Common brand names

• Onureg
• Vidaza

How it is administered

Azacitidine is given as an intravenous (IV) infusion or as a subcutaneous injection. For children with Juvenile Myelomonocytic Leukemia (JMML), it is usually administered as an IV infusion once daily for 7 days in a 28-day cycle. The dose depends on the child’s age and weight.

For adults with other blood cancers, it may be given either as a subcutaneous injection or IV infusion, typically for 7 days in a row every 4 weeks. The medication comes as a powder that is mixed with sterile water before use.

How it works

Azacitidine is a type of chemotherapy called a nucleoside metabolic inhibitor. It works by targeting abnormal cells in the bone marrow, especially those that are dividing too quickly or have lost their normal growth controls.

Azacitidine is a pyrimidine nucleoside analog of cytidine. It is believed to work in two main ways: by causing hypomethylation of DNA and by directly killing abnormal blood cells. Hypomethylation can help restore normal function to certain genes that are important for cell growth and development. The direct cytotoxic effect means it can kill cancer cells that are dividing rapidly. In JMML, azacitidine helps reduce the number of abnormal cells in the bone marrow and blood, giving healthy cells a better chance to grow.

Non-dividing (resting) cells are less affected by azacitidine, so the medication mainly targets cancerous cells.

Who should take it

Azacitidine is approved for use in pediatric patients aged 1 month and older with newly diagnosed Juvenile Myelomonocytic Leukemia (JMML). It is also used for adults with certain types of myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMMoL).

For JMML, azacitidine is often used before a stem cell transplant to help control the disease and improve the chances of a successful transplant. Your doctor will decide if azacitidine is right for you or your child based on the diagnosis, age, and overall health.

Who should not take it

Azacitidine should not be used in patients with advanced malignant liver tumors or in anyone with a known allergy to azacitidine or mannitol (an ingredient in the medication).

Patients with severe pre-existing liver or kidney problems should use azacitidine with caution, as it may increase the risk of serious side effects. Pregnant women should not use azacitidine, as it can harm the unborn baby. If you have any history of severe allergic reactions or significant liver or kidney disease, let your healthcare provider know before starting treatment.

Commonly used with

Azacitidine is often used alone for JMML, especially before a stem cell transplant. In adult blood cancers, it may be combined with supportive treatments such as antibiotics, anti-nausea medications, and blood transfusions as needed.

It is not typically combined with other chemotherapy drugs for JMML, but your doctor may recommend other medications to help manage side effects or prevent infections.

Commonly tested with

In clinical studies for JMML, azacitidine has been tested as a single agent before stem cell transplantation. In adult blood cancers, it has been studied both alone and in combination with supportive care measures.

No formal drug interaction studies have been conducted, but azacitidine is not known to interact with common liver enzymes or drug transporters. Your healthcare provider will monitor for any potential interactions with other medications you may be taking.