Advances in Iron Chelation Therapy: Towards A New Oral Formulation

Background: Patients with thalassemia major require regular monthly blood transfusion and excess iron from multiple blood transfusion deposits in different organs of the body, causing different organ damages. Cardiac iron overload is the main cause of death in patients with thalassemia major.Method: The aim of this study was to determine the effectiveness of Deferasirox (Nanojade) produced in Iran for the first time as a medicine in reducing iron overload of chronic blood transfusion in patients with Beta-thalassemia major. This is a pre-post quasi-experimental study that was performed on patients with thalassemia major involved in lifelong regular monthly blood transfusions. In this study, based on the existing protocols, injectable and dissolving iron chelating regimes were changed to a new generation of deferasirox oral tablets. To determine the effect of Nanojade, the mean level of the last three serum ferritin levels, before changing chelation, was compared with the serum ferritin levels after starting Nanojade. In the course of treatment, the possible side effects of Nanojade in various organs were monitored regularly.Results: The mean age of the patients was 16 years (ranging between 3 and 39 years). None of the patients had renal failure, elevated creatinine, or proteinuria with Nanojade. The most important side effect was elevated liver enzyme levels. The mean starting dose of Nanojade was 19.86± 5.78 mg/kg and the mean of the last dose was 23.41 ± 5.49 mg/kg. There was a statistically significant difference between the amount of ferritin at baseline with the serum ferritin of 6 and 18 months after chelation with Nanojade (P = 0.001).Conclusion: chelation with Nano Jade had less renal and hepatic side effects; and all of the patients had good compliance and significant reduction in the serum ferritin level in comparison to the results of other studies regarding the similar foreign deferasirox products