How I treat iron overload in adult MDS.

IF 21 1区 医学 Q1 HEMATOLOGY Blood Pub Date : 2025-01-23 DOI:10.1182/blood.2023022501
Heather A Leitch, Rena Buckstein
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Abstract

Abstract: Although clinical benefits of iron chelation therapy (ICT) in red blood cell (RBC) transfusion-dependent (TD) hereditary anemias such as α-thalassemia major are incontrovertible, the evidence supporting a similar benefit in patients with TD myelodysplastic neoplasms (MDS) and iron overload (IOL) is sometimes debated. MDS presents later in life, has a limited repertoire of life-extending therapies, and patients may have comorbidities acting as competing causes of death. However, refined prognostication identifies patients with MDS with a reasonable life expectancy, and because 50% of patients will ultimately become RBC TD and develop transfusional IOL, ICT should be considered in some. Using illustrative cases, we summarize mechanisms of iron toxicity, strategies for the identification of IOL, and propose definitions of IOL severity. We provide rationale for, and recommend which patients may benefit from, ICT. We discuss currently available chelators, their administration, monitoring, side effects, and their management. Given challenges with the use of iron chelators, we suggest the nuances to be considered when planning chelation initiation to include the rate of iron accumulation, the presence of organ iron and/or dysfunction, and detectable indicators of oxidative stress. Areas for future investigation are identified.

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我是如何治疗成人骨髓增生异常综合征铁超载的?
铁螯合疗法(ICT)对红细胞输血依赖型(RBC TD)遗传性贫血(如重型β地中海贫血)的临床疗效毋庸置疑,但对于红细胞输血依赖型骨髓增生异常性肿瘤(MDS)和铁超载(IOL)患者的类似疗效,有时却存在争议。骨髓增生异常性肿瘤发病较晚,可用于延长生命的治疗手段有限,而且患者可能合并有其他致死原因。然而,经过完善的预后分析发现,MDS 患者的预期寿命尚可,而且由于 50% 的患者最终将依赖红细胞输血并发展为输血铁过载(IOL),因此部分患者应考虑接受铁螯合疗法(ICT)。我们通过举例说明,总结了铁毒性的机制、IOL 的识别策略,并提出了 IOL 严重程度的定义。我们提出了 ICT 的基本原理,并建议哪些患者可以从 ICT 中获益。我们讨论了目前可用的螯合剂、给药、监测、副作用及其管理。鉴于使用铁螯合剂所面临的挑战,我们建议在计划启动螯合治疗时应考虑的细微差别包括铁积累的速度、器官铁和/或功能障碍的存在以及氧化应激的可检测指标。我们还确定了未来的研究领域。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Blood
Blood 医学-血液学
CiteScore
23.60
自引率
3.90%
发文量
955
审稿时长
1 months
期刊介绍: Blood, the official journal of the American Society of Hematology, published online and in print, provides an international forum for the publication of original articles describing basic laboratory, translational, and clinical investigations in hematology. Primary research articles will be published under the following scientific categories: Clinical Trials and Observations; Gene Therapy; Hematopoiesis and Stem Cells; Immunobiology and Immunotherapy scope; Myeloid Neoplasia; Lymphoid Neoplasia; Phagocytes, Granulocytes and Myelopoiesis; Platelets and Thrombopoiesis; Red Cells, Iron and Erythropoiesis; Thrombosis and Hemostasis; Transfusion Medicine; Transplantation; and Vascular Biology. Papers can be listed under more than one category as appropriate.
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