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Chlorambucil (marketed under the brand name Leukeran) is a chemotherapy medicine used to treat cancer by interfering with the growth of cancer cells.


Chlorambucil is approved by the Food and Drug Administration (FDA) to treat chronic lymphocytic leukemia and malignant lymphomas. It has also been less commonly used for other types of cancer including breast cancer , ovarian cancer , and choriocarcinoma. Chlorambucil is not used with the intent to cure the cancer but to improve symptoms of the disease.


Chlorambucil is a member of the group of chemotherapy drugs known as alkylating agents. Alkylating agents interfere with the genetic material (deoxyribonucleic acid, or DNA) inside the cancer cells and prevent them from further dividing and growing more cancer cells. Chlorambucil is a tablet that is taken orally.

Recommended dosage

Chlorambucil can be taken according to several different dosing schedules, depending on the disease to be treated. Chlorambucil is a 2mg oral tablet, and patients may need to take more than one tablet at a time depending on the dose. The dose is based on a patient's weight in kilograms. Patients with leukemia take chlorambucil daily for three to six weeks at a dose of 0.1 to 0.2 mg/kg/day (milligram per kilogram of body weight per day).


Patients who have received a full course of radiation therapy or chemotherapy generally should not receive chlorambucil until four weeks after the radiation or chemotherapy has been completed. Heath care providers should be notified if patients have had any previous allergic reactions to chemotherapy treatment. Patients should also increase the amount of fluids that they drink while on this medicine.

Blood counts will be monitored regularly while on chlorambucil therapy. During a certain time period after receiving chlorambucil there may be an increased risk of getting infections. Caution should be taken to avoid unnecessary exposure to infectious agents. Patients should check with their doctors before receiving live virus vaccines while on chemotherapy.

Patients who may be pregnant or trying to become pregnant should tell their doctor before receiving chlorambucil. Men and women undergoing chemotherapy are at risk of becoming sterile.

Side effects

The most common side effect from taking chlorambucil is myelosuppression , a suppression of bone marrow activity resulting in a low blood cell count. Myelo-suppression is usually the goal when treating leukemia with chlorambucil. When the white blood cell count is lower than normal (leukopenia), patients are at an increased risk of developing a fever and infections.

The platelet count can also be decreased due to chlorambucil administration. Platelets are blood cells normally found in large numbers that aid in clot formation. When the platelet count is low, patients are at an increased risk for bruising and bleeding. If the platelet count remains too low, a platelet blood transfusion is an option for treatment. More rarely, chlorambucil causes a condition called anemia in which the number of circulating red blood cells drops, resulting in dizziness and/or fatigue . Erythropoietin is a drug that can be used to increase the red blood cell count.

Less common side effects from chlorambucil include nausea, vomiting, loss of appetite, mouth sores, skin rashes, and diarrhea . Antiemetics may be given to patients before taking chlorambucil to help prevent or reduce nausea and vomiting . Liver problems may occur due to chlorambucil administration, but they are typically mild and resolve when the drug is stopped.

Damage to nerves and nervous system tissues is uncommon with chlorambucil therapy. Some reports do exist of nerve damage that has resulted in seizures, muscle twitching, muscle shaking, confusion, visual hallucinations, irritability, and loss of muscle control. Other rare reactions to chlorambucil include hair loss (alopecia ), itching , fever, lung problems, eye problems, tingling of the hands and feet, cystitis (bladder infection), and the development of another type of cancer or leukemia.


There are no significant drug interactions associated with taking chlorambucil.

Nancy J. Beaulieu, RPh., BCOP



A reduction in the normal number of red blood cells in the blood.


Specific drugs that are used to treat cancer.


An irritation of the bladder lining.


Deoxyribonucleic acid; genetic material inside of cells.

Food and Drug Administration

A government agency that oversees public safety in relation to drugs and medical devices. The FDA gives the approval to pharmaceutical companies for commercial marketing of their products.


Entering the body through a vein.


A reduction in the normal number of white blood cells in the blood.


Cancer that has spread to one or more parts of the body.

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chlorambucil (klor-am-bew-sil) n. an alkylating agent used in chemotherapy, mainly in the treatment of chronic leukaemias and lymphomas. It is given by mouth; prolonged large doses may cause damage to the bone marrow. Trade name:. Leukeran.