How it is administered

Azacitidine is given as an injection. It can be administered either under the skin (subcutaneous injection) or directly into a vein (intravenous infusion). The medication comes as a sterile powder that is mixed with sterile water before use.

The typical starting dose for adults is 75 mg/m² once daily for 7 days in a row, repeated every 4 weeks. Your healthcare team will decide the exact dose and schedule based on your individual needs and response to treatment. Azacitidine is not interchangeable with oral azacitidine, and the dosing regimens are different.

How it works

Azacitidine is a type of medication called a nucleoside metabolic inhibitor. It is a pyrimidine nucleoside analog of cytidine. Azacitidine works by two main mechanisms:

1. Hypomethylation of DNA: In some blood cancers, certain genes that help control cell growth and development are turned off by a process called methylation. Azacitidine can reduce this methylation (hypomethylation), potentially turning these important genes back on and helping blood cells to develop more normally.

2. Direct cytotoxicity: Azacitidine can also directly kill abnormal cells in the bone marrow by interfering with their ability to make DNA and RNA, which are essential for cell growth and division. This is especially effective against rapidly dividing cancer cells, such as those seen in blood cancers like Acute Myeloid Leukemia (AML) and Myelodysplastic Syndromes (MDS).

By targeting both the abnormal growth and the underlying genetic changes, azacitidine helps to control the disease and improve blood cell counts.

Who Should take it

Azacitidine is approved for adults with certain types of myelodysplastic syndromes (MDS), which are a group of blood disorders that can progress to acute myeloid leukemia (AML). These include:

• Refractory anemia (RA)
• Refractory anemia with ringed sideroblasts (RARS) (if accompanied by low white blood cells, low platelets, or requiring transfusions)
• Refractory anemia with excess blasts (RAEB)
• Refractory anemia with excess blasts in transformation (RAEB-T)
• Chronic myelomonocytic leukemia (CMMoL)

Azacitidine may also be used in some cases for patients with AML, especially if they are not able to tolerate intensive chemotherapy. Your doctor will determine if azacitidine is appropriate for your specific type of blood cancer and overall health.

Who should not take it

Azacitidine should not be used in the following situations:

• If you have advanced malignant liver tumors (advanced malignant hepatic tumors)
• If you are allergic to azacitidine or any of its ingredients, including mannitol

Caution is also needed if you have severe pre-existing liver or kidney problems, as these conditions can increase the risk of side effects. Azacitidine can cause harm to an unborn baby, so it should not be used during pregnancy unless absolutely necessary. Women of childbearing age and men with partners who could become pregnant should use effective contraception during treatment and for a period after the last dose.

Commonly used with

Azacitidine is often used as a single agent for MDS and AML, but it can also be part of a supportive care regimen that may include:

• Blood transfusions (for anemia or low platelets)
• Antibiotics (to treat or prevent infections)
• Medications to prevent nausea and vomiting

It is not typically combined with other chemotherapy drugs for MDS or AML, but your doctor may recommend additional supportive treatments as needed.

Commonly tested with

In clinical studies, azacitidine has been compared to best supportive care and to other treatments such as low-dose cytarabine or intensive chemotherapy (cytarabine with anthracyclines) in patients with MDS and AML. It is sometimes studied in combination with other supportive medications, but is most commonly tested as a single agent in these settings.