文章：

低危骨髓增生异常肿瘤的分子机制与治疗进展：临床综述

Molecular Insights and Therapeutic Advances in Low-Risk Myelodysplastic Neoplasms: A Clinical Review

原文发布日期：9 November 2025

DOI: 10.3390/cancers17223610

类型: Article

开放获取: 是

英文摘要：

Myelodysplastic neoplasms (MDS) are characterized by remarkable heterogeneity in clinical manifestations, posing significant management challenges arising due to genetic plasticity. While the Revised International Prognostic Scoring System (IPSS-R) has traditionally stratified MDS into higher-risk (HR) and lower-risk (LR) categories, the recently developed Molecular International Prognostic Scoring System (IPSS-M) integrates molecular signatures and has further enhanced prognostic stratification. In LR-MDS, current therapeutic interventions remain non-curative and the goal of treatment is centered along three critical axes: reducing transfusion dependence, improving quality of life, and reducing the risk of progression to acute myeloid leukemia (AML). This review examines recent progress made in the therapeutic landscape of LR-MDS, with particular emphasis on the molecular basis of these novel agents that may have disease-modifying potential. We evaluate the clinical trials and targeted agents in the pipeline for treating LR-MDS, providing a comprehensive perspective where these treatment modalities are placed in the current standard of care and how these novel targets can shape future therapeutic innovations.

摘要翻译： 

骨髓增生异常肿瘤（MDS）在临床表现上具有显著的异质性，其遗传可塑性给临床管理带来了重大挑战。尽管修订版国际预后评分系统（IPSS-R）传统上将MDS分为高危（HR）和低危（LR）两类，但新近开发的分子国际预后评分系统（IPSS-M）整合了分子标志物，进一步提升了预后分层能力。在低危MDS的治疗中，现有干预手段仍无法实现根治，治疗目标主要围绕三个关键方向：降低输血依赖、改善生活质量以及减少向急性髓系白血病（AML）进展的风险。本综述系统梳理了低危MDS治疗领域的最新进展，特别关注具有疾病修饰潜力的新型药物的分子作用机制。通过评估正在研发中的临床试验及靶向药物，本文全面阐述了这些治疗模式在当前标准治疗体系中的定位，并探讨这些新靶点如何引领未来治疗创新的发展方向。

原文链接：

Molecular Insights and Therapeutic Advances in Low-Risk Myelodysplastic Neoplasms: A Clinical Review