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Systematic (IUPAC) name
CAS number 55-98-1
ATC code L01AB01
PubChem 2478
DrugBank APRD00664
Chemical data
Formula C6H14O6S2 
Mol. mass 246.304 g/mol
Pharmacokinetic data
Bioavailability 80% (oral)
Protein binding 32.4%
Metabolism Hepatic
Half life 2.5 hours
Excretion  ?
Therapeutic considerations
Licence data


Pregnancy cat.


Legal status

Prescription only

Routes Oral, parenteral

Busulfan is a chemotherapy drug that is a cell cycle non-specific alkylating agent (slows the growth of cancer cells). More specifically it belongs to a subclass of alkylating agents known as alkyl sulfonates. It is marketed in the U.S. by GlaxoSmithKline under the brand name Myleran, and has been in clinical use since 1959. Busulfan is also available in an IV formulation marketed as Busulfex by PDL BioPharma, Inc. Its chemical designation is 1,4-Butanediol dimethanesulfonate.



Currently, its main uses are in bone marrow transplantation, especially in chronic myelogenous leukemia (CML), where it is used as a conditioning drug. Busulfan can control tumor burden but cannot prevent transformation or correct cytogenic abnormalities. Though not as common, it may also be used for Chronic Lymphocytic Leukemia (CLL).

Its mechanism of action through alkylation produces DNA-DNA and DNA-protein cross linking. The resulting covalent bonds inhibit DNA, RNA, and protein synthesis. The inhibition of DNA synthesis subsequently produces a cytotoxic effect.

The drug was recently used in a study to examine the role of platelet-transported serotonin in liver regeneration.[1]

Toxicity and side effects

Toxicity may include interstitial pulmonary fibrosis, hyperpigmentation, seizures, hepatic (veno-occlusive disease) and wasting syndrome. Phenytoin may be used concurrently to prevent the seizures.

1,4-Butanediol dimethanesulfonate is listed by the IARC as a Group 1 carcinogen.

Busulfan also induces thrombocytopenia, a condition of lowered blood platelet count and activity.


Busulfan use to be the standard of treatment for Chronic Myeloid Leukemia (CML) until it was replaced with the new gold standard, Imatinib.


  1. ^ Lesurtel M, Graf R, Aleil B, Walther D, Tian Y, Jochum W, Gachet C, Bader M, Clavien P (2006). "Platelet-derived serotonin mediates liver regeneration". Science 312 (5770): 104–7. PMID 16601191.

This article is licensed under the GNU Free Documentation License. It uses material from the Wikipedia article "Busulfan". A list of authors is available in Wikipedia.
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