Barton James C
Southern Iron Disorders Center, Birmingham, AL 35209, USA.
IDrugs. 2007 Jul;10(7):480-90.
Deferitrin (GT-56-252) is the first drug in a class of desferrithiocin-derived hexadentate iron chelators. Genzyme Corp is developing this compound as an oral drug for the treatment of severe iron overload in people who require repeated erythrocyte transfusion for management of chronic anemia such as beta-thalassemia major. In phase I clinical trials in adults with beta-thalassemia, deferitrin promoted iron excretion in a dose-related manner and was well tolerated as both a liquid and capsule in fed and fasted states. There were no serious adverse events or significant laboratory abnormalities. The author concludes that deferitrin may be useful as chelation monotherapy or as part of combination or doublet chelation therapy for the treatment of severe iron overload in patients with beta-thalassemia major if its favorable pharmacokinetic profile, efficacy, safety and tolerability are confirmed in more extensive clinical trials. A phase I/II clinical trial that began in September 2003 has reportedly completed recruitment.
去铁铁蛋白（GT - 56 - 252）是去铁硫菌素衍生的六齿铁螯合剂类中的第一种药物。健赞公司正在将这种化合物开发成一种口服药物，用于治疗因慢性贫血（如重型β地中海贫血）需要反复进行红细胞输血的人群的严重铁过载。在针对成人β地中海贫血的I期临床试验中，去铁铁蛋白以剂量相关的方式促进铁排泄，并且在进食和空腹状态下作为液体和胶囊服用时耐受性良好。没有严重不良事件或显著的实验室异常。作者得出结论，如果去铁铁蛋白良好的药代动力学特征、疗效、安全性和耐受性在更广泛的临床试验中得到证实，那么它可能作为螯合单药疗法或作为联合或双联螯合疗法的一部分，用于治疗重型β地中海贫血患者的严重铁过载。据报道，一项于2003年9月开始的I/II期临床试验已完成招募。
