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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Bibliografski detalji

Glavni autori:	Matthew Holt, Richard J. Kelly, Jilles M. Fermont, Soudeh Ansari, Marion Dahlke, Isabelle Brindel, Lynda Maafa, Flore Sicre deFontbrune, Régis Peffault deLatour
Format:	Artigo
Jezik:	Inglês
Izdano:	Wiley 2025-06-01
Serija:	eJHaem
Teme:	APPEX APPOINT‐PNH C5 inhibitors iptacopan paroxysmal nocturnal haemoglobinuria
Online pristup:	https://doi.org/10.1002/jha2.70055
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