Complement inhibition in medicine: Hematology and beyond; complement inhibition in hematology: PNH and beyond

Abstract

This special issue of the American Journal of Hematology is devoted to complement inhibition in the treatment of various human disorders. It was made possible by support from Apellis. Eight articles in these issues cover the complex complement cascade system from its description to the last update of its inhibition by the most recent targeted therapies, at the terminal and proximal levels.

Biomimetic proteolipid vesicles for reverting GPI deficiency in paroxysmal nocturnal hemoglobinuria

“…PNH, a benign clonal hematological condition within the BMF syndromes, is caused by a somatic mutation in the PIGA gene whose protein is involved in GPI anchor biosynthesis, and is characterized by a failure in membrane anchorage of proteins that require GPI-anchor to correctly localize on cell surface, including endogenous complement regulatory proteins CD55 and CD59. 25 Indeed, PNH symptoms are secondary to complement-induced cell lysis, such as intravascular RBC lysis and thrombosis, leading to anemia and thrombotic events. 26 Thromboembolism is the principal cause of mortality in PNH patients accounting for more than 40% of deaths, and can occur at any site, most commonly involving hepatic (Budd-Chiari syndrome, 7.5–25% of patients affected) and cerebral veins.…”