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美国的骨髓增生异常综合征:临床医生最新资讯

Myelodysplastic syndromes in the United States: an update for clinicians.

作者信息

Troy Jesse D, Atallah Ehab, Geyer Julia T, Saber Wael

机构信息

The EMMES Corporation , Rockville, MD , USA.

出版信息

Ann Med. 2014 Aug;46(5):283-9. doi: 10.3109/07853890.2014.898863. Epub 2014 Apr 10.

Abstract

Myelodysplastic syndromes (MDS) are heterogeneous malignant bone marrow disorders diagnosed most often in elderly white persons. MDS have significant clinical consequences, including cytopenias leading to infection, bleeding, and death; and approximately one-third of cases progress to acute myeloid leukemia (AML). Only one potentially curative therapy exists-allogeneic hematopoietic stem cell transplant (HSCT)-but this therapy is not widely used due to associated morbidity and mortality in elderly patients. Recent research suggests MDS occurs more frequently than previously thought and may be responsible for a substantial proportion of unexplained anemias in elderly persons. Incidence of MDS is expected to increase with increases in life expectancy. Therefore, we offer this comprehensive narrative update of MDS to inform the medical community treating the population at risk for MDS, with a focus on MDS epidemiology and clinical management in the United States. This review includes a brief historical background of MDS, provides an overview of the population burden of disease, discusses the molecular pathology of MDS, describes the clinical features and management of MDS, and discusses future directions in MDS research. Our objective is to inform general medicine practitioners and call attention to the need for translational research in MDS.

摘要

骨髓增生异常综合征(MDS)是一类异质性的恶性骨髓疾病,多见于老年白人。MDS具有显著的临床后果,包括血细胞减少导致感染、出血和死亡;约三分之一的病例会进展为急性髓系白血病(AML)。目前只有一种潜在的治愈性疗法——异基因造血干细胞移植(HSCT),但由于老年患者存在相关的发病率和死亡率,这种疗法并未得到广泛应用。最近的研究表明,MDS的发病率比之前认为的更高,可能是老年人群中相当一部分不明原因贫血的病因。随着预期寿命的增加,MDS的发病率预计也会上升。因此,我们提供这份关于MDS的全面叙述性更新,以告知治疗MDS高危人群的医学界,重点关注美国MDS的流行病学和临床管理。本综述包括MDS的简要历史背景,概述疾病的人群负担,讨论MDS的分子病理学,描述MDS的临床特征和管理,并讨论MDS研究的未来方向。我们的目标是为普通内科医生提供信息,并呼吁关注MDS转化研究的必要性。

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