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
Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing 100044, China.
Zhonghua Xue Ye Xue Za Zhi. 2025 May 14;46(5):431-436. doi: 10.3760/cma.j.cn121090-20240722-00273.
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.
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。