASE1, 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)
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:
- busulfex CI brief — competitive landscape report
- busulfex updates RSS · CI watch RSS
- Dana-Farber Cancer Institute portfolio CI