Objective: To evaluate the efficacy and safety of eculizumab in the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in China. Methods: Data from PNH patients who received at least 3 months of full-dose eculizumab and were followed for at least 3 months between December 2022 and July 2023 were retrospectively collected. We evaluated changes in clinical and laboratory parameters after 1, 2, 3, and 6 months of eculizumab treatment. The rates of breakthrough hemolysis (BTH), extravascular hemolysis (EVH), and the occurrence of adverse reactions were also monitored. Results: The study included nine patients, six males and three females, with a median age of 54 (28-69) years. 5 of the patients had classic PNH, while 4 had PNH/AA. The number of episodes of hemoglobinuria was 5 (1-25) per month before eculizumab. 4 patients required blood transfusion, 5 had thrombosis and one had renal impairment before eculizumab. The median time to eculizumab was 6 (3-7) months and the followup period was 3 (3-6) months after treatment. The number of episodes of hemoglobinuria following eculizumab was 0 (0-1). During the followup period, no additional thrombotic events occurred. LDH at any time after eculizumab was lower than at baseline, and some patients' HGB increased. All transfused patients became transfusion-independent after receiving eculizumab. The FACIT-Fatigue score improved by an average of 17.3 points following treatment. 2 patients developed BTH and improved with symptomatic treatment. There were three adverse events that caused mild symptoms. There are no serious adverse events or deaths. Conclusion: Eculizumab can effectively control the hemolytic-related symptoms of PNH in China, reducing the need for blood transfusions to some extent, while also demonstrating a higher safety profile.
目的： 观察依库珠单抗在中国治疗溶血性阵发性睡眠性血红蛋白尿症（PNH）患者中的有效性和安全性。 方法： 回顾性分析2022年12月至2023年7月接受至少3个月足量依库珠单抗治疗并随访至少3个月的溶血性PNH患者资料，评估依库珠单抗治疗1、2、3和6个月后临床及实验室指标的变化，并记录突破性溶血（BTH）、血管外溶血（EVH）的比例及不良反应发生情况。 结果： 共纳入9例PNH患者，其中男6例，女3例，中位年龄54（28～69）岁。5例患者为经典型PNH，4例为PNH/再生障碍性贫血（AA）患者。在使用依库珠单抗前，血红蛋白尿发作次数为每月5（1～25）次。4例患者在接受依库珠单抗前需要输血，5例合并血栓，1例有肾功能损害。依库珠单抗的治疗中位时间为6（3～7）个月，随访时间为治疗后3（3～6）个月。使用依库珠单抗后，血红蛋白尿发作次数为0（0～1）次。在随访期内，无新发血栓形成。治疗后各个时间节点的LDH均较基线明显下降；部分患者HGB较基线有上升；输血依赖者均摆脱输血。疲劳功能评分（FACIT-Fatigue评分）在治疗后平均提升17.3分。2例患者发生BTH，经对症好转。3例出现了轻度不良事件，无严重不良事件及死亡。 结论： 依库珠单抗对中国溶血性PNH的相关症状有较好的控制作用，在一定程度上能减少输血，改善患者生活质量，且安全性较好。.
Keywords: Eculizumab; Efficacy; Hemolysis; Paroxysmal nocturnal hemoglobinuria; Safety.