Iptacopan combined with immunosuppressive therapy for aplastic anemia-paroxysmal nocturnal hemoglobinuria syndrome: A case report. (PubMed, Medicine (Baltimore)) - Oct 18, 2026 - "This case suggests that iptacopan combined with IST may represent a potentially effective and safe therapeutic option for AA-PNH syndrome that is refractory to conventional IST and corticosteroids. Especially for AA-PNH patients with impaired bone marrow function and continuous dependence on blood transfusion, iptacopan combined with IST may be an effective and well-tolerated treatment regimen."
Journal • Anemia • Aplastic Anemia • Complement-mediated Rare Disorders • Fatigue • Hematological Disorders • Immunology • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • CD59
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Jianping Li; Jie Xu; Linying Li; Xiaoqian Liu; Yaqin Luo; Yingying Wang; Zhaoxia Liu
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