铁螯合剂在地中海贫血治疗中的研究进展

机体铁过载常见于某些遗传性疾病的患者,如地中海贫血。人体铁含量过高可对各脏器功能产生严重影响,并可催化发生氧化还原反应释放羟自由基,破坏多种生物大分子,因此临床对地中海贫血的治疗都需辅以除铁治疗。铁螯合剂可与体内铁离子有效结合并促进铁排泄,降低体内铁含量及其在各器官的病理性沉积。目前临床使用的铁螯合药物有去铁胺、去铁酮和地拉罗司,且均存在局限性。因此,半个世纪以来医药工作者一直致力于寻找口服吸收好、毒副作用小的新药,而HBED,DFT,PIH类化合物有望成为具有临床价值的新铁螯合剂。

Progress of research on the treatment of thalassemia with iron chelators

Iron overload is commonly found in patients with some genetic diseases,such as thalassemia.Iron concentration over the normal level will cause severe interference on organs’ function.And redox reaction induced by iron would release hydroxyl radical,which causes oxidative injury to several biological macromolecules.So iron chelator therapy should be involved in the treatment of thalassemia.Iron chelators can capture iron ion effectively and improve iron excretion at the same time.So they can decrease the iron level in body and protect the tissues against pathological iron deposition.There are three agents available in clinical now,namely desferrioxamine,deferiprone and deferasirox respectively,but each of them has shortages in some aspects.Researchers kept on finding a new kind of iron chelators that would provide better oral bioavailability and minor adverse effects during the last half century.And HBED,DFT,and PIH analogues are expected to be potential clinical iron chelators.