Busulfan
- Generic Name
- Busulfan
- Brand Names
- Busulfex, Myleran, Busulfan Fresenius Kabi
- Drug Type
- Small Molecule
- Chemical Formula
- C6H14O6S2
- CAS Number
- 55-98-1
- Unique Ingredient Identifier
- G1LN9045DK
- Background
Busulfan is a bifunctional alkylating agent, having a selective immunosuppressive effect on bone marrow. It is not a structural analog of the nitrogen mustards. It has been used in the palliative treatment of chronic myeloid leukemia (myeloid leukemia, chronic), but although symptomatic relief is provided, no permanent remission is brought about. According to the Fourth Annual Report on Carcinogens (NTP 85-002, 1985), busulfan is listed as a known carcinogen.
- Indication
For use in combination with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous (myeloid, myelocytic, granulocytic) leukemia (FDA has designated busulfan as an orphan drug for this use). It is also used as a component of pretransplant conditioning regimens in patients undergoing bone marrow transplantation for acute myeloid leukemia and nonmalignant diseases.
- Associated Conditions
- Chronic Myelogenous Leukemia (CML), Essential Thrombocythemia (ET), Polycythemia Vera (PV)
- Associated Therapies
- Conditioning regimens for allogeneic stem cell transplantation therapy
HLA-Mismatched Unrelated Donor Hematopoietic Cell Transplantation With Post-Transplantation Cyclophosphamide
- Conditions
- Chronic Myelogenous LeukemiaMixed Phenotype Acute LeukemiaAcute LeukemiaAcute Lymphoblastic LeukemiaChronic Lymphocytic LeukemiaAcute Myelogenous LeukemiaMyelodysplastic SyndromesLymphoma
- Interventions
- Radiation: Total-body irradiationProcedure: PBSC Hematopoietic Stem Cell Transplantation (HSCT)Procedure: Bone Marrow Hematopoietic Stem Cell TransplantationOther: Patient-Reported Outcomes
- First Posted Date
- 2021-05-27
- Last Posted Date
- 2024-05-23
- Target Recruit Count
- 300
- Registration Number
- NCT04904588
- Locations
- 🇺🇸
Levine Cancer Institute, Charlotte, North Carolina, United States
🇺🇸Ohio State Medical Center, James Cancer Center, Columbus, Ohio, United States
🇺🇸City of Hope National Medical Center, Duarte, California, United States
A Modified Dose of Rabbit Anti-thymocyte Globulin (rATG) in Children and Adults Receiving Treatment to Help Prepare Their Bodies for a Bone Marrow Transplant
- Conditions
- Acute Myeloid Leukemia (AML)Acute Lymphoid Leukemia (ALL)Myelodysplastic Syndromes (MDS)
- Interventions
- Other: Personalized rATG (P-rATG)Radiation: Hyper fractionated total body irradiationDrug: GCSF
- First Posted Date
- 2021-05-04
- Last Posted Date
- 2025-04-18
- Lead Sponsor
- Memorial Sloan Kettering Cancer Center
- Target Recruit Count
- 59
- Registration Number
- NCT04872595
- Locations
- 🇺🇸
Memorial Sloan Kettering Cancer Center, New York, New York, United States
Itacitinib for the Prevention of Graft Versus Host Disease
- Conditions
- Hematologic and Lymphocytic Disorder
- Interventions
- Procedure: Allogeneic Hematopoietic Stem Cell Transplantation
- First Posted Date
- 2021-04-26
- Last Posted Date
- 2025-04-08
- Lead Sponsor
- M.D. Anderson Cancer Center
- Target Recruit Count
- 31
- Registration Number
- NCT04859946
- Locations
- 🇺🇸
M D Anderson Cancer Center, Houston, Texas, United States
Target Busulfan Exposure in Children With HSCT in China
