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依库珠单抗治疗造血干细胞移植相关血栓性微血管病的回顾性临床分析:附11例报告

[Retrospective clinical analysis of eculizumab treatment for hematopoietic stem cell transplantation-associated thrombotic microangiopathy: a report of 11 cases].

作者信息

Luo X Y, Ma R, Wang H F, Bai L, He Y, Zhang Y Y, Han T T, Deng D X, Chen Y Y, Han W, Zhang X H, Xu L P, Wang Y, Huang X J, Sun Y Q

机构信息

Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing 100044, China.

出版信息

Zhonghua Xue Ye Xue Za Zhi. 2025 May 14;46(5):431-436. doi: 10.3760/cma.j.cn121090-20240722-00273.

Abstract

To evaluate the efficacy of eculizumab in treating hematopoietic stem cell transplantation-associated thrombotic microangiopathy (TA-TMA) . This retrospective study included 11 patients who developed TA-TMA after allogeneic hematopoietic stem cell transplantation and subsequently received eculizumab treatment at Peking University People's Hospital between June 2018 and May 2024. The incidence of TA-TMA, treatment details, and clinical outcomes were analyzed. Among the 11 included patients [4 males, 7 females; median age: 29 years (range: 9-56) ], underlying diseases were severe aplastic anemia (SAA) in 5 patients, acute lymphoblastic leukemia (ALL) in 3 patients, and acute myeloid leukemia (AML) in 3 patients. The median time to TA-TMA diagnosis was 48 days post-transplantation (range: 4-213 days), and all patients met the diagnostic criteria for high-risk TA-TMA. The median interval from TA-TMA diagnosis to the initiation of eculizumab treatment was 12 days (range: 1-56 days). Patients received a median of 3 doses of eculizumab (range: 1-14). Ten of the 11 patients were assessed as having no response (NR) to eculizumab at the end of treatment or at death. One patient achieved a partial response (PR) but subsequently died after TA-TMA relapsed due to infection. At the last follow-up, all patients were either lost to follow-up or had died. The median follow-up duration was 88 days (range: 33-326 days), and the median time from TA-TMA diagnosis to the last follow-up was 31 days (range: 21-113 days) . Eculizumab demonstrated poor efficacy in this TA-TMA cohort. This might be attributable to the critical and complex condition of the patients, delayed initiation of eculizumab treatment, and insufficient dosage.

摘要

评估依库珠单抗治疗造血干细胞移植相关血栓性微血管病(TA-TMA)的疗效。这项回顾性研究纳入了11例在异基因造血干细胞移植后发生TA-TMA并于2018年6月至2024年5月期间在北京人民医院接受依库珠单抗治疗的患者。分析了TA-TMA的发生率、治疗细节及临床结局。在纳入的11例患者中[4例男性,7例女性;中位年龄:29岁(范围:9 - 56岁)],基础疾病为5例重型再生障碍性贫血(SAA)、3例急性淋巴细胞白血病(ALL)、3例急性髓系白血病(AML)。TA-TMA诊断的中位时间为移植后48天(范围:4 - 213天),所有患者均符合高危TA-TMA的诊断标准。从TA-TMA诊断到开始依库珠单抗治疗的中位间隔时间为12天(范围:1 - 56天)。患者接受依库珠单抗的中位剂量为3剂(范围:1 - 14剂)。11例患者中有10例在治疗结束时或死亡时被评估为对依库珠单抗无反应(NR)。1例患者获得部分缓解(PR),但随后因感染导致TA-TMA复发而死亡。在最后一次随访时,所有患者均失访或死亡。中位随访时间为88天(范围:33 - 326天),从TA-TMA诊断到最后一次随访的中位时间为31天(范围:21 - 113天)。依库珠单抗在该TA-TMA队列中显示出疗效不佳。这可能归因于患者病情危急复杂、依库珠单抗治疗启动延迟以及剂量不足。

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