Sergio Di Matteo, Roberto Freilone, Giacomo Matteo Bruno, Rosario Notaro, Sabrin Moumene, Nicoletta Martone, Cristina Teruzzi, Antonio Ciccarone, Giorgio Lorenzo Colombo
BACKGROUND: Paroxysmal nocturnal hemoglobinuria is a rare, acquired disease characterized by hemolytic episodes and associated with significant clinical burden. The introduction of C5 inhibitory monoclonal antibodies (C5i) represented a major breakthrough in PNH treatment, effectively reducing intravascular hemolysis (IVH) but showing limited impact on extravascular hemolysis (EVH). In 2021, the C3 inhibitor pegcetacoplan was approved by EMA and recently reimbursed in Italy, which also has the advantages in the reduction of both IVH and EVH, increasing hemoglobin values and simultaneously improving the quality of life and fatigue of patients...
2024: ClinicoEconomics and Outcomes Research: CEOR