使用iptacopan治疗PNH患者的真实世界经验：病例系列

文献信息

中文摘要

本病例系列关于使用iptacopan管理阵发性睡眠性血红蛋白尿症（PNH），重点关注突破性溶血（BTH）或血栓事件风险增高的情况。患者1为46岁女性，患有PNH并有肝静脉血栓形成史。患者2为30岁女性，患有PNH并有右大脑后动脉卒中及脑静脉窦血栓形成伴广泛性右侧浅表血栓性静脉炎史。患者3为48岁女性，患有PNH，在开始iptacopan治疗3个月后发生胃肠道感染。患者4为39岁男性，患有PNH，出现浆液性中耳炎，这是一种补体放大状况（CAC），同时伴有上呼吸道感染。患者5为56岁女性，患有PNH，在iptacopan治疗期间接受了脊柱射频消融术。在iptacopan治疗期间，没有患者发生BTH或住院。开始iptacopan治疗后，未报告新的血栓事件。对于经历CAC或手术的患者，无需改变PNH治疗。值得注意的是，在可能引起药物吸收不良的胃肠炎患者中，未发生BTH。本病例系列表明，在有既往血栓形成、胃肠道疾病和其他CAC的情况下，iptacopan能有效维持补体抑制并预防PNH相关溶血和血栓形成。

英文摘要

This case series on managing paroxysmal nocturnal hemoglobinuria (PNH) with iptacopan focuses on scenarios in which the risk of breakthrough hemolysis (BTH) or thrombotic events is heightened. Patient 1 was a 46-year-old woman with PNH and a history of hepatic vein thrombosis. Patient 2 was a 30-year-old woman with PNH and a history of right posterior cerebral artery stroke and cerebral venous sinus thrombosis with extensive right superficial thrombophlebitis. Patient 3 was a 48-year-old woman with PNH who contracted a gastrointestinal infection 3 months after starting iptacopan. Patient 4 was a 39-year-old man with PNH who experienced serous otitis media, a complement-amplifying condition (CAC), concurrent with upper respiratory infection. Patient 5 was a 56-year-old woman with PNH who underwent radiofrequency ablation of the spine while on iptacopan. No patients experienced BTH or hospitalization while on iptacopan. No new thrombotic events were reported after initiating iptacopan. For patients experiencing CACs or surgery, no changes in PNH therapy were required. Notably, BTH did not occur in the patient with gastroenteritis, which can cause drug malabsorption. This case series suggests that iptacopan effectively maintains complement inhibition and prevents PNH-related hemolysis and thrombosis in the setting of past thromboses, gastrointestinal illness, and other CACs.