U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

Details

Stereochemistry ACHIRAL
Molecular Formula C6H14O6S2
Molecular Weight 246.302
Optical Activity NONE
Defined Stereocenters 0 / 0
E/Z Centers 0
Charge 0

SHOW SMILES / InChI
Structure of BUSULFAN

SMILES

CS(=O)(=O)OCCCCOS(C)(=O)=O

InChI

InChIKey=COVZYZSDYWQREU-UHFFFAOYSA-N
InChI=1S/C6H14O6S2/c1-13(7,8)11-5-3-4-6-12-14(2,9)10/h3-6H2,1-2H3

HIDE SMILES / InChI

Description
Curator's Comment: description was created based on several sources, including: http://www.drugbank.ca/drugs/DB01008 https://en.wikipedia.org/wiki/Busulfan

Busulfan is a bifunctional alkylating agent, having a selective immunosuppressive effect on bone marrow. It has been used in the palliative treatment of chronic myeloid leukemia (myeloid leukemia, chronic). Most common adverse reactions (incidence greater than 60%) were: myelosuppression, nausea, stomatitis, vomiting, anorexia, diarrhea, insomnia, fever, hypomagnesemia, abdominal pain, anxiety, headache, hyperglycemia and hypokalemia. Itraconazole and acetaminophen can decrease busulfan clearance. Phenytoin increases hepatic clearance of busulfan.

CNS Activity

Curator's Comment: Busulfan is a small, highly lipophilic molecule that easily crosses the blood brain barrier.

Originator

Sources: Lichtman M., 'Hematology: Landmark Papers of the Twentieth Century' (2000), P. 598. ISBN-13 | 978-0124485105

Approval Year

Targets

Targets

Primary TargetPharmacologyConditionPotency
Conditions

Conditions

ConditionModalityTargetsHighest PhaseProduct
Palliative
MYLERAN

Approved Use

BUSULFEX is indicated for use in combination with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous leukemia. BUSULFEX is an alkylating drug indicated for: •Use in combination with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous leukemia (CML) (1)

Launch Date

1954
Cmax

Cmax

ValueDoseCo-administeredAnalytePopulation
1222 ng/mL
0.8 mg/kg 4 times / day steady-state, intravenous
dose: 0.8 mg/kg
route of administration: Intravenous
experiment type: STEADY-STATE
co-administered:
BUSULFAN plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: UNKNOWN
food status: UNKNOWN
AUC

AUC

ValueDoseCo-administeredAnalytePopulation
1167 μM × min
0.8 mg/kg 4 times / day steady-state, intravenous
dose: 0.8 mg/kg
route of administration: Intravenous
experiment type: STEADY-STATE
co-administered:
BUSULFAN plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: UNKNOWN
food status: UNKNOWN
T1/2

T1/2

ValueDoseCo-administeredAnalytePopulation
3.35 h
0.8 mg/kg 4 times / day steady-state, intravenous
dose: 0.8 mg/kg
route of administration: Intravenous
experiment type: STEADY-STATE
co-administered:
BUSULFAN plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: UNKNOWN
food status: UNKNOWN
Funbound

Funbound

ValueDoseCo-administeredAnalytePopulation
67.6%
0.8 mg/kg 4 times / day steady-state, intravenous
dose: 0.8 mg/kg
route of administration: Intravenous
experiment type: STEADY-STATE
co-administered:
BUSULFAN plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: UNKNOWN
food status: UNKNOWN
Doses

