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本文引用的文献

1
A phase 2 trial of orelabrutinib showing promising efficacy and safety in patients with persistent or chronic primary immune thrombocytopenia.一项评估奥雷巴替尼治疗持续性或慢性原发性免疫性血小板减少症患者的2期试验显示出有前景的疗效和安全性。
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2
The Role of Bruton's Kinase Inhibitors in Chronic Lymphocytic Leukemia: Current Status and Future Directions.布鲁顿激酶抑制剂在慢性淋巴细胞白血病中的作用:现状与未来方向
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Bruton Tyrosine Kinase Inhibitors in B-Cell Malignancies: Their Use and Differential Features.布鲁顿酪氨酸激酶抑制剂在 B 细胞恶性肿瘤中的应用及其特点。
Target Oncol. 2022 Jan;17(1):69-84. doi: 10.1007/s11523-021-00857-8. Epub 2021 Dec 14.
4
Review of the development of BTK inhibitors in overcoming the clinical limitations of ibrutinib.BTK 抑制剂克服伊布替尼临床限制的发展综述。
Eur J Med Chem. 2022 Feb 5;229:114009. doi: 10.1016/j.ejmech.2021.114009. Epub 2021 Nov 22.
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Autoimmune Hemolytic Anemia in Chronic Lymphocytic Leukemia: A Comprehensive Review.慢性淋巴细胞白血病中的自身免疫性溶血性贫血:综述
Cancers (Basel). 2021 Nov 19;13(22):5804. doi: 10.3390/cancers13225804.
6
The role of Bruton's tyrosine kinase in the immune system and disease.布鲁顿酪氨酸激酶在免疫系统和疾病中的作用。
Immunology. 2021 Dec;164(4):722-736. doi: 10.1111/imm.13416. Epub 2021 Oct 4.
7
Kinase inhibition in autoimmunity and inflammation.激酶抑制在自身免疫和炎症中的作用。
Nat Rev Drug Discov. 2021 Jan;20(1):39-63. doi: 10.1038/s41573-020-0082-8. Epub 2020 Oct 19.
8
[Ibrutinib treatment for 2 cases of relapsed/refractory autoimmune hemolytic anemia: a pilot study].伊布替尼治疗2例复发/难治性自身免疫性溶血性贫血的初步研究
Zhonghua Xue Ye Xue Za Zhi. 2020 May 14;41(5):412-416. doi: 10.3760/cma.j.issn.0253-2727.2020.05.009.
9
Diagnosis and treatment of autoimmune hemolytic anemia in adults: Recommendations from the First International Consensus Meeting.成人自身免疫性溶血性贫血的诊断和治疗:首次国际共识会议的建议。
Blood Rev. 2020 May;41:100648. doi: 10.1016/j.blre.2019.100648. Epub 2019 Dec 5.
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Autoimmune thrombocytopenia: Current treatment options in adults with a focus on novel drugs.自身免疫性血小板减少症:成人的当前治疗选择,重点关注新型药物。
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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.

DOI:10.3760/cma.j.cn121090-20241110-00442
PMID:40425458
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12158813/
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。