EFFICACY AND SAFETY OF DEFERASIROX WHEN COMPARED TO D EFERIPRONE AS ORAL IRON CHELATING AGENT : A RANDOMIZED CONTROL TRIAL

BACKGROUND: Thalassemia is one of the most common inherited hemoglobinopathy seen in southern India.With regular blood transfusion, these children live longer but associated morbidity due to iron overload impairs the quality of life.We studied the efficacy and safety of new oral iron chelator, deferasirox, compared with deferiprone which was used for long time.MATERIAL AND METHODS: We conducted a prospective randomised control study, between January 2011 to June 2012 at thalassemia day care centre of Indira Gandhi Institute of Child Health, Bengaluru.The children who were diagnosed as Thalassemia and receiving regular blood transfusion with serum ferritin levels more than 1000ng/ml and not receiving any chelation therapy were included in the study.These children were randomly divided into two groups as group 1 and group 2 by computer generated randomization.The children included in group 1 received Deferasirox and group 2 received Deferiprone as chelation therapy.The dosage of deferasirox was 20mg/kg/day once daily and that of deferiprone 75 mg/kg/day in three divided daily doses.The primary study outcome was to measure and compare the decrease in serum ferritin levels between the two study groups.The secondary outcome measures were to compare the side effect profiles among the two groups.RESULTS: We included 41 thalessemic children and 19 of them were included in group 1 (Deferasirox) and 22 children in Group 2 (Deferiprone).At the end of study period of 18 months three children in group II discontinued therapy due to side effects, hence the remaining 19 were available for final analysis in group 2 whereas no drop outs in the group 1.During the study period, the serum ferritin decreased from 3261±2613ng/dl to 1586±766 ng/dl in group 1 as compared in group 2 from 4109±3153ng/dl to 1743±1138ng/dl (fig 2).This was also not statistically significant.In group 2, 68% of the children experienced adverse effect as compared to 36.8% in group 1 children.In group 2 children 9 had arthralgia out of which 3 children were discontinued their treatment as compared to one child in group who had arthralgia but none discontinued the treatment.CONCLUSION: Both deferasirox and deferiprone are equally efficacious in reducing the serum ferritin levels in thalassemic children receiving chronic blood transfusion therapy.However the side effects profile including severe adverse effects like arthralgia and arthritis requiring discontinuation of therapy was higher in children receiving deferiprone as compared to deferasirox.