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泽布替尼治疗难治性/复发性温抗体型自身免疫性溶血性贫血的疗效和安全性

[Efficacy and safety of zanubrutinib in treating refractory/relapsed warm autoimmune hemolytic anemia].

作者信息

Shen S P, Yang C, Chen M, Han B

机构信息

Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China.

出版信息

Zhonghua Xue Ye Xue Za Zhi. 2025 Apr 14;46(4):355-358. doi: 10.3760/cma.j.cn121090-20241110-00442.

Abstract

This retrospective analysis included 10 patients with relapsed/refractory warm autoimmune hemolytic anemia (wAIHA) who received zanubrutinib (treatment course: 3-6 months) and completed at least 3 months of follow-up at Peking Union Medical College Hospital from July 2022 to January 2024. The cohort included two male and eight female patients with a median age of 63 years (range: 36-76), who had received a median of 9 months (range: 3-22) of previous therapies. At 1-month, 3-month, and final follow-up (median 7 months), the overall response (OR) rates were 30%, 60%, and 60%, with corresponding complete response (CR) rates of 20%, 40%, and 40%, respectively. The median time to achieve a response was 2 months (range: 1-2) among the responders. No disease relapse or clonal progression was documented during follow-up. Treatment-related adverse events occurred in 30% of the patients (all grade 1-2 reversible events). One responder died of infectious multiple organ failure at 8 months after treatment initiation. Our results indicate that zanubrutinib provides rapid amelioration of anemia with manageable short-term safety in patients with relapsed/refractory wAIHA.

摘要

这项回顾性分析纳入了10例复发/难治性温抗体型自身免疫性溶血性贫血(wAIHA)患者,这些患者于2022年7月至2024年1月在北京协和医院接受了泽布替尼治疗(疗程:3 - 6个月)并完成了至少3个月的随访。该队列包括2例男性和8例女性患者,中位年龄为63岁(范围:36 - 76岁),既往治疗的中位时间为9个月(范围:3 - 22个月)。在1个月、3个月和最终随访(中位7个月)时,总体缓解(OR)率分别为30%、60%和60%,相应的完全缓解(CR)率分别为20%、40%和40%。缓解者达到缓解的中位时间为2个月(范围:1 - 2个月)。随访期间未记录到疾病复发或克隆进展。30%的患者发生了与治疗相关的不良事件(均为1 - 2级可逆事件)。1例缓解者在治疗开始后8个月死于感染性多器官功能衰竭。我们的结果表明,泽布替尼可使复发/难治性wAIHA患者的贫血迅速改善,且短期安全性可控。

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