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PQI: Pegcetacoplan (Empaveli®) Use in Paroxysmal Nocturnal Hemoglobinuria

Pegcetacoplan (Empaveli®) Use in Paroxysmal Nocturnal Hemoglobinuria

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Last Updated: November 29, 2023

By: Michael Verbosky, PharmD, BCOP, BCPS | Matt Snyder, PharmD, BCOP | Timothy Kubal, MD, MBA

About this PQI

Pegcetacoplan is a proximal complement inhibitor indicated for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare, acquired condition caused by dysregulation of the complement pathway leading to hemolysis, fatigue, thrombosis, organ dysfunction and bone marrow hypocellularity. This PQI will focus on the role of pegcetacoplan in the management of PNH.

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