MedPath

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

G-CSF+DAC+BUCY vs G-CSF+DAC+BF Conditioning Regimen for High-risk MDS Undergoing Allo-HSCT

Phase 2
Recruiting
Conditions
Myelodysplastic Syndrome
Allogeneic Hematopoietic Stem Cell Transplantation
Conditioning
Interventions
First Posted Date
2022-07-12
Last Posted Date
2023-03-21
Lead Sponsor
Nanfang Hospital, Southern Medical University
Target Recruit Count
242
Registration Number
NCT05453552
Locations
🇨🇳

Department of Hematology,Nanfang Hospital, Southern Medical University, Guangzhou, Guangdong, China

G-CSF+DAC+BUCY vs G-CSF+DAC+BF Conditioning Regimen for Secondary Acute Myeloid Leukemia Undergoing Allo-HSCT

Phase 2
Recruiting
Conditions
Conditioning
Secondary Acute Myeloid Leukemia Evolving From MDS
Allogeneic Hematopoietic Stem Cell Transplantation
Interventions
First Posted Date
2022-07-08
Last Posted Date
2022-07-08
Lead Sponsor
Nanfang Hospital, Southern Medical University
Target Recruit Count
232
Registration Number
NCT05449899
Locations
🇨🇳

Department of Hematology,Nanfang Hospital, Southern Medical University, Guangzhou, Guangdong, China

Donor Lymphocyte Infusion After Allogeneic Hematopoietic Cell Transplantation for High-Risk Hematologic Malignancies

Phase 1
Recruiting
Conditions
Hematologic Neoplasms
Interventions
First Posted Date
2022-04-14
Last Posted Date
2025-04-27
Lead Sponsor
National Cancer Institute (NCI)
Target Recruit Count
430
Registration Number
NCT05327023
Locations
🇺🇸

National Institutes of Health Clinical Center, Bethesda, Maryland, United States

Allogeneic Hematopoietic Stem Cell Transplantation for 4/M Neuroblastoma

First Posted Date
2022-03-31
Last Posted Date
2022-07-01
Lead Sponsor
Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
Target Recruit Count
64
Registration Number
NCT05303727
Locations
🇨🇳

Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou, Guangdong, China

Cryopreserved MMUD BM With PTCy for Hematologic Malignancies

Phase 1
Withdrawn
Conditions
T-lymphoblastic Lymphoma
Acute Biphenotypic Leukemia
Acute Lymphocytic Leukemia
Acute Myeloid Leukemia
Acute Leukemia
Acute Undifferentiated Leukemia
Myelodysplastic Syndromes
Interventions
First Posted Date
2021-12-28
Last Posted Date
2023-11-01
Lead Sponsor
Ossium Health, Inc.
Registration Number
NCT05170828

A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome

Phase 2
Recruiting
Conditions
Immunodeficiency
Hematopoietic Stem Cell Transplantation
Interventions
First Posted Date
2021-08-31
Last Posted Date
2025-05-15
Lead Sponsor
National Cancer Institute (NCI)
Target Recruit Count
54
Registration Number
NCT05027945
Locations
🇺🇸

National Institutes of Health Clinical Center, Bethesda, Maryland, United States

Induction Therapy for Patients With FLT3 Mutated Acute Myeloid Leukemia

Phase 1
Withdrawn
Conditions
Acute Myeloid Leukemia
Interventions
Biological: CD34+ selected allogeneic stem cell transplant from an HLA-compatible donor
First Posted Date
2021-07-29
Last Posted Date
2024-10-01
Lead Sponsor
Guenther Koehne
Target Recruit Count
20
Registration Number
NCT04982354
Locations
🇺🇸

Miami Cancer Institute at Baptist Health of South Florida, Miami, Florida, United States

Outcomes of Patients After Allo-HSCT With Decitabine and NAC

First Posted Date
2021-06-30
Last Posted Date
2021-06-30
Lead Sponsor
The First Affiliated Hospital of Soochow University
Target Recruit Count
100
Registration Number
NCT04945096

Benadamustine, Fludarabine and Busulfan Conditioning in Recipients of Haploidentical Stem Cell Transplantation (FluBuBe)

Phase 2
Completed
Conditions
Leukemia, Acute Lymphoblastic
Myeloid Leukemia, Acute
Myelodysplastic Syndromes
Myeloproliferative Neoplasm
Lymphoblastic Lymphoma
Biphenotypic Acute Leukemia
Interventions
First Posted Date
2021-06-28
Last Posted Date
2024-05-07
Lead Sponsor
St. Petersburg State Pavlov Medical University
Target Recruit Count
50
Registration Number
NCT04942730
Locations
🇷🇺

RM Gorbacheva Research Institute, Saint Petersburg, Russian Federation

© Copyright 2025. All Rights Reserved by MedPath