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 b-thalassemia major.

Methods: This cross-sectional study included 40 patients (age range 9 to 38 years) with thalassemia major receiving deferiprone for ≥5 years. Serum ferritin, and myocardial iron concentration (MIC) and liver iron concentration (LIC) assessed by T2*MRI were recorded.

Results: The patients were receiving deferiprone for a mean (SD) duration of 12.1 (4.7) years. The median (IQR) dose of deferiprone was 85 (74.3, 95) mg/kg/day. The MIC 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 severe elevation in 2 (5%), 4 (10%), 11 (27.5%) and 23 (57.5%) patients.

Conclusions: The majority of 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.