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. Although DFP was initially approved as a thrice daily formulation, a twice daily formulation was developed to improve patients' treatment adherence. Here, we assess the safety of DFP twice daily in patients with thalassemia, SCD, or other anemias in real-world clinical practice in the United States. Data for patients referred for DFP twice daily between 1 July 2020 and 31 August 2023 were obtained from the Ferriprox Total Care Registry. The frequencies of adverse events (AEs), serious AEs (SAEs), AEs of special interest, and fatal outcomes were assessed. Of the 425 patients referred to the registry, 348 received ≥1 DFP shipment, and was designated the safety analysis group. The primary diagnosis was a thalassemia syndrome in 129, SCD in 189, and other anemia in 30 patients. Mean (standard deviation) DFP exposure was 343.0 (281.9) days. In total, 1021 AEs and 369 SAEs were reported. As seen with the thrice daily formulation, the most common AEs associated with DFP twice daily 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 cases recovered. None of the 26 fatal outcomes reported were assessed as related to DFP treatment. DFP twice daily was well tolerated in real-world clinical practice, and its safety profile was consistent with DFP thrice daily. No new safety concerns were observed.