文章：

低风险骨髓增生异常综合征患者的管理

Management of patients with lower-risk myelodysplastic syndromes

原文发布日期：2022-12-14

DOI: 10.1038/s41408-022-00765-8

类型: Review Article

开放获取: 是

英文摘要：

Myelodysplastic syndromes (MDS) are a heterogeneous group of hematopoietic stem cell disorders characterized by ineffective hematopoiesis with abnormal blood cell development (dysplasia) leading to cytopenias and an increased risk for progression to acute myeloid leukemia (AML). Patients with MDS can generally be classified as lower- (LR-MDS) or higher-risk (HR-MDS). As treatment goals for patients with LR-MDS and those with HR-MDS differ significantly, appropriate diagnosis, classification, and follow-up are critical for correct disease management. In this review, we focus on the diagnosis, prognosis, and treatment options, as well as the prediction of the disease course and monitoring of treatment response in patients with LR-MDS. We discuss how next-generation sequencing, increasing knowledge on mechanisms of MDS pathogenesis, and novel therapies may change the current treatment landscape in LR-MDS and why structured assessments of responses, toxicities, and patient-reported outcomes should be incorporated into routine clinical practice.
 

摘要翻译： 

骨髓增生异常综合征（MDS）是一组异质性造血干细胞疾病，其特征是无效造血伴随血细胞发育异常，导致血细胞减少并增加进展为急性髓系白血病（AML）的风险。MDS患者通常可分为低危（LR-MDS）与高危（HR-MDS）两类。由于LR-MDS与HR-MDS患者的治疗目标存在显著差异，准确的诊断、分型及随访对疾病管理至关重要。本文重点综述LR-MDS的诊断、预后评估、治疗方案选择、病程预测及治疗反应监测，并探讨下一代测序技术、对MDS发病机制认识的深化以及新型疗法如何可能改变当前LR-MDS的治疗格局，同时阐述为何应将结构化评估治疗反应、毒性及患者报告结局纳入常规临床实践。

原文链接：

Management of patients with lower-risk myelodysplastic syndromes