[Interpretation of the guidelines for diagnosing and treating paroxysmal nocturnal hemoglobinuria in China (2024)]

【中国阵发性睡眠性血红蛋白尿症诊疗指南(2024年版)解读】

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Abstract

Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal disorder of hematopoietic stem cells induced by PIG-A gene mutations. It is clinically manifested by hemolysis, bone marrow failure, and high-risk concurrent thrombosis, which are life-threatening in severe cases. Significant progress has been made in the pathogenesis research and clinical diagnosis and treatment of PNH in recent years. The Red Blood Cell Disease (Anemia) Group Chinese Society of Hematology, Chinese Medical Association, combined with the latest diagnosis and treatment progress of PNH, relevant foreign guidelines/consensus, and China's national conditions, jointly formulated the "Guidelines for the diagnosis and management of paroxysmal nocturnal hemoglobinuria (2024) " based on extensive solicitation of expert suggestions and opinions. This article discussed the key and difficult issues in PNH diagnosis and treatment, interpreted the updated part of the guidelines, and expanded the relevant recommendations of the guidelines according to the latest research progress at home and abroad, thereby providing more references for clinical practice.

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