Paroxysmal nocturnal hemoglobinuria: Where we stand

Abstract

For the last 20 years, therapy of paroxysmal nocturnal hemoglobinuria (PNH) relied—up until recently—on antibody based terminal complement inhibitionon. PNH pathophysiology—a mutational defect leading to partial or complete absence of complement-regulatory proteins on blood cells—leads to intravascular hemolysis and consequences such as thrombosis and other sequelae. A plethora of new drugs interfering with the proximal and terminal complement cascade are under recent development and the first “proof-of-pinciple” proximal complement inhibitor targeting C3 has been approved in 2021. “PNH: where we stand” will try to give a brief account on where we came from and where we stand focusing on approved therapeutic options. The associated improvements as well as potential consequences of actual and future treatments as well as their impact on the disease will continue to necessitate academic and scientific focus on improving treatment options as well as on side effects and outcomes relevant to individual patient lives and circumstances in order to develop effective, safe, and available treatment for all hemolytic PNH patients globally.