Myelodysplastic syndromes (MDS) are a heterogeneous group of myeloid disorders characterized by cytopenias and increased risk of acute leukemia transformation. Prognosis of MDS patients can be assessed by various scoring systems, the most common being the International Prognostic Scoring System (IPSS) now refined by the revised version (IPSS-R). Genomic information at baseline, that is currently not included in clinical prognostic scores, will, in the future, help us to stratify patients with various prognoses.

Therapy of MDS is based on risk stratification. The aim of therapy in low-risk MDS is to improve anemia or thrombocytopenia, decrease transfusion needs, improve quality of life, attempt to prolong overall survival, and reduce the risk of progression. In higher-risk MDS, the goal of therapy is to prolong survival and reduce the risk of transformation into acute leukemia. Only a few drugs are currently available for treatment, but more drugs are now under clinical investigation, in line with new, recently discovered molecular and immunological pathways. This review describes potential new drugs for low and high-risk MDS. The increasing knowledge of immunological and signalling pathways in MDS will assist us in identifying targeted patient-oriented treatments. In the near future, initial molecular stratification will lead the way to a personalized approach and targeted therapy.

骨髓增生异常综合征 (MDS) 是一组异质性骨髓疾病，其特征是血细胞减少和急性白血病转化风险增加。MDS 患者的预后可以通过各种评分系统进行评估，最常见的是国际预后评分系统 (IPSS)，现在由修订版 (IPSS-R) 完善。基线时的基因组信息（目前未包含在临床预后评分中）将在未来帮助我们对具有各种预后的患者进行分层。

MDS 的治疗基于风险分层。低危 MDS 的治疗目标是改善贫血或血小板减少症，减少输血需求，提高生活质量，尝试延长总生存期，并降低进展风险。在高危 MDS 中，治疗的目标是延长生存期并降低转化为急性白血病的风险。目前只有少数药物可用于治疗，但更多的药物现在正在临床研究中，符合新的、最近发现的分子和免疫学途径。本综述描述了用于治疗低风险和高风险 MDS 的潜在新药。对 MDS 中免疫学和信号通路的日益了解将帮助我们确定以患者为导向的靶向治疗。在不远的将来，