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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]

Control haemoglobinopathy blood safety project Keywords: sickle cell disease; thalassaemia; vaccines; iron overload; hepatitis EN sickle cell disease thalassaemia vaccines iron overload hepatitis e160 e164 5 11/30/21 20211201 NES 211201 Non-concordance with preventive recommendations is common among patients with chronic conditions, including sickle cell disease (SCD) and transfusion-dependent thalassaemia (TDT).1,2 Iron overload is common among chronically transfused patients with SCD and TDT leading to increased morbidity, mortality and cost of care, especially as they live longer.3-5 The Centers for Disease Control and Prevention (CDC) Advisory Committee on Immunization Practices (ACIP) recommendations support completion of hepatitis A (HepA) and hepatitis B (HepB) vaccinations, including SCD and TDT.6,7 Cardiac iron overload is the leading cause of mortality in TDT,8 while it is rare in SCD.9 Guidelines recommend liver iron overload assessment every 1-2 years for patients with SCD receiving chronic transfusions.10,11 The recommendation for TDT is annual assessment of liver and cardiac iron concentration.3,8 However, there are limited data on concordance with these recommendations. Child/adolescent patients with TDT who were concordant with HepA vaccination recommendations were concordant with the other two preventive service recommendations (Fig 1B). [Extracted from the article]