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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| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
| Published: |
Taylor & Francis Group
2025-12-01
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| Series: | Hematology |
| Subjects: | |
| Online Access: | https://www.tandfonline.com/doi/10.1080/16078454.2025.2548091 |
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| Summary: | 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. |
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| ISSN: | 1607-8454 |