Azacytidine is a medication employed in the treatment of certain cancers, specifically classified as a hypomethylating agent. This drug influences cellular processes by impacting gene activity, which can help regulate cell growth and differentiation. Its use aims to restore more typical cellular functions in the context of cancer.
How Azacytidine Works
Azacytidine functions as a DNA methyltransferase inhibitor, interfering with DNA methylation, a natural cellular process. DNA methylation involves adding methyl groups to DNA, which turns genes “on” or “off,” controlling their activity. In cancer cells, certain genes, such as tumor suppressor genes, can become abnormally silenced through excessive methylation, preventing their protective roles.
Azacytidine incorporates into DNA after cellular uptake and phosphorylation. This incorporation leads to the trapping and degradation of DNA methyltransferase enzymes, effectively reducing their ability to add methyl groups to DNA. As a result, previously silenced genes, including tumor suppressor genes, can be re-expressed, restoring normal cell growth and differentiation. It also promotes the programmed death of abnormal cells (apoptosis).
Medical Conditions Treated
Azacytidine is primarily prescribed for specific blood cancers, particularly Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML). MDS are conditions where the bone marrow fails to produce sufficient healthy blood cells. Azacytidine is a standard treatment option across various types of MDS, including those classified as higher-risk forms.
For AML, azacytidine is often used in older patients or those unable to tolerate intensive chemotherapy regimens. It helps control the rapid proliferation of abnormal white blood cells characteristic of AML. The medication is also considered when a bone marrow transplant, which is often the only curative option for these conditions, is not a suitable choice or has not been successful. Azacytidine is also under investigation for treating other hematologic malignancies.
Receiving Treatment and Managing Side Effects
Azacytidine can be administered in a healthcare setting through either subcutaneous injection or intravenous infusion. The typical treatment schedule often involves daily administration for seven days, followed by a break period, and these cycles are then repeated, usually monthly. This treatment is commonly performed on an outpatient basis.
Patients undergoing azacytidine treatment may experience various side effects, categorized as blood-related, gastrointestinal, or general. Blood-related side effects, known as myelosuppression, include low blood counts such as anemia (low red blood cells), neutropenia (low white blood cells, increasing infection risk), and thrombocytopenia (low platelets, increasing bleeding risk). Regular blood tests are performed to monitor these counts, and patients should report any signs of fatigue, infection, or unusual bleeding to their healthcare team.
Gastrointestinal side effects include nausea, vomiting, diarrhea, and constipation. General side effects include fatigue, fever, and injection site reactions like redness, pain, or bruising, especially with subcutaneous administration. Less common but more serious side effects involve liver or kidney problems, closely monitored by the healthcare team. Side effects are managed with supportive care, such as anti-nausea medications, growth factors for blood counts, or antibiotics for infections. Patients should communicate any side effects to their healthcare providers, as these are often manageable and can improve over time.