1. Current landscape of paroxysmal nocturnal hemoglobinuria in the era of complement inhibitors and regulators.
- Author
-
Shi JJ, Ozcan YM, Santos CIA, Patel H, Shammo J, and Bat T
- Abstract
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disorder which is caused by mutations in phosphatidylinositol glycan class A leading to hemolysis of red blood cells via complement inhibition. The first treatment for PNH, eculizumab, was FDA approved in 2007. Since then, many new treatment options for PNH have arisen. This critical review will examine all medications available for PNH on the US market, highlight several major medications in development, and discuss the risks and treatment considerations associated with each option. It is not intended to address PNH clonal dynamics, disease presentation, or discussions on when to initiate treatment., Competing Interests: Julia J. Shi, Yusuf M. Ozcan, Carlos I. Ayala Santos, Hetalkumari Patel: None. Jamile Shammo: Consultancy: Alexion, Astra Zeneca, Apellis Pharmaceutical, BMS, CTI, Celgene, Sobi, GSK, Incyte, MJH, Novartis, Sanofi; Equity Ownership: AbbVie, Baxter; Research Funding: Alexion, Astra Zeneca, Incyte, Novartis, Protagonist Therapeutics; Speakers Bureau: Alexion, Sanofi; Membership on a Board or Advisory Committee: Alexion, Apellis; Pharmaceutical, GSK, MJH, Novartis; Honoraria: Alexion, Astra Zeneca, Apellis, BMS, Celgene, CTI Pharma, Sobi, GSK, Incyte, MJH, Novartis, Sanofi. Taha Bat: Membership on Alexion Board of Directors or Advisory Committees., (© The Author(s), 2024.)
- Published
- 2024
- Full Text
- View/download PDF