Zhang Yawen, Gong Yuemin, He Guangsheng
Department of Hematology, The First Affiliated Hospital with Nanjing Medical University, Jiangsu Province Hospital, Key Laboratory of Hematology of Nanjing Medical University, Collaborative Innovation Center for Cancer Personalized Medicine, Nanjing, People's Republic of China.
Hematology. 2025 Dec;30(1):2548091. doi: 10.1080/16078454.2025.2548091. Epub 2025 Aug 25.
Autoimmune hemolytic anemia (AIHA) is characterized by autoimmune-mediated destruction of erythrocytes. Both AIHA and Evans syndrome are rare, manifesting a severe clinical course, high relapse rate, and potentially fatal outcomes. Refractory AIHA shows poor responsiveness to multiple treatment regimens, and there is still a lack of effective treatment regimens for serious hemolytic episodes.
Three refractory AIHA cases with a positive complement component C3d and hemolytic flare were treated with the oral factor B inhibitor ptacopan in conjunction with cyclophosphamide and prednisone.
After treatment with iptacopan plus cyclophosphamide and prednisone, all three patients showed a rapid increase in Hb levels, a decrease in reticulocyte proportion, and a significant reduction in hemolysis manifestations (Lower LDH level and unconjugated bilirubin).
Three refractory cases of AIHA showed good therapeutic efficacy after treatment with iptacopan combined with cyclophosphamide and prednisone. These cases provide a potentially effective treatment option for severe hemolytic episodes in refractory AIHA.
自身免疫性溶血性贫血（AIHA）的特征是自身免疫介导的红细胞破坏。AIHA和Evans综合征均较为罕见，临床病程严重，复发率高，且可能导致致命后果。难治性AIHA对多种治疗方案反应不佳，对于严重溶血发作仍缺乏有效的治疗方案。
对3例补体成分C3d阳性且有溶血发作的难治性AIHA患者，采用口服因子B抑制剂培他卡朋联合环磷酰胺和泼尼松进行治疗。
培他卡朋联合环磷酰胺和泼尼松治疗后，所有3例患者的血红蛋白水平迅速升高，网织红细胞比例降低，溶血表现显著减轻（乳酸脱氢酶水平和非结合胆红素降低）。
3例难治性AIHA患者经培他卡朋联合环磷酰胺和泼尼松治疗后显示出良好的治疗效果。这些病例为难治性AIHA的严重溶血发作提供了一种潜在有效的治疗选择。
