AUTHOR=Fang Baozhi , Lu Hongbin , Yu Xiao , Wang Peng , Zhou Yifei , Shen Qiudan , Yuan Muzhi , Lyu Mingen , He Guangsheng 

TITLE=Iptacopan for cold agglutinin disease: a case report with literature review

JOURNAL=Frontiers in Immunology

VOLUME=Volume 16 - 2025

YEAR=2025

URL=https://www.frontiersin.org/journals/immunology/articles/10.3389/fimmu.2025.1672590

DOI=10.3389/fimmu.2025.1672590

ISSN=1664-3224

ABSTRACT=This study reports a case of cold agglutinin disease (CAD) secondary to lymphoplasmacytic lymphoma in a patient intolerant to rituximab plus bendamustine and with persistent uncontrolled hemolysis following zanubrutinib therapy. The addition of the complement C3 inhibitor iptacopan to a cyclophosphamide and dexamethasone regimen successfully controlled hemolysis and improved hemoglobin levels. Within one week of treatment, the patient achieved transfusion independence, with hemoglobin increasing from 67 g/L to 90 g/L by week 3 and 101 g/L by week 7, alongside normalized bilirubin levels and no adverse events. The follow-up period was 4 months, during which the patient showed sustained remission. These findings suggest that iptacopan can rapidly ameliorate hemolysis in CAD, warranting further investigation into its therapeutic potential.