骨髓增生异常综合征（MDS）是一组恶性克隆性血液疾病，具有异质性临床病程和转化为急性髓系白血病的风险。遗传和表观遗传失调，包括 microRNA (miRNA) 表达的改变，在影响疾病发生和进展的 MDS 发病机制中发挥着关键作用。 miRNA 以其在基因表达中的调节作用而闻名，已成为各种恶性疾病中有前途的生物标志物。本综述旨在探讨 miRNA 在 MDS 中的诊断和预后作用。我们讨论旨在了解 miRNA 在 MDS 管理中的临床效用的研究工作。 MiRNA 失调与 MDS 中的特定染色体异常有关，有助于深入了解该疾病的分子状况。血浆中的循环 miRNA 为诊断和预后评估提供了一种侵入性较小的途径，在 MDS 患者中发现了独特的 miRNA 谱。此外，我们还讨论了有关 miRNA 作为低甲基化和免疫调节剂治疗反应标记物作用的研究，这可能会改善治疗决策和监测。尽管取得了重大进展，但仍需要对更大的患者群体进行进一步研究，以充分阐明 miRNA 在 MDS 发病机制中的作用，并完善个性化的患者护理方法。版权所有 © 2024 Micheva 和 Atanasova。

Myelodysplastic syndromes (MDS) are a group of malignant clonal hematological disorders with heterogeneous clinical course and risk of transformation to acute myeloid leukemia. Genetic and epigenetic dysregulation, including alterations in microRNA (miRNA) expression, plays a pivotal role in MDS pathogenesis influencing disease development and progression. MiRNAs, known for their regulatory roles in gene expression, have emerged as promising biomarkers in various malignant diseases. This review aims to explore the diagnostic and prognostic roles of miRNAs in MDS. We discuss research efforts aimed at understanding the clinical utility of miRNAs in MDS management. MiRNA dysregulation is linked to specific chromosomal abnormalities in MDS, providing insights into the molecular landscape of the disease. Circulating miRNAs in plasma offer a less invasive avenue for diagnostic and prognostic assessment, with distinct miRNA profiles identified in MDS patients. Additionally, we discuss investigations concerning the role of miRNAs as markers for treatment response to hypomethylating and immunomodulating agents, which could lead to improved treatment decision-making and monitoring. Despite significant progress, further research in larger patient cohorts is needed to fully elucidate the role of miRNAs in MDS pathogenesis and refine personalized approaches to patient care.Copyright © 2024 Micheva and Atanasova.