骨髓增生异常综合征预后评分系统分析及低风险患者治疗研究进展
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国家自然科学基金资助项目(82370171)


Research progress on risk stratification and treatment of low-risk patients with myelodysplastic syndrome
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    摘要:

    骨髓增生异常综合征(MDS)是一种恶性骨髓造血肿瘤,其特征是形态学发育不良、无效的克隆性贫血,导致外周血细胞减少,并伴有转化为急性髓系白血病的风险。针对MDS的诊断和预测系统一直在共同发展,两者现已都整合了分子标记物。基于预后评估系统的风险分层,经历了国际预后评分系统(IPSS)、世界卫生组织预后评分系统(WPSS)、修订版的国际预后评分系统(IPSS-R)到现在纳入基因突变的分子国际预后评分系统(IPSS-M)等的发生发展,使MDS有低风险和高风险之分,进一步改善了风险分层,提高了MDS预后评估的准确性。对MDS的治疗,基于患者的风险分层,低风险和高风险的MDS患者治疗存在差别,对于低风险的MDS(LR-MDS)患者,治疗目的是改善贫血等血细胞减少,使患者脱离输血依赖、提高生活质量等,除了红细胞生成刺激剂 (ESA)、来那度胺、免疫抑制剂等治疗方法,目前针对LR-MDS的多项新的治疗方法正在研究中

    Abstract:

    Myelodysplastic syndrome (MDS) is a malignant hematopoietic neoplasm characterized by morphological dysplasia, clonal anemia with ineffective erythropoiesis, leading to peripheral blood cell reduction, and with a risk of transforming into acute myeloid leukemia. Diagnostic and prognostic systems for MDS have been jointly developed, and both have now incorporated molecular markers. The evolution from the International Prognostic Scoring System (IPSS), the World Health Organization (WHO) Prognostic Scoring System (WPSS), the revised International Prognostic Scoring System (IPSS-R), to the Molecular International Prognostic Scoring System (IPSS-M), which incorporates gene mutations, has resulted in the distinction between low-risk and high-risk MDS, further improving risk stratification and increasing the accuracy of MDS prognostic assessment. The treatment of MDS is based on the patient's risk stratification, with different treatments for low-risk and high-risk MDS patients. For low-risk MDS(LR-MDS) patients, the treatment goal is to improve anemia and other hematopoietic deficiencies, allowing patients to discontinue blood transfusion dependence and improve quality of life, in addition to Erythropoiesis stimulating agents (ESA), lenalidomide, and immunosuppressive agents. Currently, several new treatments for low-risk myelodysplastic syndrome are being studied

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  • 在线发布日期: 2025-07-21
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