Combination therapy - Deferasirox and deferoxamine - In thalassemia major patients in emerging countries with limited resources

Abstract

Background: The problem of iron-overload observed in thalassemia patients can be overcome using chelating agents such as deferiprone (Ferroprox®), deferasirox (Exjade®) and deferoxamine (Desferal®). Although these drugs can be used as monotherapy, combined therapy, especially deferiprone with deferoxamine, has led to promising outcomes in various studies. Methods and Materials: In this quasi-experimental study, serum ferritin levels were evaluated in 32 β-thalassemia major patients with severe iron overload before and after receiving combined deferasirox (30-40 mgkg-1day-1) and deferoxamine (40-50 mgkg-1day-1) 2 days a week. This study was conducted from September 2012 to September 2013 in Southern Iran. Results: The mean of serum ferritin levels significantly reduced from 4031±1955 to 2416±1653 ngmL-1 after 12 months of therapy (P<0·001). Echocardiograph findings showed significant improvement 1year after end of the study (P<0·001). No drug toxicity was observed by monitoring serum creatinine, liver enzymes and blood urea nitrogen (BUN) during the study period. We observed no correlation between mean serum ferritin change and age (P=0·87). In addition, the mean serum ferritin change did not differ between male and female thalassemia patients (P=0·454). No difference in mean serum ferritin change was observed between patients who had undergone splenectomy compared to those who had not done so (P=0·307). Conclusion: The study suggests that combination chelating therapy with deferasirox and deferoxamine can effectively reduce iron burden in β-thalassemia major patients with heavy iron overload without any significant complications. © 2015 British Blood Transfusion Society.