李瑞萍, 马艳萍. 罗特西普治疗骨髓增生异常综合征的研究进展[J]. 中国肿瘤临床, 2023, 50(18): 964-968. DOI: 10.12354/j.issn.1000-8179.2023.20230874
引用本文: 李瑞萍, 马艳萍. 罗特西普治疗骨髓增生异常综合征的研究进展[J]. 中国肿瘤临床, 2023, 50(18): 964-968. DOI: 10.12354/j.issn.1000-8179.2023.20230874
Ruiping Li, Yanping Ma. Research progress in the treatment of myelodysplastic syndromes with luspatercept[J]. CHINESE JOURNAL OF CLINICAL ONCOLOGY, 2023, 50(18): 964-968. DOI: 10.12354/j.issn.1000-8179.2023.20230874
Citation: Ruiping Li, Yanping Ma. Research progress in the treatment of myelodysplastic syndromes with luspatercept[J]. CHINESE JOURNAL OF CLINICAL ONCOLOGY, 2023, 50(18): 964-968. DOI: 10.12354/j.issn.1000-8179.2023.20230874

罗特西普治疗骨髓增生异常综合征的研究进展

Research progress in the treatment of myelodysplastic syndromes with luspatercept

  • 摘要: 骨髓增生异常综合征(myelodysplastic syndromes,MDS)是一种常见的髓系肿瘤,随着年龄增长发病率增加。MDS患者主要依据预后分组选择不同的治疗方案,不同危险度分层的患者治疗目标不同。绝大多数患者属于较低危组,并且合并贫血,改善贫血、提高生存质量是其主要治疗目标。由于反复输血带来的危害,以及红细胞生成刺激剂(erythropoiesis-stimulating agents,ESAs)、来那度胺等经典药物治疗反应的不足,需要开发更为有效、更安全的药物治疗贫血。罗特西普是一种红细胞成熟剂,已被国内外批准用于治疗β-地中海贫血。本文将对罗特西普治疗MDS的作用机制及相关临床研究进行综述,分析其疗效及安全性,为临床实践提供进一步指导。

     

    Abstract: Myelodysplastic syndromes (MDS) are a group of common myeloid neoplasms, the incidence of which increases with age. The treatment options for patients with MDS differ primarily according to the prognosis group in which they fall, and patients with different risk stratifications have different treatment goals. Most patients belong to the lower-risk group and their presentation is complicated with anemia. Therefore, improving both the anemia and quality of life is the main therapeutic goal. Owing to the harm of repeated transfusions as well as the lack of patient response to classical drugs such as erythropoiesis-stimulating agents (ESAs) and lenalidomide, the development of more effective and safer drugs for anemia is needed. Luspatercept, which is a red blood cell maturation agent, has been approved nationally and abroad for the treatment of patients with beta-thalassemia. In this article, we review the mechanism of action of luspatercept and clinical studies related to its use in the treatment of MDS as well as its efficacy and safety. Additionally, further guidance for its use in clinical practice is provided.

     

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