Significance Of Combo Chelation Therapy Over Mono Chelation Therapy In Patients With Thalassemia

Thalassaemia or "thalassemia" is an inherited autosomal recessive blood disease. In thalassemia, the genetic defect results in reduced rate of synthesis of one of the globin chains that make up hemoglobin. Reduced synthesis of one of the globin chains can cause the formation of abnormal hemoglobin molecules, thus causing anemia, the characteristic presenting symptom of the thalassemias.Alpha, beta and delta are different types of thalassemia dependong upon pathophysiology. Both alpha and beta thalassemia include the following two forms:Thalassemia major and Thalassemia minor. Children born with thalessemia major (Cooley's anemia) are normal at birth, but develop severe anemia during the first year of life.Other symptoms can include:bone deformities in the face, fatigue, growth failure, shortness of breath and yellow skin (jaundice). Persons with the minor form of alpha and beta thalassemia have small red blood cells (which are identified by looking at their red blood cells under a microscope), but no symptoms. Treatment for thalassemia major often involves regular blood transfusions and folate supplements. Persons who receive significant numbers of blood transfusions need a treatment called chelation therapy to remove excess iron from the body. Bone marrow transplant may help treat the disease in some patients, especially children. Two main subtypes thalassemia major (TM) and thalassemia intermedia (TI). TI has a later clinical onset with a milder anemia that does not require transfusions at least during the first few years of life. Nowadays, well-treated TM patients with regular transfusion-chelation therapy showed suppression of the anemiarelated disorders in parallel to prolongation of life [1] Thalassemia intermedia encompass a wide clinical spectrum of beta-thalassemia phenotypes. Some thalassemia intermedia patients are asymptomatic until adult life, whereas others are symptomatic from as young as 2 years of age. A number of clinical complications commonly associated with thalassemia intermedia are rarely seen in thalassemia major, including extramedullary hematopoiesis, leg ulcers, gallstones and thrombophilia. Prevention of these complications, possibly with blood transfusion therapy, is ideal since they may be difficult to manage. Currently, many patients with thalassemia intermedia receive only occasional or no transfusions . Blood product transfusions are a valuable health-care resource. Guidelines for transfusion exist, but variability in their application, particularly in children, remains. The risk factors that threaten transfusion safety are well established, but because their occurrence in children is rare, single-institution studies have limited utility in determining the rates of occurrence. Red blood cells and platelets were the two most frequently transfused products [3] Blood transfusion in thalassemia is critical for the survival of patients. The hypertransfusion therapy has significantly increased the life expectancy and quality of life in such patients. However, this treatment has also increased the frequency of complications due to iron overload. Despite recent treatment with Desferal in beta-thalassemia major, the risk of secondary endocrine dysfunction remains high, with hypogonadism being one of the most frequent endocrine complications [4] Previous studies have shown that the iron overload that results from such therapy in other patient populations is associated with significant morbidity and mortality. In patients Mehreen Babar et al /J. Pharm. Sci. & Res. Vol.2 (12), 2010,837-843