Wu Ting, Wang Xiaoqin
Department of Hematology, Huashan Hospital, Fudan University, Shanghai, China.
Front Med (Lausanne). 2025 Aug 5;12:1583394. doi: 10.3389/fmed.2025.1583394. eCollection 2025.
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.
阵发性睡眠性血红蛋白尿（PNH）是一种罕见的疾病，其特征为补体介导的溶血、血栓形成和骨髓衰竭。Iptacopan是一种口服因子B抑制剂，已证明在治疗PNH方面有效，但尚未在严重肾功能不全的患者中进行研究。我们报告了一例患有终末期肾病的PNH患者，该患者需要肾脏替代治疗，且在治疗期间放置了腹膜透析导管。从依库珠单抗换用Iptacopan后，患者实现了输血独立，血液学持续改善，血管内和血管外溶血均得到缓解。Iptacopan耐受性良好，仅有轻微不良反应，未出现突破性溶血或感染。该病例突出了Iptacopan作为治疗需要透析的严重肾功能损害PNH患者的一种治疗选择的潜力。
