Objective: To evaluate the efficacy of eculizumab in treating hematopoietic stem cell transplantation-associated thrombotic microangiopathy (TA-TMA) . Methods: 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. Results: 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) . Conclusion: 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.
目的： 评估依库珠单抗（Eculizumab）治疗造血干细胞移植相关血栓性微血管病（TA-TMA）的疗效。 方法： 纳入2018年6月至2024年5月在北京大学人民医院接受异基因造血干细胞移植后发生TA-TMA并应用依库珠单抗治疗的11例患者，对TA-TMA发生情况、治疗及临床结局进行回顾性研究。 结果： 纳入研究的11例患者中男4例，女7例，中位年龄29（9~56）岁；重型再生障碍性贫血（SAA）5例、急性淋巴细胞白血病（ALL）3例、急性髓系白血病（AML）3例。TMA中位诊断时间为移植后48（4~213）d，所有患者均符合高危TA-TMA诊断标准。诊断TMA到启动依库珠单抗治疗的中位间隔时间为12（1~56）d，依库珠单抗治疗中位次数为3（1~14）次。10例患者在依库珠单抗治疗结束和死亡时评估疗效均为无反应（NR）。1例曾获得部分缓解（PR），后续因感染引起TA-TMA复发后死亡。截止末次随访，所有患者均失访/死亡，中位随访时间88（33~326）d，诊断TA-TMA至末次随访时间为31（21~113）d。 结论： 依库珠单抗治疗TA-TMA疗效较差，可能与患者病情危重、病情复杂、启动依库珠单抗治疗较晚及剂量不足有关。.
Keywords: Eculizumab; Hematopoietic stem cell transplantation; Transplant-associated thrombotic microangiopathy.