Current Landscape of Paroxysmal Nocturnal Hemoglobinuria in the Era of Complement Inhibitors and Regulators

Overview

Journal Ther Adv Hematol

Specialty Hematology

Date 2024 Dec 30

PMID 39734592

Authors

Julia J Shi

Yusuf M Ozcan

Carlos I Ayala Santos

Hetalkumari Patel

Jamile Shammo

Taha Bat

Affiliations

Soon will be listed here.

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.

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