Changes in Hemoglobin Measures Observed in PNH Patients Treated with Both C5 Inhibitors Ravulizumab and Eculizumab: Real-World Evidence from a US-Based EMR Network

Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired blood disease characterized by hemolytic anemia, bone marrow failure, and thrombosis. Prior to the FDA approval of the C3 inhibitor pegcetacoplan, PNH treatment only included the C5 inhibitors (C5i) ravulizumab (RAV) and eculizumab (ECU) to decrease intravascular hemolysis and improve anemia. A Phase III trial of RAV in a treatment-naïve PNH population reported hemoglobin (Hb) stabilization (avoidance a >2 g/dL decrease in baseline Hb) occurred in 68% of RAV and 64% of ECU patients after 6 months. (Lee et al, 2019). However, real world (RW) evidence of the efficacy of these agents on Hb measures in clinical practice are lacking.