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骨髓增生异常综合征的铁螯合治疗:最近我们学到了什么?

Iron chelation therapy in MDS: what have we learnt recently?

机构信息

University of Ulm, Ulm, Germany.

出版信息

Blood Rev. 2009 Dec;23 Suppl 1:S21-5. doi: 10.1016/S0268-960X(09)70006-2.

Abstract

Patients with myelodysplastic syndromes (MDS) who receive chronic blood transfusions for anaemia are at risk of developing iron overload, which can negatively affect organ function and survival. Evidence suggests that iron chelation therapy can restore iron balance in these patients and may improve their chances of survival. Recently, several guidelines on the management of patients with MDS have been published that address iron overload and the use of iron chelation therapy. While these guidelines differ in some specific details, they generally agree that patients with lower-risk MDS are most likely to develop iron overload and therefore benefit from iron chelation therapy. The oral iron chelator, deferasirox, has been shown to reduce serum ferritin levels and labile plasma iron in patients with MDS, and has an acceptable safety profile. Unlike other iron chelators, deferasirox also appears to inhibit the nuclear factor kappa-light-chain-enhancer of activated B cells (NF-kappaB) pathway in MDS blast cells, which may lead to additional beneficial effects.

摘要

患有骨髓增生异常综合征(MDS)的患者因贫血而接受慢性输血,有发生铁过载的风险,这可能会对器官功能和生存产生负面影响。有证据表明,铁螯合疗法可以使这些患者恢复铁平衡,并可能提高他们的生存机会。最近,发表了几项关于 MDS 患者管理的指南,涉及铁过载和铁螯合疗法的使用。虽然这些指南在一些具体细节上有所不同,但它们普遍认为低危 MDS 患者最有可能发生铁过载,因此受益于铁螯合疗法。口服铁螯合剂地拉罗司已被证明可降低 MDS 患者的血清铁蛋白水平和不稳定血浆铁水平,且具有可接受的安全性。与其他铁螯合剂不同,地拉罗司似乎还可以抑制 MDS 原始细胞中的核因子 kappa 轻链增强子的活化 B 细胞(NF-kappaB)途径,这可能会带来额外的有益效果。

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