Yesim Aydinok

TL;DR: A comparative phase 3 trial was conducted to demonstrate the efficacy of deferasirox in regularly transfused patients with beta-thalassemia aged 2 years or older, and found it to be a promising once-daily oral therapy for the treatment of transfusional iron overload.

TL;DR: It is shown that iron deficiency anemia refractory to oral iron therapy can be caused by germline mutations in TMPRSS6, which encodes a type II transmembrane serine protease produced by the liver that regulates the expression of the systemic iron regulatory hormone hepcidin.

TL;DR: This first prospective study of long-term deferasirox use in pediatric and adult patients with β-thalassemia suggests treatment for ≤ 5 years is generally well tolerated and effectively reduces iron burden.

TL;DR: It is shown that deferasirox is effective in removing and preventing myocardial iron accumulation and in reducing or preventing cardiac iron overload in patients with beta-thalassemia in a 1-year prospective, multicenter study.

TL;DR: CORDELIA demonstrated the noninferiority of deferasirox compared with deferoxamine for myocardial iron removal and left ventricular ejection fraction remained stable in both arms.