Doses

DosePopulationAdverse events​
40 mg/m2 4 times / day multiple, intravenous
MTD
Dose: 40 mg/m2, 4 times / day
Route: intravenous
Route: multiple
Dose: 40 mg/m2, 4 times / day
Sources:
unhealthy, 1.2–17.2
Health Status: unhealthy
Age Group: 1.2–17.2
Sex: M+F
Sources:
Disc. AE: Hepatotoxicity...
AEs leading to
discontinuation/dose reduction:
Hepatotoxicity
Sources:
4 mg/kg 4 times / day multiple, oral
Overdose
Dose: 4 mg/kg, 4 times / day
Route: oral
Route: multiple
Dose: 4 mg/kg, 4 times / day
Sources:
unhealthy, 14
Health Status: unhealthy
Age Group: 14
Sex: F
Sources:
Disc. AE: Seizures...
AEs leading to
discontinuation/dose reduction:
Seizures
Sources:
18 mg/kg single, oral
Overdose
Dose: 18 mg/kg
Route: oral
Route: single
Dose: 18 mg/kg
Sources:
unhealthy, 48
Health Status: unhealthy
Age Group: 48
Sex: F
Sources:
0.8 mg/kg 4 times / day multiple, intravenous
Recommended
Dose: 0.8 mg/kg, 4 times / day
Route: intravenous
Route: multiple
Dose: 0.8 mg/kg, 4 times / day
Sources:
unhealthy
Disc. AE: Myelosuppression, Seizures...
AEs leading to
discontinuation/dose reduction:
Myelosuppression (severe)
Seizures
Venoocclusive disease
Fetal damage
Sources:
AEs

AEs

AESignificanceDosePopulation
Hepatotoxicity Disc. AE
40 mg/m2 4 times / day multiple, intravenous
MTD
Dose: 40 mg/m2, 4 times / day
Route: intravenous
Route: multiple
Dose: 40 mg/m2, 4 times / day
Sources:
unhealthy, 1.2–17.2
Health Status: unhealthy
Age Group: 1.2–17.2
Sex: M+F
Sources:
Seizures Disc. AE
4 mg/kg 4 times / day multiple, oral
Overdose
Dose: 4 mg/kg, 4 times / day
Route: oral
Route: multiple
Dose: 4 mg/kg, 4 times / day
Sources:
unhealthy, 14
Health Status: unhealthy
Age Group: 14
Sex: F
Sources:
Fetal damage Disc. AE
0.8 mg/kg 4 times / day multiple, intravenous
Recommended
Dose: 0.8 mg/kg, 4 times / day
Route: intravenous
Route: multiple
Dose: 0.8 mg/kg, 4 times / day
Sources:
unhealthy
Seizures Disc. AE
0.8 mg/kg 4 times / day multiple, intravenous
Recommended
Dose: 0.8 mg/kg, 4 times / day
Route: intravenous
Route: multiple
Dose: 0.8 mg/kg, 4 times / day
Sources:
unhealthy
Venoocclusive disease Disc. AE
0.8 mg/kg 4 times / day multiple, intravenous
Recommended
Dose: 0.8 mg/kg, 4 times / day
Route: intravenous
Route: multiple
Dose: 0.8 mg/kg, 4 times / day
Sources:
unhealthy
Myelosuppression severe
Disc. AE
0.8 mg/kg 4 times / day multiple, intravenous
Recommended
Dose: 0.8 mg/kg, 4 times / day
Route: intravenous
Route: multiple
Dose: 0.8 mg/kg, 4 times / day
Sources:
unhealthy
Overview

OverviewOther

Drug as perpetrator​

Drug as perpetrator​

TargetModalityActivityMetaboliteClinical evidence
no [IC50 >10 uM]
no [IC50 >10 uM]
no [IC50 >10 uM]
no [IC50 >10 uM]
no [IC50 >10 uM]
no [IC50 >10 uM]
no [IC50 >1000 uM]
no [IC50 >133 uM]
no [IC50 >133 uM]
no [IC50 >133 uM]
no [IC50 >812 uM]
yes
yes
Drug as victimTox targets
PubMed

