Disease Activity After Switching From Eculizumab to Ravulizumab in Patients With Paroxysmal Nocturnal Haemoglobinuria: Real World Data From Denmark and Finland.

ABSTRACT: 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.

SUBMITTER: Ronneholt-Frederiksen A 

PROVIDER: S-EPMC12366438 | biostudies-literature | 2025 Aug

REPOSITORIES: biostudies-literature