Section of Hematology, Department of Medicine, Yale University, Yale Cancer Center, New Haven, CT, USA.
Department of Medicine, Roswell Park Cancer Institute, Buffalo, NY, USA.
Blood Rev. 2018 Sep;32(5):368-377. doi: 10.1016/j.blre.2018.03.002. Epub 2018 Mar 8.
Myelodysplastic syndromes (MDS) are a heterogeneous group of hemopathies that exhibit physical manifestations with clinical consequences of bone marrow failure and inherent risk of progression to acute myeloid leukemia. Iron overload (IO) is common in MDS due to chronic transfusion support and disease-related alterations in iron metabolism. IO has been conclusively associated with inferior outcomes among MDS patients. Despite lack of randomized trials showing a survival impact of iron chelation therapy (ICT), ICT is recommended by experts and guidelines for select MDS patients with IO and is often used. The availability of effective oral ICT agents has reignited the controversy regarding ICT use in patients with MDS and IO. Here we summarize the studies evaluating the value of ICT in MDS and suggest a practical approach for use of these therapies. We also highlight controversies regarding use of ICT in MDS and discuss some ongoing efforts to answer these questions.
骨髓增生异常综合征(MDS)是一组异质性血液疾病,表现为骨髓衰竭的临床表现和临床后果,并有向急性髓系白血病进展的固有风险。由于慢性输血支持和疾病相关的铁代谢改变,MDS 患者常发生铁过载(IO)。IO 与 MDS 患者的不良预后密切相关。尽管缺乏随机试验表明铁螯合治疗(ICT)对生存有影响,但专家和指南仍建议对 IO 且适合的 MDS 患者进行 ICT,并且该疗法经常被使用。有效口服 ICT 药物的出现再次引发了关于 MDS 和 IO 患者使用 ICT 的争议。在此,我们总结了评估 ICT 在 MDS 中的价值的研究,并提出了这些疗法的实用应用方法。我们还强调了在 MDS 中使用 ICT 的争议,并讨论了一些正在努力回答这些问题的方法。