The safety and acceptability of twice‐daily deferiprone for transfusional iron overload: A multicentre, open‐label, phase 2 study.

Given that appropriate long-term iron chelation is essential in the treatment of transfusion-dependent anaemias,14,15 deferiprone b.i.d. has the potential to improve treatment adherence and health outcomes in patients with transfusional iron overload. Twenty-seven patients had a primary diagnosis of thalassaemia major, one patient had -thalassaemia/haemoglobin H disease and one patient had sickle -thalassaemia. Fourteen patients reported mild AEs, 13 patients reported moderate AEs and one patient reported a severe AE. Lifelong iron chelation therapy is critical for patients with transfusion-dependent anaemias, and full adherence to therapy is essential to optimise long-term patient outcomes.1,2 Deferiprone is an oral iron chelator with high efficiency in binding and removing excess intracellular and extracellular iron.3,4 Owing to its elimination half-life of approximately 2 h, deferiprone is administered three times daily (t.i.d.) to promote longer extent of exposure and better control of labile iron.5-7 Clinical trial data show adherence rates with deferiprone t.i.d. from 79% to 98%8,9; however, real-world adherence is generally lower than in clinical trials, as the t.i.d. regimen may be inconvenient and the midday dose may often be missed.10-12 The United States Food and Drug Administration recently approved a twice-a-day modified-release formulation of deferiprone (Ferriprox TAD 1000 mg tablet; manufactured by Apotex Inc., Toronto, Ontario, Canada) for the treatment of patients with transfusional iron overload due to thalassaemia syndromes, sickle cell disease or other anaemias.13 In single-dose and multiple-dose pharmacokinetic studies of deferiprone in healthy volunteers (Tables S1 and S2), we found that the twice-daily (b.i.d.) formulation had equivalent 24-h drug exposure to the original immediate-release tablet administered t.i.d. (Figure S1), and exposure was not affected by administration with food (Table S3). [Extracted from the article]