罗特西普治疗伴有SF3B1突变的低危骨髓增生异常综合征：一项中国单中心真实世界研究
Luspatercept for the treatment of lower-risk myelodysplastic syndrome with SF3B1 mutation: a real-world single-center research in China.
作者信息
Liang Weiru, Kang Rui, Zhao Yufei, Xing Lingxiao, Zhang Baohang, Shi Yimeng, Li Yuan, Peng Guangxin, Zhao Xin, Liu Xu, Hu Jing, Hu Xiangrong, Zhou Kang, Yang Yang, Xiong Youzhen, Li Jianping, Fan Huihui, Yang Wenrui, Ye Lei, Jing Liping, Zhang Li, Zhang Fengkui
机构信息
Anemia Therapeutic Centre, State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, People's Republic of China.
出版信息
Hematology. 2025 Dec;30(1):2506858. doi: 10.1080/16078454.2025.2506858. Epub 2025 Jun 5.
BACKGROUND
Luspatercept, approved by the FDA and EMA for patients with transfusion-dependent lower-risk myelodysplastic syndrome (LR-MDS) unresponsive to erythropoiesis-stimulating agents (ESAs), lacks extensive real-world data, particularly in China.
METHODS
We retrospectively analyzed 14 LR-MDS-SF3B1 patients treated with luspatercept for ≥12 weeks.
RESULTS
Median age was 60 years (range 47-72); 42.9% were male. Before treatment, 78.6% were transfusion-dependent, and 42.9% had prior ESA therapy. At median 24-week follow-up (range 12-44), erythroid response rates were 71.43% (week 12), 75.00% (week 16), and 62.50% (week 24). Hemoglobin levels significantly improved at weeks 12 and 24 ( = 0.013, = 0.005). No grade 3-4 adverse events occurred. Hematologic improvement-erythroid (HI-E) patients exhibited higher white blood cells, neutrophils, and reticulocytes at week 12 versus non-HI-E patients. Bone marrow analysis revealed erythroid hyperplasia in HI-E patients, with higher erythrocyte percentage (56.00% vs. 34.00%, = 0.023), lower myeloid-to-erythroid ratio (0.60 vs. 1.59, = 0.024), and increased polychromatic erythroblasts (19.50% vs. 10.00%, = 0.034).
CONCLUSIONS
Luspatercept demonstrated efficacy and safety in Chinese LR-MDSSF3B1 patients. Greater erythroid hyperplasia correlated with better clinical response.
背景
罗特西普已获美国食品药品监督管理局（FDA）和欧洲药品管理局（EMA）批准，用于治疗对促红细胞生成素（ESA）无反应的输血依赖型低危骨髓增生异常综合征（LR-MDS）患者，但缺乏广泛的真实世界数据，尤其是在中国。
方法
我们回顾性分析了14例接受罗特西普治疗≥12周的LR-MDS-SF3B1患者。
结果
中位年龄为60岁（范围47-72岁）；42.9%为男性。治疗前，78.6%的患者依赖输血，42.9%的患者曾接受过ESA治疗。在中位24周随访时（范围12-44周），红系反应率在第12周为71.43%，第16周为75.00%，第24周为62.50%。血红蛋白水平在第12周和第24周显著改善（P = 0.013，P = 0.005）。未发生3-4级不良事件。血液学改善-红系（HI-E）患者在第12周时的白细胞、中性粒细胞和网织红细胞水平高于非HI-E患者。骨髓分析显示，HI-E患者红系增生，红细胞百分比更高（56.00%对34.00%，P = 0.023），髓系与红系比值更低（0.60对1.59，P = 0.024），多染性幼红细胞增加（19.50%对10.00%，P = 0.034）。
结论
罗特西普在中国LR-MDS-SF3B1患者中显示出疗效和安全性。更大程度的红系增生与更好的临床反应相关。
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