阵发性睡眠性血红蛋白尿症的治疗进展

Progress in treatment of paroxysmal nocturnal hemoglobinuria

阵发性睡眠性血红蛋白尿症(PNH)是一种罕见的血液系统疾病,其临床主要表现为溶血性贫血、骨髓衰竭和血栓形成。按国际PNH兴趣小组(I-PIG)提出的诊断分类,PNH可分为经典型、合并其他骨髓衰竭疾病的PNH及亚临床型PNH。在我国,对于合并骨髓衰竭的患者,主要的治疗为免疫抑制剂联合造血刺激因子或促血小板生成素受体激动剂(TPO-RA),而经典型PNH,我国使用最为广泛的治疗是糖皮质激素、输血、铁剂、抗凝、造血干细胞移植及化疗。文章将就PNH的治疗进展做一简要介绍。

Paroxysmal nocturnal hemoglobinuria(PNH)is a rare disease of blood system,and its clinical manifestations are hemolytic anemia,bone marrow failure and thrombosis.According to the diagnostic classification put forward by the International PNH Interest Group(I-PIG),PNH can be divided into classic PNH,PNH with other bone marrow failure diseases and subclinical PNH.In China,for patients with bone marrow failure,the main treatment is immunosuppressant combined with hematopoietic stimulating factor or thrombopoietin receptor agonist(TPO-RA),while the most widely used treatment for classic PNH in China is glucocorticoid,blood transfusion,iron,anticoagulation,hematopoietic stem cell transplantation and chemotherapy.This article will briefly introduce the treatment progress of PNH.