Uygun Vedat, Kurtoglu Erdal
Antalya Egitim ve Arastirma Hastanesi Çocuk Hematoloji Bolumu, 07100 Antalya, Turkey.
Hematology. 2013 Jan;18(1):50-5. doi: 10.1179/1607845412Y.0000000046. Epub 2012 Nov 19.
In thalassemia major (TM), without iron chelation therapy, iron-mediated free radical damage causes liver, endocrine, and myocardial toxicities. Deferoxamine has universally been the standard therapeutic option for iron chelation therapy; however, its usage is troublesome, leading to suboptimal patient compliance. In order to maximize the effectiveness of iron chelation therapy, oral iron chelators deferiprone and deferasirox constitute an important development, offering a potential to improve compliance. Although both oral drugs are effective, they have differences including different pharmacokinetics and side-effect profiles. Our retrospective evaluation of TM patients using oral chelators showed that oral chelators are effective in reducing iron overload regarding ferritin level and partially in cardiac T2* value. However, in our study side effects and discontinuation rates were unexpectedly high and close follow-up of TM patients using oral chelators should be carefully done. The variability in rate of side effects and drug discontinuation in spelenectomized patients using oral chelators suggests that spleen may have a role in pharmacokinetics of these drugs, as well.
在重型地中海贫血（TM）中，若不进行铁螯合治疗，铁介导的自由基损伤会导致肝脏、内分泌及心肌毒性。去铁胺一直是铁螯合治疗的标准选择；然而，其使用较为麻烦，导致患者依从性欠佳。为使铁螯合治疗效果最大化，口服铁螯合剂去铁酮和地拉罗司是一项重要进展，有望提高依从性。尽管这两种口服药物均有效，但它们存在差异，包括不同的药代动力学和副作用谱。我们对使用口服螯合剂的TM患者进行的回顾性评估表明，口服螯合剂在降低铁蛋白水平的铁过载方面有效，且在一定程度上对心脏T2*值也有效。然而，在我们的研究中，副作用和停药率意外地高，因此对使用口服螯合剂的TM患者应仔细进行密切随访。使用口服螯合剂的脾切除患者的副作用发生率和药物停药率的差异表明，脾脏可能在这些药物的药代动力学中也起作用。
