CASE REPORT article
Front. Med.
Sec. Hematology
Volume 12 - 2025 | doi: 10.3389/fmed.2025.1583394
This article is part of the Research TopicComplementRarity – Complement System in Rare Systemic and Renal Diseases: A New Vision of an Old System.View all articles
Case report: Iptacopan in a paroxysmal nocturnal hemoglobinuria patient with severe renal insufficiency
Provisionally accepted
Ting Wu
Qin Xiao Wang*- Huashan Hospital, Fudan University, Shanghai, China
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Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disorder characterized by complement-mediated hemolysis, thrombosis, and bone marrow failure. Iptacopan, an oral factor B inhibitor, has demonstrated efficacy in managing PNH but has not been studied in patients with severe renal insufficiency. We report a case of a PNH patient with end-stage renal disease who required renal replacement therapy and had a peritoneal dialysis catheter placed during treatment. After switching from eculizumab to iptacopan, the patient achieved transfusion independence, sustained hematologic improvement, and resolution of both intravascular and extravascular hemolysis. Iptacopan was well tolerated, with only mild adverse effects and no breakthrough hemolysis or infections. This case highlights the potential of iptacopan as a therapeutic option in PNH patients with severe renal impairment requiring dialysis.
Keywords: Paroxysmal nocturnal hemoglobinuria, iptacopan, Complement inhibition, Extravascular hemolysis, Renal Insufficiency
Received: 23 Apr 2025; Accepted: 11 Jul 2025.
Copyright: © 2025 Wu and Wang. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: Qin Xiao Wang, Huashan Hospital, Fudan University, Shanghai, China
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