Safety profile of a liquid formulation of deferiprone in young children with transfusion-induced iron overload: a 1-year experience

Background: Data on the use of deferiprone in young children with iron overload are limited. Objective: To study the safety profile of a liquid formulation of deferiprone in chelating young children with transfusion-induced iron overload. Patients and methods: A daily dose of 50-100 mg/kg BW in three divided doses of oral deferiprone was given to young patients who had received at least ten packed red cell transfusions and achieved a serum ferritin level > 1000 mu g/L during a 12-month period from 2011 to 2012. Results: Nine children (four males) diagnosed with various types of thalassaemia (n=8) and hereditary spherocytosis (n=1) were enrolled. Their mean (SD) age was 4.5 (1.9) years. The patients received 15-20 ml/kg BW of packed red cell transfusions every 4-8 weeks from a mean (SD) age of 2.1 (1.7) years to maintain a pre-transfusion haematocrit at 27%. A mean (SD) total of packed red cells of 5132 (2725) ml were given within a mean (SD) duration of 2.4 (1.1) years before the study. During the 1-year study period, they received a mean (SD) total of packed red cells of 2194 (680) ml or 138 (50) ml/kg BW with a mean (SD) daily iron load of 0.29 (0.12) mg/kg BW. The pre-treatment geometric mean of serum ferritin of 1863.8 mg/L decreased to 1279.7 mg/L after 1 year of treatment (P=0.05). All patients tolerated the liquid formulation well and did not experience any gastro-intestinal discomfort, nausea or vomiting. Conclusion: The liquid formulation of deferiprone is safe in young children with transfusion-induced iron overload.