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EBMT 2024 | Complement inhibition with iptacopan has high efficacy in PNH: insights from APPLY and APPOINT

Antonio Risitano • 16 Apr 2024
EBMT 2024
Anemias Paroxysmal Nocturnal Hemoglobinuria Iptacopan
APPLY-PNH APPOINT-PNH

Antonio Risitano, MD, PhD, AORN San Giuseppe Moscati Di Avellino, Avellino, Italy, presents encouraging data on the anti-complement agent iptacopan for treating paroxysmal nocturnal hemoglobinuria (PNH), as evidenced by the APPLY-PNH (NCT04558918) and APPOINT-PNH (NCT04820530) trials. In the APPOINT trial, sustained responses, with hemoglobin levels nearing normal in some patients, were observed after 48 weeks. Dr Risitano also addresses the issue of breakthrough hemolysis (BTH) associated with complement inhibitors which has been rare and low severity in these trials. This interview took place at the 50th Annual Meeting of the EBMT in Glasgow, Scotland.

These works are owned by Magdalen Medical Publishing (MMP) and are protected by copyright laws and treaties around the world. All rights are reserved.

Disclosures

Novartis: Speaker’s Bureau and Advisory Board.

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