Severe hemolysis flare of refractory autoimmune hemolytic anemia with positive complement component C3d responsive to Iptacopan with cyclophosphamide and prednisone: a case report
Objectives: 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 tr...
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| Format: | Article |
| Language: | English |
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Taylor & Francis Group
2025-12-01
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| Series: | Hematology |
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| Online Access: | https://www.tandfonline.com/doi/10.1080/16078454.2025.2548091 |
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| _version_ | 1849224193898446848 |
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| author | Yawen Zhang Yuemin Gong Guangsheng He |
| author_facet | Yawen Zhang Yuemin Gong Guangsheng He |
| author_sort | Yawen Zhang |
| collection | DOAJ |
| description | Objectives: 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.Methods: 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.Results: 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).Conclusion: 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. |
| format | Article |
| id | doaj-art-1391b3a0b695427e9b23602cb636bd11 |
| institution | Kabale University |
| issn | 1607-8454 |
| language | English |
| publishDate | 2025-12-01 |
| publisher | Taylor & Francis Group |
| record_format | Article |
| series | Hematology |
| spelling | doaj-art-1391b3a0b695427e9b23602cb636bd112025-08-25T13:33:27ZengTaylor & Francis GroupHematology1607-84542025-12-0130110.1080/16078454.2025.2548091Severe hemolysis flare of refractory autoimmune hemolytic anemia with positive complement component C3d responsive to Iptacopan with cyclophosphamide and prednisone: a case reportYawen Zhang0Yuemin Gong1Guangsheng He2Department 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 ChinaDepartment 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 ChinaDepartment 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 ChinaObjectives: 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.Methods: 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.Results: 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).Conclusion: 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.https://www.tandfonline.com/doi/10.1080/16078454.2025.2548091Autoimmune hemolytic anemiaIptacopancomplement inhibitorcomplement component C3dcase report |
| spellingShingle | Yawen Zhang Yuemin Gong Guangsheng He Severe hemolysis flare of refractory autoimmune hemolytic anemia with positive complement component C3d responsive to Iptacopan with cyclophosphamide and prednisone: a case report Hematology Autoimmune hemolytic anemia Iptacopan complement inhibitor complement component C3d case report |
| title | Severe hemolysis flare of refractory autoimmune hemolytic anemia with positive complement component C3d responsive to Iptacopan with cyclophosphamide and prednisone: a case report |
| title_full | Severe hemolysis flare of refractory autoimmune hemolytic anemia with positive complement component C3d responsive to Iptacopan with cyclophosphamide and prednisone: a case report |
| title_fullStr | Severe hemolysis flare of refractory autoimmune hemolytic anemia with positive complement component C3d responsive to Iptacopan with cyclophosphamide and prednisone: a case report |
| title_full_unstemmed | Severe hemolysis flare of refractory autoimmune hemolytic anemia with positive complement component C3d responsive to Iptacopan with cyclophosphamide and prednisone: a case report |
| title_short | Severe hemolysis flare of refractory autoimmune hemolytic anemia with positive complement component C3d responsive to Iptacopan with cyclophosphamide and prednisone: a case report |
| title_sort | severe hemolysis flare of refractory autoimmune hemolytic anemia with positive complement component c3d responsive to iptacopan with cyclophosphamide and prednisone a case report |
| topic | Autoimmune hemolytic anemia Iptacopan complement inhibitor complement component C3d case report |
| url | https://www.tandfonline.com/doi/10.1080/16078454.2025.2548091 |
| work_keys_str_mv | AT yawenzhang severehemolysisflareofrefractoryautoimmunehemolyticanemiawithpositivecomplementcomponentc3dresponsivetoiptacopanwithcyclophosphamideandprednisoneacasereport AT yuemingong severehemolysisflareofrefractoryautoimmunehemolyticanemiawithpositivecomplementcomponentc3dresponsivetoiptacopanwithcyclophosphamideandprednisoneacasereport AT guangshenghe severehemolysisflareofrefractoryautoimmunehemolyticanemiawithpositivecomplementcomponentc3dresponsivetoiptacopanwithcyclophosphamideandprednisoneacasereport |