5azacitidin

5-azacytidine, also known as azacitidine, is a pyrimidine nucleoside analog of cytidine. It is a hypomethylating agent used in the treatment of myelodysplastic syndromes (MDS) and certain types of leukemia. 5-azacytidine works by incorporating into DNA and RNA, leading to the inhibition of DNA methyltransferases. This inhibition disrupts the process of DNA methylation, which can reactivate tumor suppressor genes that have been silenced by aberrant methylation in cancer cells.

The drug is typically administered via subcutaneous injection or intravenous infusion. Its primary indication is for patients with intermediate or high-risk MDS. It has also shown efficacy in treating acute myeloid leukemia (AML) in patients who are not candidates for intensive chemotherapy. The effectiveness of 5-azacytidine is often monitored through blood counts and bone marrow examinations.

Common side effects associated with 5-azacytidine treatment include fatigue, nausea, vomiting, diarrhea, constipation, and fever. Hematological side effects such as neutropenia, thrombocytopenia, and anemia are also frequently observed due to the drug's impact on bone marrow function. Close monitoring of blood cell counts is essential during treatment. 5-azacytidine is a vital therapeutic option for certain hematological malignancies, offering a mechanism to reawaken silenced genes and potentially control disease progression.