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

Anders Ronneholt-Frederiksen, Dennis Lund Hansen, Vuokko Nummi, Jesper Stentoft, Ulrik M. Overgaard, Rie Sander Bech, Anna-Elina Lehtinen, Andreas Glenthoj, Henrik Frederiksen*

*Corresponding author af dette arbejde

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Abstract

Introduction 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.Methods We conducted a case series of 20 PNH patients who transitioned from eculizumab to ravulizumab, assessing transfusion needs and breakthrough haemolysis episodes (BTH).Results 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.Conclusion Overall, ravulizumab provided comparable or improved disease control, supporting its use as a long-term treatment in PNH.Trial Registration The authors have confirmed clinical trial registration is not needed for this submission
OriginalsprogEngelsk
Artikelnummere70132
TidsskrifteJHaem
Vol/bind6
Udgave nummer4
Antal sider5
ISSN2688-6146
DOI
StatusUdgivet - 2025

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