Highlights on Advances in the Treatment of Paroxysmal Nocturnal Hemoglobinuria From ASH 2024

Ilene Weitz, MD

December 16, 2024

Ilene Weitz, MD, reflects on key data from ASH 2024, including advances in oral and intravenous inhibitors for paroxysmal nocturnal hemoglobinuria (PNH). Notable findings included improvements in quality of life and symptoms with iptacopan in the APPLY-PNH and APPOINT-PNH trials, as well as positive results from the ALPHA trial evaluating danicopan as an add-on to ravulizumab or eculizumab.

Early-phase data on OMS906, a mannose-binding lectin-associated serine protease inhibitor, showed promise for addressing persistent anemia in PNH patients. Additionally, a study from the French group revealed that higher proportions of type 2 neutrophils are linked to increased thrombosis risk, regardless of clone size.

Any views expressed above are the author's own and do not necessarily reflect the views of WebMD or Medscape.

Cite this: Highlights on Advances in the Treatment of Paroxysmal Nocturnal Hemoglobinuria From ASH 2024 - Medscape - Dec 16, 2024.

Comments

Commenting is limited to medical professionals. To comment please Log-in.

Comments on Medscape are moderated and should be professional in tone and on topic. You must declare any conflicts of interest related to your comments and responses. Please see our Commenting Guide for further information. We reserve the right to remove posts at our sole discretion.