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Systematic (IUPAC) name
4-[bis(2-chlorethyl)amino]benzenebutanoic acid
CAS number 305-03-3
ATC code L01AA02
PubChem 2708
DrugBank APRD00115
Chemical data
Formula C14H19Cl2NO2 
Mol. mass 304.212 g/mol
Pharmacokinetic data
Bioavailability  ?
Metabolism Hepatic
Half life 1.5 hours
Excretion N/A
Therapeutic considerations
Pregnancy cat.


Legal status

Prescription only

Routes Oral

Chlorambucil (marketed as Leukeran by GlaxoSmithKline) is a chemotherapy drug that has been mainly used in the treatment of chronic lymphocytic leukemia. It is a nitrogen mustard alkylating agent and can be given orally.

In the past, it has been used for treating some types of non-Hodgkin lymphoma, Waldenström macroglobulinemia, polycythemia vera, trophoblastic neoplasms, ovarian carcinoma. It also has been used as an immunosuppressive drug for various autoimmune and inflammatory conditions, e.g. nephrotic syndrome. Its current use is mainly for CLL as it is well tolerated by most patients, though this has been primarily replaced by fludarabine.[1]

Side Effects

The IARC has found strong evidence that chlorambucil is itself a cancer agent, and listed it as such in its list 1.

Myelosuppression (anemia, neutropenia, thrombocytopenia), or bone marrow suppression is the most commonly occurring side effect of chlorambucil. Withdrawn from the drug, this side effect may be reversible, but bone marrow failure can occur in rare cases.

Less commonly occurring side effects include:

  • Gastrointestinal Distress (nausea, vomiting, diarrhea, and oral ulcerations).
  • Central Nervous System: Seizures, tremors, muscular twitching, confusion, agitation, ataxia, and hallucinations.
  • Skin reactions
  • Hepatotoxicity
  • Infertility


  1. ^ Rai KR, Peterson BL, Appelbaum FR, Kolitz J, Elias L, Shepherd L, Hines J, Threatte GA, Larson RA, Cheson BD, Schiffer CA (2000). "Fludarabine compared with chlorambucil as primary therapy for chronic lymphocytic leukemia.". N Engl J Med 343 (24): 1750-7. PMID 11114313.

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