GOOD NEWS FOR SICKLE CELL DISEASE AND THALASSAEMIA PATIENTS

               Iron overload is a common clinical problem, arising from disorders of increased iron absorption such as hereditary haemochromatosis or thalassaemia intermedia syndromes or as a consequence of chronic blood transfusions for various blood disorders.

               Regular red blood cell (RBC) transfusions are the principal supportive therapy for many rare anaemias involving a decrease in RBC production, an increase in cell destruction, or chronic blood loss¹. Anaemias such as beta-thalassaemia and sickle cell disease are examples of chronic diseases that require long-term transfusion therapy to improve life expectancy.

Deferoxamine (DFO) was the standard of care for transfusional iron overload for >40 years, requiring subcutaneous infusion for 8-12 h/day, 5-7 days/week. Oral iron chelators are an important development, offering the potential to improve compliance and patients' quality of life. The oral, three-times-daily agent deferiprone appeared to be a promising advance; however, its use has been limited owing to serious adverse events, such as neutropenia and agranulocytosis.
               Therapy combining deferiprone with DFO has proved effective in the management of severe cardiac siderosis. Deferasirox is a novel, orally active agent that provides 24-h chelation with a once-daily dose. An extensive clinical trial program has demonstrated that deferasirox at appropriate doses is effective in reducing or maintaining iron burden in adult and pediatric patients. The clinical program demonstrated that deferasirox has a safety profile that is clinically manageable with regular monitoring.It is an oral iron-chelating agent that reduces liver iron concentration and serum ferritin levels. Deferasirox binds iron with high affinity in a 2:1 ratio. It is approved for treatment of treat chronic iron overload due to multiple blood transfusions and nontransfusion-dependent thalassemia.