Abstract
PNH is a rare, chronic, and potentially fatal disease. It is caused by uncontrolled complement activation resulting in hemolysis, which clinically manifests as anemia, fatigue, and in severe cases, thrombosis. Current treatments (e.g., C3/C5 inhibitors) inhibit complement activation to manage complement-driven hemolysis and associated symptoms. In this analysis, we assessed the clinical effectiveness of pegcetacoplan for real-world PNH patients in the USA.
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