PubMed

TitleDatePubMed
Standardized, unrelated donor cord blood transplantation in adults with hematologic malignancies.
2001-10-15
Considerations in the selection of an appropriate conditioning regimen for the treatment of rheumatoid arthritis by autologous peripheral blood stem cell transplantation.
2001-10
Early full donor myeloid chimerism after reduced-intensity stem cell transplantation using a combination of fludarabine and busulfan.
2001-10
Monitoring of busulfan area under the curve: estimation by a single measurement.
2001-10
New method for thyroid transplantation across major histocompatibility complex barriers using allogeneic bone marrow transplantation.
2001-09-27
Arsenic trioxide in the management of acute promyelocytic leukaemia.
2001-09-08
Sexual differentiation of germ cell deficient gonads in the medaka, Oryzias latipes.
2001-09-01
[Hematopoietic stem cell transplantation with busulfanthiotepa-cyclophosphamide conditioning for pediatric patients with high-risk acute lymphoblastic leukemia].
2001-09
Allogeneic bone marrow transplantation-mediated transfer of specific immunity against Toxocara canis associated with excessive IgE.
2001-09
Intravenous busulphan for conditioning before autologous or allogeneic human blood stem cell transplantation.
2001-09
Unexplained pulmonary hypertension in chronic myeloproliferative disorders.
2001-09
Non-myeloablative conditioning regimen of fludarabine, busulfan, anti-thymocyte globulin, and methylprednisolone for allogeneic peripheral blood hematopoietic cell transplantation.
2001-09
Neonatal bone marrow transplantation for severe combined immunodeficiency.
2001-09
Developing a pediatric outpatient transplantation program. The Children's Memorial Hospital experience.
2001-08-01
Myeloablation and autologous peripheral blood stem cell rescue results in hematologic and clinical responses in patients with myeloid metaplasia with myelofibrosis.
2001-08-01
Rhenium 188-labeled anti-CD66 (a, b, c, e) monoclonal antibody to intensify the conditioning regimen prior to stem cell transplantation for patients with high-risk acute myeloid leukemia or myelodysplastic syndrome: results of a phase I-II study.
2001-08-01
[Successful second transplant from one-locus HLA-mismatched unrelated donor for graft rejection following initial transplant from another unrelated donor in a patient with chronic myelogenous leukemia].
2001-08
Griscelli syndrome: report of the first peripheral blood stem cell transplant and the role of mutations in the RAB27A gene as an indication for BMT.
2001-08
Atypical retinal microvasculopathy after bone marrow transplantation.
2001-08
Immune reconstitution following allogeneic stem cell transplantation in recipients conditioned by low intensity vs myeloablative regimen.
2001-08
Dose-reduced conditioning and allogeneic hematopoietic stem cell transplantation from unrelated donors in 42 patients.
2001-08
The pharmacodynamic effect of busulfan in the P39 myeloid cell line in vitro.
2001-08
Sensitive and rapid quantification of busulfan in small plasma volumes by liquid chromatography-electrospray mass spectrometry.
2001-08
Changes in erythropoietin pharmacokinetics following busulfan-induced bone marrow ablation in sheep: evidence for bone marrow as a major erythropoietin elimination pathway.
2001-08
Thiotepa, busulfan, and cyclophosphamide as a preparative regimen for allogeneic transplantation for advanced myelodysplastic syndrome and acute myelogenous leukemia.
2001-08
Overview of the treatment of infant central nervous system tumors: medulloblastoma as a model.
2001-07-24
