中国实用内科杂志2025，Vol.45Issue(7)：548-553,6.DOI:10.19538/j.nk2025070102
阵发性睡眠性血红蛋白尿症治疗目标与药物选择
Therapeutic goal and drug choice of paroxysmal nocturnal hemoglobinuria
施均1
作者信息
- 1. 中国医学科学院血液病医院(中国医学科学院血液学研究所),天津 300020
- 折叠
摘要
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".关键词
阵发性睡眠性血红蛋白尿症/治疗/药物Key words
paroxysmal nocturnal hemoglobinuria/treatment/medicine分类
医药卫生引用本文复制引用
施均..阵发性睡眠性血红蛋白尿症治疗目标与药物选择[J].中国实用内科杂志,2025,45(7):548-553,6.
