Cost per Responder Analysis for Pegcetacoplan and Eculizumab in the Treatment of Adults with Paroxysmal Nocturnal Hemoglobinuria

Background: In the absence of clinical guidelines for paroxysmal nocturnal hemoglobinuria (PNH), the European Society for Blood and Marrow Transplantation (EBMT) developed categories for classifying hematological response to complement inhibitor treatment (per Risitano AM, et al. Front Immunol. 2019;10:1157). These categories are: complete (no transfusions, normal stable Hb, and no evidence of hemolysis); major (no transfusions, normal Hb, but evidence of residual intravascular/extravascular hemolysis); good (no transfusions, but persistent chronic mild anemia); partial (persistent chronic moderate anemia and/or occasional red blood cell transfusions); minor (3-6 transfusions every 6 months); no response (>6 transfusions every 6 months). In the phase 3 PEGASUS trial (NCT03500549), 41 and 39 patients previously treated with C5 inhibitors with Hb