骨髓增生異常綜合征是一組影響骨髓造血干細(xì)胞、祖細(xì)胞的克隆性疾病。近年來，隨著對(duì)骨髓增生異常綜合征發(fā)病機(jī)制的深入研究產(chǎn)生了許多新型藥物。目前對(duì)骨髓增生異常綜合征治療的藥物包括缺氧誘導(dǎo)因子脯氨酰羥化酶抑制劑、端粒酶抑制劑、剪切體抑制劑、異檸檬酸脫氫酶抑制劑、新型去甲基化劑、免疫檢測(cè)點(diǎn)抑制劑、針對(duì)TP53突變藥物、酪氨酸激酶抑制劑、抗CD47的單克隆抗體。對(duì)這些藥物的作用機(jī)制、臨床使用情況進(jìn)行了總結(jié)，為患者治療藥物的選擇提供參考。

Myelodysplastic syndromes are a group of clonal diseases that affect bone marrow hematopoietic stem cells and progenitor cells. In recent years, with the in-depth research on the pathogenesis of myelodysplastic syndrome, many new drugs have been produced. At present, drugs for treatment of myelodysplastic syndromes include hypoxia-inducible factor prolyl hydroxylase inhibitors, telomerase inhibitors, spliceosome inhibitors, isocitrate dehydrogenase inhibitors, new demethylation drugs, immune checkpoint inhibitors, targeting TP53 mutant drugs, tyrosine kinase inhibitors, and anti-CD47 monoclonal antibodies. This article summarizes the mechanism of action and clinical use of these drugs, in order to provide a reference for the selection of therapeutic drugs for patients.

R977