Vlachaki Efthymia, Mainou Maria, Bekiari Eleni, Vetsiou Evaggelia, Tsapas Apostolos
Thalassemia Unit, Second Medical Department, Hippokratio General Hospital, Aristotle University Thessaloniki, Greece.
Hemoglobin. 2013;37(1):94-100. doi: 10.3109/03630269.2012.746696. Epub 2012 Dec 7.
Deferasirox (DFRA) is a novel oral chelator agent for treatment of iron overload. Although well established in the treatment of β-thalassemia major (β-TM), it has not yet been fully investigated in patients with sickle cell disease. The aim of this report is to present the preliminary results of a pilot study assessing the effect of 4 years of DFRA treatment in six patients with sickle cell disease who are in need of recurrent transfusions. Our results show a significant reduction of ferritin levels and improvement of liver hemosiderosis, assessed by means of magnetic resonance imaging T2* (MRI T2*). None of the patients presented any serious adverse effects and the treatment was well tolerated. These results are in accordance with previous studies about the use of DFRA in sickle cell disease.
地拉罗司（DFRA）是一种用于治疗铁过载的新型口服螯合剂。尽管它在重型β地中海贫血（β-TM）的治疗中已得到充分证实，但在镰状细胞病患者中尚未得到充分研究。本报告的目的是展示一项初步研究的结果，该研究评估了地拉罗司对6名需要反复输血的镰状细胞病患者进行4年治疗的效果。我们的结果显示，通过磁共振成像T2*（MRI T2*）评估，铁蛋白水平显著降低，肝脏含铁血黄素沉着症得到改善。没有患者出现任何严重不良反应，治疗耐受性良好。这些结果与先前关于地拉罗司在镰状细胞病中应用的研究一致。
