内科理论与实践

内科理论与实践 ›› 2025, Vol. 20 ›› Issue (06): 482-486.doi: 10.16138/j.1673-6087.2025.06.09

• 研究报道 • 上一篇    下一篇

罗特西普治疗骨髓增生异常综合征伴环形铁粒幼细胞难治性贫血患者的疗效及安全性分析

宋陆茜1,*, 张玉梅2,*, 张征1, 苏基滢1, 赵佑山1, 吴东1, 贺琪1, 吴凌云1,2, 常春康1,*()   

  1. 1.上海交通大学医学院附属第六人民医院血液科，上海　200233
    2.上海市第八人民医院血液科，上海　200235
  • 收稿日期:2025-02-12 出版日期:2025-12-30 发布日期:2026-01-30
  • 通讯作者: 常春康　E-mail：changchunkang@sjtu.edu.cn
  • 作者简介:*宋陆茜与张玉梅为共同第一作者

Efficacy and safety analysis of luspatercept in patients with myelodysplastic syndromes with ringsideroblasts and refractory anemia

SONG Luxi1,*, ZHANG Yumei2,*, ZHANG Zheng1, SU Jiying1, ZHAO Youshan1, WU Dong1, HE Qi1, WU Lingyun1,2, CHANG Chunkang1,*()   

  1. 1. Department of Hematology, Shanghai Sixth People’s Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai 200233, China
    2. Department of Hematology, Shanghai Eighth People's Hospital, Shanghai 200235, China
  • Received:2025-02-12 Online:2025-12-30 Published:2026-01-30

RichHTML

4

PDF (PC)

323

摘要：

目的：观察新型促红细胞成熟剂罗特西普治疗骨髓增生异常综合征伴环形铁粒幼细胞（myelodysplastic syndrome with ring sideroblast, MDS-RS）难治性贫血患者的临床疗效及安全性。方法：纳入2022年8月至2023年12月上海交通大学医学院附属第六人民医院血液科连续收治接受罗特西普治疗的MDS-RS贫血患者，根据WHO2016标准诊断为MDS-RS，并符合国际预后评分系统修订版（revised International Prognostic Scoring System, IPSS-R）预后分组的非常低危、低危或中等风险组。罗特西普按照说明书方案给药，并限定治疗前后维持一致的输血策略。收集患者每个治疗周期的血液学数据和红细胞输血次数，并根据修订后的国际工作组（International Working Group, IWG）2018年标准评估血液学改善（hematopoietic improvement, HI）。结果：共纳入9例患者，达到HI者7例，其中5例获得红系HI（hematopoietic improvement-erythroid, HI-E），2例获得红细胞输注不依赖（red blood cell transfusion independence, TI-RBC）≥8周。HI的中位缓解持续时间为17（9~46）周。7例HI患者中5例在用药过程中失效，中位首次失效时间为15（9~23）周。研究期间受新型冠状病毒疫情影响，5例患者共发生6例次感染。6例共发生11例次延迟用药，均导致血红蛋白减少[平均下降（18±6）g/L]或需红细胞输注，直接触发5次剂量爬坡和2例次疗效丧失。3例报告骨痛，1例骨髓纤维化进展至3级。结论：本研究证实罗特西普在真实世界中对MDS-RS难治性贫血患者有效，但基线输血负担及延迟用药影响疗效，其疗效依赖于连续和定期给药。

关键词: 骨髓增生异常综合征伴环形铁粒幼细胞, 罗特西普, 输血依赖

Abstract:

Objective To observe the clinical efficacy and safety of luspatercept in managing refractory anemia among patients with myelodysplastic syndrome with ring sideroblast (MDS-RS). Methods A retrospective analysis was performed on consecutive MDS-RS patients with refractory anemia who were treated with luspatercept at Department of Hematology, Shanghai Sixth People’s Hospital, Shanghai Jiao Tong University School of Medicine between August 2022 and December 2023. Eligible patients met the 2016 WHO diagnostic criteria for MDS-RS and were in the very low-, low-, or intermediate- risk groups according to the revised International Prognostic Scoring System (IPSS-R). Luspatercept was administered per label instructions, and a consistent transfusion strategy was required for each patient before and during treatment. Hematologic data and red blood cell transfusion episodes were recorded for each treatment cycle, and hematologic improvement (HI) was assessed using the revised International Working Group (IWG) 2018 criteria. Results 9 patients were enrolled. 7 patients achieved HI, among whom 5 achieved HI-erythroid (HI-E) and 2 achieved red blood cell transfusion independence (TI-RBC) for ≥8 weeks. The median duration of HI response was 17 (9−46) weeks. 5 of the 7 responding patients experienced loss of response, with a median time to first loss of 15 (9−23) weeks. During the study, impacted by the COVID-19 pandemic, 5 patients experienced 6 infection episodes. Treatment delays occurred 11 times in 6 patients, and all delays led to a decrease in hemoglobin [mean decrease, (18±6) g/L] or the need for red blood cell transfusions, directly resulting in 5 dose escalation and loss of response in 2 cases. 3 patients reported bone pain. 1 patient showed progression to grade 3 marrow fibrosis. 33.3% (3/9) reported bone pain, and 1 case showed progression to grade 3 marrow fibrosis. Conclusions This study confirms the real-world efficacy of luspatercept in patients with MDS-RS and refractory anemia. However, its efficacy is influenced by baseline transfusion burden and treatment delays, and depended on continuous and regular administration.

Key words: Myelodysplastic syndrome with ring sideroblast, Luspatercept, Transfusion dependence

中图分类号: