阵发性睡眠性血红蛋白尿症诊断与治疗中国指南(2024年版)

Guidelines for the diagnosis and management of paroxysmal nocturnal hemoglobinuria(2024)

阵发性睡眠性血红蛋白尿症(PNH)是一种罕见的后天获得性造血干细胞克隆性疾病,临床主要表现为血管内溶血、骨髓衰竭和高风险并发血栓等,虽为良性血管内溶血性疾病,严重者可危及生命。近年来,PNH在诊断及治疗等方面均取得较大进展,尤其是补体抑制剂治疗领域。为进一步规范和提高我国PNH的诊治水平,中华医学会血液学分会红细胞疾病(贫血)学组在广泛征集专家建议和意见的基础上,结合阵发性睡眠性血红蛋白尿症(PNH)最新诊治进展、国外相关指南/共识及我国国情对2013年版PNH专家共识进行更新,制订《阵发性睡眠性血红蛋白尿症诊断与治疗中国指南(2024年版)》。

Paroxysmal nocturnal hemoglobinuria(PNH)is a rare acquired clonal disorder of hematopoietic stem cells characterized by intravascular hemolysis,bone marrow failure,and a high risk of thrombosis.Although PNH is a benign intravascular hemolytic disease,severe cases may be life-threatening.In recent years,remarkable progress has been made in the diagnosis and treatment of PNH,particularly in the field of complement inhibitor therapy.To further standardize and improve the diagnosis and treatment level of PNH in China,the Red Blood Cell Disease(Anemia)Group of the Chinese Society of Hematology updated the 2013 PNH Expert Consensus by combining the latest diagnosis and treatment progress of PNH,consulting relevant foreign guidelines/consensus and China’s national conditions,and soliciting expert advice and opinions to formulate the Chinese Guidelines for the Diagnosis and Treatment of Paroxysmal Nocturnal Hemoglobinuria(2024).