- Conditions
- Hematopoietic Stem Cell Transplantation
- First Posted Date
- 2021-03-08
- Last Posted Date
- 2021-03-10
- Lead Sponsor
- The First Affiliated Hospital of Soochow University
- Target Recruit Count
- 500
- Registration Number
- NCT04786002
- Locations
- 🇨🇳
the First Affiliated Hospital of Soochow University, Suzhou, Jiangsu, China
Pre-transplant Immunosuppression and Donor Stem Cell Transplant for the Treatment of Severe Hemoglobinopathies
- Conditions
- Sickle Beta ThalassemiaBeta Thalassemia MajorSickle Beta 0 ThalassemiaSickle Beta Plus ThalassemiaSickle Cell-SS DiseaseSickle Cell Disease
- Interventions
- Procedure: Hematopoietic Cell TransplantationProcedure: Plasmapheresis
- First Posted Date
- 2021-03-02
- Last Posted Date
- 2024-10-24
- Lead Sponsor
- M.D. Anderson Cancer Center
- Registration Number
- NCT04776850
G-CSF+DAC+BUCY vs G-CSF+DAC+BF Conditioning Regimen for RAEB-1,REAB-2 and AML Secondary to MDS Undergoing Allo-HSCT
- Conditions
- ConditioningMyelodysplastic SyndromeAllogeneic Hematopoietic Stem Cell Transplantation
- Interventions
- Drug: Granulocyte Colony-Stimulating Factor(G-CSF)
- First Posted Date
- 2021-01-19
- Last Posted Date
- 2021-01-19
- Lead Sponsor
- Nanfang Hospital, Southern Medical University
- Target Recruit Count
- 242
- Registration Number
- NCT04713956
- Locations
- 🇨🇳
Department of Hematology,Nanfang Hospital, Southern Medical University, Guangzhou, Guangdong, China
Venetoclax to Improve Outcomes of Fractionated Busulfan Regimen in Patients With High-Risk AML and MDS
- Conditions
- Myelodysplastic SyndromeChronic Myelomonocytic LeukemiaAcute Myeloid Leukemia
- Interventions
- Procedure: Hematopoietic Cell Transplantation
- First Posted Date
- 2021-01-13
- Last Posted Date
- 2025-04-25
- Lead Sponsor
- M.D. Anderson Cancer Center
- Target Recruit Count
- 324
- Registration Number
- NCT04708054
- Locations
- 🇺🇸
M D Anderson Cancer Center, Houston, Texas, United States
CNCT19 Following ASCT in Patients With Relapsed or Refractory B-cell Lymphoma
- Conditions
- Large B-cell Lymphoma
- Interventions
- First Posted Date
- 2020-12-30
- Last Posted Date
- 2025-05-13
- Target Recruit Count
- 30
- Registration Number
- NCT04690192
- Locations
- 🇨🇳
Institute of Hematology & Blood Diseases Hospital, Tianjin, Tianjin, China
A Single-arm Trial of Prophylactic Tocilizumab for Acute GVHD Prevention After Haploidentical HSCT.
- Conditions
- LeukemiaGraft-versus-host-disease
- Interventions
- First Posted Date
- 2020-12-29
- Last Posted Date
- 2023-02-21
- Lead Sponsor
- Yi Luo
- Target Recruit Count
- 46
- Registration Number
- NCT04688021
- Locations
- 🇨🇳
The First Affiliated Hospital, College of Medicine, Zhejiang University, Hangzhou, China
Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders
- Conditions
- AMLALLNHL or HLInherited Metabolic DisordersBone Marrow FailureHLHMDSMPD Withou MyelofibrosisHemoglobinopathies
- Interventions
- Biological: Cord Blood Graft
- First Posted Date
- 2020-11-25
- Last Posted Date
- 2024-12-02
- Lead Sponsor
- Memorial Sloan Kettering Cancer Center
- Target Recruit Count
- 31
- Registration Number
- NCT04644016
- Locations
- 🇺🇸
Memorial Sloan Kettering Cancer Center, New York, New York, United States
Drug Development Updates
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