骨髓增生异常综合征最新研究进展：第54届美国血液学会年会报道

Report on recent progress of myeiodysplastic syndrome in the 54th ASH annual meeting

骨髓增生异常综合征（MDS）是一组起源于造血干／祖细胞水平损伤而产生的获得性克隆性疾病，以无效造血和易转化为急性白血病为特点。第54届美国血液学会（ASH）报道了大量MDS发病相关分子机制（如ASXL1、Didol突变等）以及其与患者预后之间的关系的一系列报道。治疗方面，分子机制的进展使临床新药不断出现，但造血干细胞移植仍是目前MDS的唯一根治方法，大会也报道了造血干细胞移植的最新进展。

The myelodysplastic syndromes （MDS） are a group of clonal disorders of the hematopoietic stem/progenitor cells characterized by the presence of ineffective hematopoiesis and an increased risk of transformation to acute myeloid leukemia （AML）. In the 54th ASH annual meeting, lots of new discoveries about the molecular mechanisms （such as mutations in ASXL1 and Didol） behind MDS and the relationship between the mechanisms and the clinical outcomes were introduced. On therapy, novel agents based on the molecular mechanisms occured extending survival. Haematopoietic stem cell transplantation （HSCT） remains the only curative therapy, there re also many developments in HSCT reported in the meeting.