用luspatercept治疗耐火性或复发性骨髓质瘤:一个多中心的中国研究
Zhuxin Zhang1, Qinglin Hu1, Xudong Tang2
1Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Science, Beijing, China.
Annals of hematology
|September 8, 2023
概括
卢斯帕特塞普特在治疗复发/耐药低风险骨髓质瘤 (MDS-SF3B1) 的中国患者中显示出有效性. 该药物改善了血液学参数,并在很大一部分患者中实现了输血独立性,具有良好的安全性.
科学领域:
- 血液学 血液学 血液学
- 在瘤学瘤学.
- 临床药理学 临床药理学
背景情况:
- 对于复发性/耐药性骨髓质瘤 (MDS) 存在有限的有效疗法.
- 在中国患有低风险MDS (LR-MDS),特别是患有SF3B1突变 (MDS-SF3B1) 的MDS的患者中,关于卢斯帕特塞普特疗效的现实数据很少.
- 这项研究回顾性地分析了中国的一组MDS-SF3B1患者的luspatercept治疗结果.
研究的目的:
- 评估luspatercept在中国患者中复发/耐药MDS-SF3B1.1.1.的疗效和安全性.
- 为了确定可能从luspatercept治疗中获益更多的患者子组.
主要方法:
- 对23名被诊断患有MDS-SF3B的患者进行了回顾性分析1.1.
- 患者接受了luspatercept治疗,平均持续时间为22.9周.
- 通过血液学改善-红细胞 (HI-E) 反应和红细胞输血独立性 (RBC-TI) 评估有效性;通过不良事件评估安全性.
主要成果:
- 在12周后,60.9%的患者实现了HI-E反应,57.1%的输血依赖患者实现了≥8周的RBC-TI.
- 在治疗后观察到血红蛋白度显著增加 (P < 0.001).
- 受试者显示血清费里丁的减少更大 (P = 0.021). 基线血清EPO<500 IU/L的患者和非输血或低输血负担 (LTB) 亚组中的患者表现出更高的HI-E率 (P = 0.024对于非输血/LTB).
结论:
- 卢斯帕特塞普特在中国患者中有效且耐受性良好,耐火性LR-MDS-SF3B1.1.
- 不依赖输血或在基线时输血负担较低的患者可能会获得更大的益处.
- 现实世界的数据支持luspatercept在管理这种患者群体中的作用.
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