中国实用内科杂志2025，Vol.45Issue(7)：593-599,605,8.DOI:10.19538/j.nk2025070109
依库珠单抗治疗高疾病活动状态阵发性睡眠性血红蛋白尿症后持续贫血原因分析
Analysis of persistent anemia following Eculizumab treatment in paroxysmal nocturnal hemoglobinuria patients with high disease-activity status
摘要
Abstract
Objective To investigate the factors contributing to persistent anemia in Chinese patients with high disease-activity paroxysmal nocturnal hemoglobinuria(PNH)undergoing Eculizumab therapy.Methods A prospective registry-based analysis was conducted on 33 high disease-activity PNH patients treated with Eculizumab for at least 6 months between December 2023 and December 2024 in the hospitals of Chinese Eastern Collaboration Group of Anemia,analyze the patient's condition and causes of persistent anemia.Results Among 33 patients,9 patients maintained Hb＞100 g/L(1 patient ≥120 g/L),and 24 patients exhibited Hb＜100 g/L.The incidence of comorbid aplastic anemia was lower in patients with Hb level of 10 or more(22％vs.67％,P=0.029).Anti-C3d positivity via direct antiglobulin test(DAT)was detected in 13 patients(13/33,39％),with median onset time of 12(2-32)weeks and median anti-C3d titer of 64(16-256).7 patients demonstrated C3d titers ≥1:64.At 6-month follow-up after eculizumab treatment,the median PNH clone size in type Ⅱ+Ⅲerythrocytes was 17.7％(1.8％-99.7％),which is lower than that in neutrophil clones.Twenty patients exhibited PNH size in type Ⅱ+Ⅲ erythrocyte were 10 percentage or lower by comparing to neutrophil,while 5 patients showed 5％-9％difference.More patients with Hb levels＞100 g/L in whom with lower differences in PNH clone size between erythrocyte and neutrophil(40％vs.100％,P=0.024).Twelve patients had elevated LDH levels.All 12 patients with elevated LDH showed＞10％lower type Ⅱ+Ⅲ erythrocyte clones compared to neutrophil clones.Hb＞100 g/L was achieved in 25％(3/12)of elevated LDH patients vs.77％(10/13)of normal LDH patients(25％vs.77％,P=0.013).3 patients experienced breakthrough hemolysis(BTH),all occurring after 32(15-91)days of delayed Eculizumab administration.Conclusion Persistent anemia post-Eculizumab treatment in high disease activity statePNHcorrelates significantly with concurrent aplastic anemia,extravascular hemolysis,residual intravascular hemolysis and breakthrough hemolysis.关键词
阵发性睡眠性血红蛋白尿症/高疾病活动状态/依库珠单抗/持续贫血病因Key words
paroxysmal nocturnal hemoglobinuria/high disease-activity status/Eculizumab/persistent anemia etiology分类
医药卫生引用本文复制引用
张婷,施小凤,何广胜,师锦宁,袁颖莹,秦程涛,马永超,宫跃敏,张雅文,方宝枝,吕明恩,徐金格..依库珠单抗治疗高疾病活动状态阵发性睡眠性血红蛋白尿症后持续贫血原因分析[J].中国实用内科杂志,2025,45(7):593-599,605,8.基金项目
国家自然科学基金(81900109,82400138) （81900109,82400138）
江苏省自然科学基金(BK20230734) （BK20230734）
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(JNYYZXKY202214) （JNYYZXKY202214）
南京市江宁区科技惠民计划项目(2023081S) （2023081S）
南京医科大学附属江宁医院青年创新科研基金临床研究项目(JNYYZXKY202414) （JNYYZXKY202414）
