依库珠单抗治疗溶血性阵发性睡眠性血红蛋白尿症的中国真实世界研究

Eculizumab in patients with paroxysmal nocturnal hemoglobinuria:a real-world study in China

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。

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 O(O-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.