铁过载概述及口服祛铁新药地拉罗司

Overview of iron overload and the new iron chelator deferasirox

规则输血是维持重度慢性贫血患者生命的重要治疗手段,患者长期依赖输血治疗不可避免地引起体内铁沉积增加。输血相关性铁过载可导致多脏器的损害,特别是沉积在肝脏或心脏,甚至可危及生命。作为传统的铁螯合剂,去铁酮和去铁胺因其不良反应或治疗依从性差等问题无法满足临床治疗需要。地拉罗司是一种新型的口服铁螯合剂,多个II期或III期试验证实其在输血依赖性患者中可获得与去铁胺相似的疗效。近期前瞻性、多中心EPIC研究也证实了其祛铁疗效,且有助于改善地中海贫血患者的心脏铁沉积。本文就铁过载的临床特征、危害性以及祛铁新药地拉罗司对比传统药物的优势做一综述。

For many patients with severe chronic anemia,regular blood transfusion is the important lifesaving therapy available.Long-term blood transfusions will unavoidably and invariably produce accumulation of iron（iron overload）,and thereby induced iron toxicity.Transfusional hemosiderosis,particularly in the liver or heart,can cause considerable morbidity that may be fatal eventually.Traditionally,iron chelating agents include deferiprone and deferoxamine,but they are not the satisfied treatment choice due to their side effects or non-convenience for administration.Deferasirox,a rationally-designed oral iron chelator,is validated as defined by several phase II trials.Moreover,a pivotal phase III trial revealed that its efficacy is similar to that of deferoxamine in transfusion-dependent patients.Recently reported results from the large,prospective,multicenter EPIC study confirmed its efficacy,and in this study deferasirox was also found to be capable of removing iron from the heart in patients with β-thalassemia and myocardial hemosiderosis.This review summarized the morbidity,mortality,and clinical features of iron overload,and introduced the advantages of the new chelator deferasirox over traditional agents.