Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children

Inclusion Criteria: 1. Male or female aged ≥ 6 months to \< 10 years 2. Confirmed diagnosis of beta-thalassemia, as determined by high performance liquid chromatography (HPLC) or DNA testing 3. Started on a red blood cell (RBC) transfusion regimen, with a minimum of 2 transfusions already completed 4. Screening level of serum ferritin (SF) greater than \>200 μg/L but not more than 600 μg/L. Since SF level may be impacted by the presence of infection, it must additionally be verified that the child has had no signs of infection in the previous 7 days, including the day of screening, and that the level of C-reactive protein (CRP) is no greater than 20% higher than the normal range for the patient's age. If there are signs of infection and/or the CRP level is above this threshold, the SF level must be checked again a minimum of one week later. Exclusion Criteria: 1. Prior use of iron chelation 2. Diagnosis of hepatitis B or C, or HIV infection 3. Evidence of abnormal liver or kidney function at screening: serum alanine transaminase (ALT) level \> 5 times upper limit of normal or creatinine levels \>2 times upper limit of normal 4. Disorders associated with neutropenia (absolute neutrophil count \< 1.5 x 10\^9/L) prior to the initiation of study medication 5. A serious, unstable illness, as judged by the investigator, during the previous 3 months before screening/baseline visit including but not limited to hepatic, renal, gastro-enterologic, respiratory, cardiovascular, endocrinologic, neurologic or immunologic disease. 6. Presence of any medical condition which in the opinion of the investigator would cause participation in the study to be unwise.