Hepatic and Cardiac Iron-load in Children on Long-term Chelation with Deferiprone for Thalassemia Major

Objective: To evaluate the efficacy of prolonged deferiprone monotherapy in patients with ?-thalassemia major. Methods: This cross-sectional study included 40 patients (age range 9 to38 years) with thalassemia major receiving deferiprone for ?5 years. Serum ferritin, andmyocardial iron concentration (MIC) and liver iron concentration (LIC) assessed by T2*MRIwere recorded. Results: The patients were receiving deferiprone for a mean (SD) duration of12.1 (4.7) years. The median (IQR) dose of deferiprone was 85 (74.3, 95) mg/kg/day. TheMIC was normal or had a mild, moderate or severe elevation in 29 (72.5%), 3 (7.5%), 3(7.5%), and 5 (12.5%) patients. The LIC was normal or had a mild, moderate or severeelevation in 2 (5%), 4 (10%), 11 (27.5%) and 23 (57.5%) patients. Conclusions: The majorityof patients receiving deferiprone had a moderate/severe hepatic but normal cardiac iron load.Prolonged deferiprone monotherapy was suboptimal for hepatic iron load in the majority.

A concurrent parallel study to compare the efficacy and safety of oral iron chelators, defrasirox and defriprone in patients of beta thalassaemia major

Background: This study was planned to evaluate all the cases of ? thalassaemia major, already receiving one of the oral iron chelators for a comparison among the efficacy, safety and economy of deferasirox and deferiprone to establish the better option in an Indian scenario.Methods: We identified two groups of patients: 38 treated with deferasirox and 35 treated with deferiprone. Laboratory parameters such as serum ferritin, creatinine, SGPT, Hb, CBC and urine were recorded at the time of inclusion and at 1, 3 and 6 months after the inclusion. The primary outcome variable was serum Ferritin level at the start and at the end of study. Serum ferritin level was carried out by microparticle enzyme linked immunoassay.Results: Before the study, the mean hemoglobin level was 7.32±1.50mg/dL ranged from 4 to 10.8 in deferasirox group and 7.54±1.15mg/dL ranged from 5.5 to 8.8 in deferiprone group. At the time of inclusion, study population was characterized by a mean serum ferritin value of 4735.11±450.01 SE in deferasirox and 4315.97±340.75 SE in deferiprone group. After one month the mean serum ferritin increases to 4578.66±371.96 in deferasirox and 4388.82±316.16 in deferiprone group. After three month the mean serum ferritin reduces to 4295.60±377.37 in deferasirox and 3988.88±349.84 in Deferiprone group.Conclusions: Thus, we conclude that deferasirox and deferiprone are well tolerated, have few adverse effects and almost have a comparable effect in lowering of the patient's serum ferritin level. Deferiprone is more cost effective but needs a strict control on compliance owing to requirement in three divided doses per day.