No disadvantage in outcome of using matched unrelated donors as compared with matched sibling donors for bone marrow transplantation in children with acute lymphoblastic leukemia in second remission.
2001-07-15
Costimulation blockade, busulfan, and bone marrow promote titratable macrochimerism, induce transplantation tolerance, and correct genetic hemoglobinopathies with minimal myelosuppression.
2001-07-15
Autologous peripheral blood stem cell transplantation for patients with malignancies: the Tri-Service General Hospital experience.
2001-07
Lethal adenovirus infection in a patient who had undergone nonmyeloablative stem cell transplantation.
2001-07
Evaluation of the Murex CMV DNA Hybrid Capture assay (version 2.0) for early diagnosis of cytomegalovirus infection in recipients of an allogeneic stem cell transplant.
2001-07
High-dose melphalan with autologous hematopoietic stem cell transplantation for acute myeloid leukemia: results of a retrospective analysis of the Italian Pediatric Group for Bone Marrow Transplantation.
2001-07
Successful HLA-identical bone marrow transplantation in a patient with PNP deficiency using busulfan and fludarabine for conditioning.
2001-07
Therapeutic activity of 7-[(2-trimethylsilyl)ethyl)]-20 (S)-camptothecin against central nervous system tumor-derived xenografts in athymic mice.
2001-07
Bone marrow features improve prognostic efficiency in multivariate risk classification of chronic-phase Ph(1+) chronic myelogenous leukemia: a multicenter trial.
2001-06-15
Busulfan levels are influenced by prior treatment and are associated with hepatic veno-occlusive disease and early mortality but not with delayed complications following marrow transplantation.
2001-06
A cautionary tale: how to delete mouse haemopoietic stem cells with busulphan.
2001-06
Congenital sideroblastic anaemia successfully treated using allogeneic stem cell transplantation.
2001-06
Marked reduction in the incidence of hepatic veno-occlusive disease after allogeneic hematopoietic stem cell transplantation with CD34(+) positive selection.
2001-05
High-dose melphalan with G-CSF-stimulated whole blood rescue followed by stem cell harvesting and busulphan/cyclophosphamide with autologous stem cell transplantation in multiple myeloma.
2001-05
Hydroxyurea and periodicity in myeloproliferative disease.
2001-05
Oral busulfan pharmacokinetics and engraftment in children with Hurler syndrome and other inherited metabolic storage diseases undergoing hematopoietic cell transplantation.
2001-04
Thiotepa, busulfan, cyclophosphamide (TBC) and autologous hematopoietic transplantation: an intensive regimen for the treatment of multiple myeloma.
2001-04
Allogeneic stem cell transplantation reduces disease progression compared to autologous transplantation in patients with multiple myeloma.
2001-04
Pharmacodynamics of high-dose chemotherapy.
2001-03
Cidofovir treatment of human polyomavirus-associated acute haemorrhagic cystitis.
2001-03
Systemic fusariosis after a preparative regimen including thiotepa, VP-16 and busulfan used for blood stem cell transplantation in Hodgkin's disease.
2001-01
A new method for tolerance induction: busulfan administration followed by intravenous injection of neuraminidase-treated donor bone marrow.
2001
Cytoreduction and stem cell mobilization with a regimen of paclitaxel, etoposide and cyclophosphamide followed by autologous transplantation using a preparative regimen of busulfan, etoposide and cyclophosphamide for patients with advanced lymphoma.
2001
[Hemorrhagic cystitis related to the high-dose conditioning therapy in a bone marrow recipient].
2001
Patents

