Conference Proceedings

Results from a Phase 3, Multicenter, Non-Inferiority Study of Ravulizumab (ALXN1210) Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with Eculizumab

Austin G Kulasekararaj, Anita Hill, Scott T Rottinghaus, Saskia Langemeijer, Richard A Wells, F Ataulfo Gonzalez-Fernandez, Anna Gaya, Jong-Wook Lee, Emilio Ojeda Gutierrez, Caroline I Piatek, Jeffrey Szer, Antonio M Risitano, Shinji Nakao, Eric Bachman, Lori Shafner, Andrew I Damokosh, Stephan Ortiz, Alexander Roeth, Regis Peffault De latour

BLOOD | AMER SOC HEMATOLOGY | Published : 2018

Abstract

Abstract Introduction Ravulizumab, an innovative complement C5 inhibitor given every 8 weeks (q8w), was recently demonstrated in a large phase 3 study of patients (pts) with paroxysmal nocturnal hemoglobinuria (PNH) and naïve to complement inhibitor therapy to be non-inferior to eculizumab given every 2 weeks (q2w) across all endpoints that measured different aspects of PNH disease (transfusion avoidance [TA], lactate dehydrogenase normalization [LDH-N], percent LDH reduction, breakthrough hemolysis [BTH], Functional Assessment of Chronic Illness Therapy [FACIT]-Fatigue, and hemoglobin stabilization [HGB-S]) (Lee JW et al. EHA Learning Center, Ju..

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