Progress in Paroxysmal Nocturnal Hemoglobinuria Treatment: The Impact of New Data From ASH 2024

Ilene Weitz, MD

January 02, 2025

Ilene Weitz, MD, discusses research on several new therapies for paroxysmal nocturnal hemoglobinuria (PNH) with promising clinical impact presented at ASH 2024. The APPLY-PNH and APPOINT-PNH studies demonstrated that iptacopan significantly improved quality of life and global health status for PNH patients, with notable improvements in cognitive, emotional, physical, and social well-being, particularly at the 48-week mark.

In comparisons between pegcetacoplan (a C3 inhibitor) and danicopan (a factor D inhibitor), breakthrough events were more severe with pegcetacoplan, and danicopan, when used as an add-on to C5 inhibitors such as eculizumab or ravulizumab, resulted in fewer breakthroughs. Iptacopan, an oral agent with a convenient dosing schedule, requires patient compliance to prevent breakthrough hemolysis and manage the risk for hyperlipidemia. Pegcetacoplan is a suitable option for patients comfortable with home infusion, whereas danicopan offers a balance of fewer breakthrough events but necessitates both intravenous infusions and multiple daily pills.

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Cite this: Progress in Paroxysmal Nocturnal Hemoglobinuria Treatment: The Impact of New Data From ASH 2024 - Medscape - Jan 02, 2025.

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