Effectiveness of Iptacopan Versus C5 Inhibitors in Complement Inhibitor‐Naive Patients With Paroxysmal Nocturnal Haemoglobinuria

Effectiveness of Iptacopan Versus C5 Inhibitors in Complement Inhibitor‐Naive Patients With Paroxysmal Nocturnal Haemoglobinuria

ABSTRACT Background Paroxysmal nocturnal haemoglobinuria (PNH) is characterised by haemolytic anaemia, bone marrow failure and thrombosis. The single‐arm phase 3 APPOINT‐PNH trial (NCT04820530) investigating iptacopan monotherapy in complement inhibitor‐naive patients demonstrated significant haemog...

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Bibliographic Details
Main Authors: Matthew Holt, Richard J. Kelly, Jilles M. Fermont, Soudeh Ansari, Marion Dahlke, Isabelle Brindel, Lynda Maafa, Flore Sicre deFontbrune, Régis Peffault deLatour
Format: Article
Language:English
Published: Wiley 2025-06-01
Series:eJHaem
Subjects:
APPEX
APPOINT‐PNH
C5 inhibitors
iptacopan
paroxysmal nocturnal haemoglobinuria
Online Access:https://doi.org/10.1002/jha2.70055
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https://doi.org/10.1002/jha2.70055

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