Therapeutic Approaches: Drugs Acting on Thalassemia

Abstract

Thalassemia is a genetic blood disorder manifested by abnormal hemoglobin production, resulting in the reduction of red blood cell formation. Hydroxyurea Ian anti metabolite drug for treating Thalassemia. It performs by enhancing the production of fetal hemoglobin, which can compensate for deficient adult hemoglobin in individuals along with Thalassemia. An enhanced level of fetal hemoglobin can enhance overall red cell formation and decreasr complications related to Thalassemia. Excessive iron accumulation is a common complication in thalassemia patients who undergo frequent blood transfusions. Deferasirox is an iron chelator that binds to excess iron especially in the body. Permitting to be excreted by stopping iron overload, Deferasirox helps in mitigating the risk of organ damage, especially to the heart and liver, which can happen over time especially in thalassemia patients. Luspatercept is a new therapeutic option especially for individuals with transfusion_ dependent beta_ thalassemia. It performs by targeting a particular pathways participated in erythropoiesis, the process pf red blood cell production. By modulating this pathway. Luspatercept activates the production of mature red blood cells. Decreasing the need especially for frequent blood transfusions in some patients. An important emerging treatments for thalassemia include gene therapy approaches aimed at addressing generic mutations. Finally it is concluded that the management of thalassemia involves a multidisciplinary approach with drugs playing a critical role especially in alleviating symptoms and enhancing the overall _ well being of patients