Real-world safety profile of twice a day deferiprone in patients with thalassemia, sickle cell disease, or other anemias

Iron overload is a significant cause of transfusion-related morbidity and mortality. Deferiprone (DFP) is an established oral iron chelator approved for the treatment of transfusional iron overload in patients with thalassemia, sickle cell disease (SCD), or other anemias. Initially approved as a three times-a-day (TID) formulation, a twice-a-day (BID) formulation was developed to improve patient adherence. Here, we assess the safety of DFP BID in patients with thalassemia, SCD, or other anemias in real-world clinical practice in the United States. Data for patients referred for DFP BID between July 1, 2020 to August 31, 2023 were obtained from the Ferriprox® Total Care Registry. The frequency of adverse events (AEs), serious AEs (SAEs), AEs of special interest and fatal outcomes was assessed. Of the 425 patients referred to the registry, 348 received ≥one DFP shipment and had a primary diagnosis of thalassemia syndromes (N=129), SCD (N=189), or other anemias (N=30) (safety analysis group). Mean (SD) DFP exposure was 343.0 (281.9) days. In total, 1021 AEs and 369 SAEs were reported. As seen with the TID formulation, the most common adverse events with DFP BID were predominantly gastrointestinal or related to underlying disease. Cases of neutropenia and agranulocytosis were rare, reported in 1.7% and 0.6% of patients, respectively and all recovered. None of the 26 fatal outcomes reported were assessed as related to DFP treatment. DFP BID was well-tolerated in real-world clinical practice, and the safety profile was consistent with DFP TID. No new safety concerns were observed.