Case report: Transfusion independence and abolition of extravascular hemolysis in a PNH patient treated with pegcetacoplan

• DOI: 10.3389/fimmu.2022.1060923
• Publication Date: 2022-12-01T17:54:18Z
• Authors: Bruno Fattizzo; Francesco Versino; Anna Zaninoni; Anna Paola Maria ...; Cristina Vercellati; Silvia Artuso; Wilma Barcellini
• Keywords: Eculizumab; Paroxysmal nocturnal hemoglobinuria; Hemoglobinuria; Ineffective erythropoiesis

More than half of patients with paroxysmal nocturnal hemoglobinuria (PNH) treated complement fraction C5 inhibitors experience residual anemia and hemolysis. This is partly due to the persistent activation cascade upstream C5, resulting in C3 deposition on PNH erythrocytes extravascular hemolysis reticuloendothelial system. Pegcetacoplan first proximal inhibitor be approved for basing favorable efficacy safety data both naïve eculizumab PNH. Here we report Italian patient pegcetacoplan a named program. The suffered from hemolytic associated CALR+ myeloproliferative neoplasm was heavily transfusion dependent despite therapy. Treatment induced dramatic improvement Hb, along normalization unconjugated bilirubin reticulocytes, as markers Sequential laboratory workup showed disappearance by direct anti-globulin test, increase clone erythrocytes, peculiar right shift ektacytometry curve. drug well tolerated, reported significant his quality life. Overall, appears safe effective option “ready use” clinic suboptimal response anti-C5 agents.