依库珠单抗控制阵发性睡眠性血红蛋白尿症的溶血危机  

Efficacy of eculizumab in hemolytic crisis of paroxysmal nocturnal hemoglobinuria

作　　者：杨慧[1] 隋秀芳 方宝枝 张婷 徐金格 吕明恩[3] 师锦宁 陈小玉 宫跃敏 张雅文[1] 何广胜[1] YANG Hui;SUI Xiufang;FANG Baozhi;ZHANG Ting;XU Jinge;LV Ming'en;SHI Jinning;CHEN Xiaoyu;GONG Yuemin;ZHANG Yawen;HE Guangsheng(Department of Hematology,the First Affiliated Hospital of Nanjing Medical University,Jiangsu Province Hospital,Nanjing,210029,China;Department of Hematology,Qingdao Women and Children's Hospital;Department of Hematology,Suzhou Municipal Hospital;Department of Hematology,Nanjing Jiangning Hospital;Department of Hematology,the Second Affiliated Hospital of Xuzhou Medical University)

机构地区：[1]南京医科大学附属第一医院江苏省人民医院血液科,南京210029 [2]青岛妇女儿童医院血液科 [3]苏州市立医院血液科 [4]南京医科大学附属江宁医院血液科 [5]徐州医科大学第二附属医院血液科

出　　处：《临床血液学杂志》2024年第11期799-802,共4页Journal of Clinical Hematology

基　　金：国家自然科学基金(No:81900109);南京医科大学附属江宁医院免疫细胞转化研究中心开放课题(No:JNYYZXKY202214);南京市江宁区科技惠民计划项目(No:2023081S)。

摘　　要：目的末端补体C5抑制剂依库珠单抗(eculizumab)被推荐为阵发性睡眠性血红蛋白尿症(paroxysmal nocturnal hemoglobinuria,PNH)的一线治疗方案。本研究探索了21例PNH患者接受依库珠单抗治疗的疗效及安全性。方法使用依库珠单抗治疗21例溶血发作的PNH患者,评价患者溶血、贫血、肾功能及D-二聚体水平。结果治疗后随访中位时间7天时,患者溶血、贫血及肾功能指标改善,D-二聚体水平下降,仅1例患者需要输血。长期规律应用依库珠单抗的PNH患者,在第2剂使用后4例(4/5,80%)达到溶血控制(乳酸脱氢酶<1.5×正常值上限),第3剂使用后全部达到溶血完全控制。9例仅接受1次依库珠单抗治疗,在随访28天时3例出现突破性溶血。12例提前接受脑膜炎球菌疫苗接种,另外9例紧急治疗者接受抗生素预防感染,随访中均未发生感染。结论依库珠单抗能够有效控制PNH患者的溶血危机,改善肾功能,降低D-二聚体水平。紧急治疗未接受脑膜炎球菌疫苗接种者经预防性使用抗生素,可以避免感染。Objective Eculizumab as a terminal complement inhibitor is recommended as the first-line treatment for paroxysmal nocturnal hemoglobinuria(PNH).This study aimed to explore the efficacy and safety of eculizumab.Methods The indicators of hemolysis,anemia,kidney function,and the D-dimer in 21 PNH patients receiving with eculizumab treatment were retrospectively evaluated.Results After being treated with eculizumab,the hemolysis,anemia,and renal function of patients were improved and D-dimer levels declined compared to the baseline.Only one patient needed a blood transfusion.Five patients who were administered long-term regular eculizumab had achieved hemolysis control(lactate dehydrogenase<1.5 upper limit of normal)after the third dose,and lactate dehydrogenase returned to normal levels.Three of nine PNH patients who only received eculizumab once had breakthrough hemolysis after 28 days.Among 21 patients,12 cases received meningococcal vaccination in advance,9 cases were treated urgently with antibiotics against infection,and all remained uninfected during the follow-up period.Conclusion The hemolytic breakthrough in PNH patients benefited from eculizumab in improving anemia,kidney function,and D-dimer levels.In emergency treatment,antibiotics are necessary to be given and could avoid infection.

关 键 词：依库珠单抗 阵发性睡眠性血红蛋白尿症 溶血 感染 突破性溶血 

分 类 号：R556.64[医药卫生—血液循环系统疾病]