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一名骨髓增生异常综合征患者使用地拉罗司实现输血独立,停药后该状态持续了两年。

A case of transfusion independence in a patient with myelodysplastic syndrome using deferasirox, sustained for two years after stopping therapy.

作者信息

Sanford D, Hsia C C

机构信息

Department of Medicine, Division of Hematology, London Health Sciences Centre, University of Western Ontario, London, ON.

出版信息

Curr Oncol. 2015 Apr;22(2):e128-32. doi: 10.3747/co.22.2100.

Abstract

Patients with myelodysplastic syndrome (mds) experience clinical complications related to progressive marrow failure and have an increased risk of developing acute myeloid leukemia. Frequent red blood cell transfusion can lead to clinical iron overload and is associated with decreased survival in mds patients. Iron chelation therapy reduces markers of iron overload and prevents end-organ damage. Here, we present the case of a patient with low-risk mds with transfusional iron overload. He was treated for 2 years with an oral iron chelator, deferasirox, and after 12 months of treatment, he experienced a hemoglobin increase of more than 50 g/L, becoming transfusion-independent. He has remained transfusion-independent, with a normal hemoglobin level, for more than 2 years since stopping chelation therapy. Hematologic and erythroid responses have previously been reported in mds patients treated with iron chelation. The durability of our patient's response suggests that iron chelation might alter the natural history of mds in some patients.

摘要

骨髓增生异常综合征(MDS)患者会经历与进行性骨髓衰竭相关的临床并发症,并且发生急性髓系白血病的风险增加。频繁输注红细胞可导致临床铁过载,并与MDS患者生存率降低相关。铁螯合疗法可降低铁过载标志物并预防终末器官损伤。在此,我们报告一例低危MDS伴输血性铁过载患者的病例。他接受口服铁螯合剂地拉罗司治疗2年,治疗12个月后,血红蛋白升高超过50 g/L,不再依赖输血。自停止螯合治疗以来,他已超过2年保持不依赖输血且血红蛋白水平正常。此前已有报道铁螯合治疗的MDS患者出现血液学和红系反应。我们患者反应的持久性表明,铁螯合可能会改变部分患者MDS的自然病程。

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本文引用的文献

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Leuk Res. 2012 Dec;36(12):1463-9. doi: 10.1016/j.leukres.2012.08.005. Epub 2012 Sep 12.
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Epidemiology of myelodysplastic syndromes.骨髓增生异常综合征的流行病学。
Am J Med. 2012 Jul;125(7 Suppl):S2-5. doi: 10.1016/j.amjmed.2012.04.014.

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