Rønneholt-Frederiksen Anders, Hansen Dennis Lund, Nummi Vuokko, Stentoft Jesper, Overgaard Ulrik M, Bech Rie Sander, Lehtinen Anna-Elina, Glenthøj Andreas, Frederiksen Henrik
Department and Research Unit of Hematology Odense University Hospital Odense Denmark.
Department of Clinical Research University of Southern Denmark Odense Denmark.
EJHaem. 2025 Aug 20;6(4):e70132. doi: 10.1002/jha2.70132. eCollection 2025 Aug.
Ravulizumab replaced eculizumab as the preferred complement inhibitor for Paroxysmal Nocturnal Haemoglobinuria (PNH) in Denmark and Finland after 2020. Data on real-world disease activity after switching are limited.
We conducted a case series of 20 PNH patients who transitioned from eculizumab to ravulizumab, assessing transfusion needs and breakthrough haemolysis episodes (BTH).
In 19 of 20 patients, transfusions and BTH were stable or reduced after switching. Particularly, patients with high transfusion or BTH burden had a marked reduction in these parameters after switching.
Overall, ravulizumab provided comparable or improved disease control, supporting its use as a long-term treatment in PNH.
The authors have confirmed clinical trial registration is not needed for this submission.
2020年后，ravulizumab在丹麦和芬兰取代依库珠单抗成为阵发性夜间血红蛋白尿（PNH）的首选补体抑制剂。转换治疗后真实世界疾病活动的数据有限。
我们对20例从依库珠单抗转换为ravulizumab的PNH患者进行了病例系列研究，评估输血需求和突破性溶血发作（BTH）情况。
20例患者中有19例在转换治疗后输血需求和BTH情况稳定或减少。特别是，输血或BTH负担高的患者在转换治疗后这些参数有显著降低。
总体而言，ravulizumab提供了相当或更好的疾病控制效果，支持其作为PNH的长期治疗药物。
作者已确认本研究无需临床试验注册。
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