Journal article

Effect of Pegcetacoplan on Quality of Life in Patients with Paroxysmal Nocturnal Hemoglobinuria from the Pegasus Phase 3 Trial Comparing Pegcetacoplan to Eculizumab

Alexander Röth, Britta Hoechsmann, Morag Griffin, Carlos M de Castro, Jeffrey Szer, Kensuke Usuki, Juliette Soret, Mohamed Hamdani, Temitayo Ajayi, Sujata P Sarda, Jens Panse

Blood | American Society of Hematology | Published : 2020


INTRODUCTION Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired, rare, clonal, nonmalignant hematologic disease characterized by complement-mediated red blood cell hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes, and/or some degree of bone marrow dysfunction. The current standard treatment for patients with PNH is eculizumab (ECU) or ravulizumab, both C5 inhibitors. Despite the proven efficacy of anti-C5 therapy in the control of intravascular hemolysis, 72% of patients have persistent hemolytic anemia despite ECU treatment and 36% require transfusions, resulting in significant impact on quality of life (QoL) including persist..

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