Therapeutic goal and drug choice of paroxysmal nocturnal hemoglobinuria
SHI Jun
Abstract：Paroxysmal nocturnal hemoglobinuria(PNH)is a rare non-malignant clonal disease of blood system,and its clinical manifestations are mainly hemolysis,bone marrow failure and thrombosis.In the pre-complement inhibitor era,supportive therapy or allogeneic hematopoietic stem cell transplantation was the main treatment.In 2007,the complement C5 inhibitor came out,which significantly reduced the risk of thrombosis and prolonged the survival of patients.Then,the proximal complement inhibitor blocked the bypass pathway upstream to solve the problem of extravascular hemolysis left by C5 inhibitor.The research and development of oral preparations promotes the chronic disease model with the convenience of medication;Gene therapy is trying to cure the disease at the molecular level.The treatment path of PNH is undergoing constant iterations and breakthroughs,and it is moving towards the goal of"functional cure disease".
Keywords：paroxysmal nocturnal hemoglobinuriatreatmentmedicine
Publication Date：2025-07-01
Online Publishing Date：2025-09-04(First online date of this platform, not the publication date of the document)
Pages：6( 548-553 )