Azacitidine

• Azacitidine is a hypomethylating agent used to treat myelodysplastic syndromes (MDS) and certain types of acute myeloid leukemia (AML).
• Its mechanism of action involves inhibiting DNA methyltransferases, leading to gene re-expression and cellular differentiation.
• The medication helps improve blood counts and reduce the progression of these conditions.
• Common side effects include nausea, fatigue, and blood count abnormalities, requiring careful monitoring.
• Patients receiving azacitidine should be closely monitored for adverse reactions and potential complications.

What is Azacitidine?

What is Azacitidine? It is a pyrimidine nucleoside analog, classified as a hypomethylating agent. This medication is designed to treat specific hematologic malignancies by interfering with the normal function of DNA. It is administered as an injection and plays a crucial role in managing conditions where abnormal blood cell production is a concern.

Mechanism of Action

The azacitidine mechanism of action involves its incorporation into both DNA and RNA. Once incorporated into DNA, it covalently binds to and inhibits DNA methyltransferases (DNMTs). This inhibition leads to hypomethylation of DNA, which can reactivate silenced tumor suppressor genes and promote cellular differentiation and apoptosis (programmed cell death). When incorporated into RNA, azacitidine interferes with RNA metabolism, further contributing to its therapeutic effects. This dual action helps to restore normal cell growth and differentiation patterns in affected cells.

What Azacitidine Is Used For

What is azacitidine used for primarily includes the treatment of myelodysplastic syndromes (MDS) and certain types of acute myeloid leukemia (AML). For MDS, it is approved for all FAB subtypes (refractory anemia, refractory anemia with ring sideroblasts, refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, and chronic myelomonocytic leukemia). In AML, it is indicated for newly diagnosed adults who are not candidates for intensive curative therapy due to comorbidities or age. The azacitidine drug information highlights its role in improving overall survival and delaying progression to AML in patients with MDS, as well as extending survival in specific AML populations. It helps to improve blood counts and reduce the need for transfusions in many patients.

Azacitidine Side Effects and Warnings

Like all medications, azacitidine side effects and warnings are important considerations during treatment. Common side effects often include nausea, vomiting, diarrhea, constipation, fatigue, fever, and injection site reactions such as erythema, pain, or bruising. Hematologic side effects are also frequent and can be serious, including myelosuppression leading to neutropenia (low white blood cells), thrombocytopenia (low platelets), and anemia (low red blood cells). These can increase the risk of infection and bleeding, necessitating regular blood count monitoring.

More serious warnings associated with azacitidine include embryo-fetal toxicity, meaning it can cause harm to a fetus if administered during pregnancy. Patients should be advised to use effective contraception. Renal toxicity and hepatic impairment have also been reported, requiring careful monitoring of kidney and liver function, especially in patients with pre-existing conditions. Hypersensitivity reactions, though rare, can also occur. Patients should report any unusual symptoms or severe reactions to their healthcare provider promptly.