[How I diagnose and treat paroxysmal nocturnal hemoglobinuria]

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired clonal disorder of hematopoietic stem cells with diverse clinical manifestations, making it susceptible to missed diagnosis or misdiagnosis. Early clinical screening through multidisciplinary collaboration is therefore essential. Treatment strategies should be guided by precise clinical classification. For patients with classic PNH, complement inhibitors are the first-line treatment, whereas for patients with bone marrow failure, the primary treatment should be focused on the underlying bone marrow failure. In cases accompanied by hemolysis, a combination of immunosuppressants and complement inhibitors can be used. During treatment with complement inhibitors, close monitoring of treatment efficacy and complications is required. Based on three representative cases admitted to our hospital, this article highlights the importance of early screening, summarizes key points for standardized diagnosis and treatment of PNH, and provides recommendations for managing breakthrough hemolysis and extravascular hemolysis, with the aim of improving clinical practices.