Sample Use Guides

In Vivo Use Guide
Curator's Comment: Oral administration: adult dose range for remission induction is 4 to 8 mg, total dose, daily. Dosing on a weight basis is the same for both pediatric patients and adults, approximately 60 mcg/kg of body weight or 1.8 mg/m2 of body surface, daily. http://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=bf456fc7-3a79-47f7-8acc-600b5e2f0dc2
0.8 mg per kg intravenously six hours for four consecutive days for a total of 16 doses
Route of Administration: Intravenous
In Vitro Use Guide
P39 myeloid cells were incubated with busulfan in concentrations ranging from 10 to 100 microg/ml for 2, 4 or 8 h, then washed and cultured in busulfan-free medium for 72 h.
Name Type Language
BUSULFAN
EMA EPAR   EP   HSDB   INN   MART.   MI   ORANGE BOOK   USP   VANDF   WHO-DD   WHO-IP  
INN  
Official Name English
1,4-BUTANEDIYL DIMETHANESULFONATE
Preferred Name English
BUSULFANUM [WHO-IP LATIN]
Common Name English
BUSULFAN [MI]
Common Name English
BUSULFAN [JAN]
Common Name English
BUSULFAN [EMA EPAR]
Common Name English
BUSILVEX
Brand Name English
BUSULFAN [USP MONOGRAPH]
Common Name English
BUSULFAN [MART.]
Common Name English
BUSULFAN [IARC]
Common Name English
BUSULFAN [VANDF]
Common Name English
1,4-BUTANEDIOL DIMETHANESULPHONATE
Systematic Name English
TETRAMETHYLENE DI(METHANESULFONATE)
Systematic Name English
BUSULFAN [HSDB]
Common Name English
LEUCOSULFAN
Common Name English
SULPHABUTIN
Common Name English
BUSULFAN [EP IMPURITY]
Common Name English
1,4-BUTANEDIOL, DIMETHANESULFONATE
Systematic Name English
Busulfan [WHO-DD]
Common Name English
1,4-Butanediol dimethanesulfonate
Systematic Name English
BUSULPHAN
Systematic Name English
BUSULFEX
Brand Name English
BUSULFAN FRESENIUS KABI
Brand Name English
NSC-750
Code English
NCI-C01592
Code English
BUSULFAN [ORANGE BOOK]
Common Name English
busulfan [INN]
Common Name English
MYELOSANUM [WHO-IP]
Common Name English
1,4-BUTANEDIOL, DIMETHANESULPHONATE
Systematic Name English
BUSULFAN [EP MONOGRAPH]
Common Name English
BUSULFAN [WHO-IP]
Common Name English
Classification Tree Code System Code
EMA ASSESSMENT REPORTS BUSULFAN FRESENIUS KABI (AUTHORIZED: HEMATOPOIETIC STEM CELL TRANSPLANTATION)
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
FDA ORPHAN DRUG 140700
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
FDA ORPHAN DRUG 394513
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
NDF-RT N0000000236
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
FDA ORPHAN DRUG 160402
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
FDA ORPHAN DRUG 81694
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
LIVERTOX NBK548886
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
FDA ORPHAN DRUG 161602
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
NDF-RT N0000175558
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
EU-Orphan Drug EU/3/00/011
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
WHO-VATC QL01AB01
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
FDA ORPHAN DRUG 105897
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
EMA ASSESSMENT REPORTS BUSILVEX (AUTHORIZED: HEMATOPOIETIC STEM CELL TRANSPLANTATION)
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
FDA ORPHAN DRUG 83094
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
WHO-ATC L01AB01
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
IARC Busulfan
NCI_THESAURUS C222
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
Code System Code Type Description
MESH
D002066
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PRIMARY
EVMPD
SUB05993MIG
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
PRIMARY
ALANWOOD
busulfan
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
PRIMARY
WIKIPEDIA
BUSULFAN
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
PRIMARY
RXCUI
1828
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
PRIMARY RxNorm
DRUG BANK
DB01008
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
PRIMARY
WHO INTERNATIONAL PHARMACOPEIA
BUSULFAN
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
PRIMARY Description: A white, crystalline powder. Solubility: Very slightly soluble in water; sparingly soluble in acetone R; slightly soluble in ethanol (~750 g/l) TS. Category: Cytotoxic drug. Storage: Busulfan should be kept in a well-closed container, protected from light. Additional information: CAUTION: Busulfan must be handled with care, avoiding contact with the skin and inhalation of airborne particles. Definition: Busulfan contains not less than 98.5% and not more than 100.5% of C6H14O6S2, calculated with reference to the dried substance.
CAS
55-98-1
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PRIMARY
SMS_ID
100000085245
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PRIMARY
IUPHAR
7136
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PRIMARY
DRUG CENTRAL
438
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PRIMARY
PUBCHEM
2478
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
PRIMARY
NCI_THESAURUS
C321
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
PRIMARY
HSDB
7605
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PRIMARY
FDA UNII
G1LN9045DK
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
PRIMARY
LACTMED
Busulfan
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
PRIMARY
DAILYMED
G1LN9045DK
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
PRIMARY
CHEBI
28901
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
PRIMARY
MERCK INDEX
m2778
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
PRIMARY Merck Index
NSC
750
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
PRIMARY
ECHA (EC/EINECS)
200-250-2
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
PRIMARY
INN
564
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
PRIMARY
EPA CompTox
DTXSID3020910
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
PRIMARY
ChEMBL
CHEMBL820
Created by admin on Mon Mar 31 17:50:41 GMT 2025 , Edited by admin on Mon Mar 31 17:50:41 GMT 2025
PRIMARY