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Azacitidine is used for treatment of high risk myelodysplastic syndromes (disorders of the bone marrow that results in poorly formed or dysfunctional blood cells), chronic myelomonocytic leukemia (cancer in blood and bone marrow) and acute myeloid leukemia (cancer of white blood cells characterized by production of immature white blood cells).

How it works

Azacitidine is cytotoxic drug and belongs to a class of medications called antimetabolites. It is toxic to cancer cells and interferes with the cell’s DNA synthesis, thus slowing down or stopping its growth and multiplication (cytotoxic effect) of cancer cell.

Common side effects

Pneumonia, Abdominal pain, Anemia, Blood in urine, Cellulitis, Chest pain, Cold sores, Increased blood pressure, Decreased blood pressure, Dehydration, Fever, Gastrointestinal bleeding, Bleeding gums, Mouth ulcer, Lip ulcer, Nose bleed, Reduced blood platelets, Skin infection


Expert advice

• You will be treated with drugs to prevent nausea and vomiting at the start of azacitidine treatment cycle. 
• You will be monitored with lab test to measure complete blood count before every treatment cycle to check development of anemia (low red blood count), neutropenia (abnormally low white blood cell count) or thrombocytopenia (abnormally low count of platelets in the blood).
• Take extreme care to avoid pregnancy while on azacitidine therapy. 
• Inform your doctor if you have a history of congestive heart failure, unstable heart or lung disease.
• Seek immediate medical attention if you experience difficulty in breathing, swelling of the lips, itching or rash.
• Patients allergic to azacitidine or any of its ingredients should not be administered this.
• Patients with advanced malignant liver cancer should not be given azacitidine.
• Pregnant and breastfeeding women should also not be given azacitidine.
• Children and adolescents below 18 years should not be given azacitidine.

Frequently asked questions


Q. Is azacitidine a vesicant? 
Azacitidine is not a vesicant and is not known to cause severe local tissue damage upon extravasation (leaking of injected Azacitidine into the surrounding tissues from the site of administration) when injected.
Q. Does azacitidine cause hair loss?
No, azacitidine is not known to cause hair loss
Q. Is azacitidine chemotherapy?
Yes. Azacitidine is chemotherapy and a cytotoxic drug used in the treatment of various cancers. It is toxic to cancer cells and interferes with the cell’s DNA synthesis, thus slowing down or stopping its growth and multiplication (cytotoxic effect).

Q. Is Azacitidine a cytotoxic drug?
Yes. Azacitidine is  a cytotoxic drug.

Content on this page was last updated on 18 October, 2016, by Dr. Varun Gupta (MD Pharmacology)