Consensus of the Hematology Society of Taiwan on the management of paroxysmal nocturnal hemoglobinuria (PNH)

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare complement-mediated disorder characterized by the clonal expansion of hematopoietic stem cells with mutations affecting genes involved in glycosylphosphatidylinositol anchor synthesis. Bone marrow dysfunction, unexplained thrombosis, and hemolysis are cardinal features of the disease. However, clinical presentations can vary, ranging from classic hemolytic PNH to non-hemolytic forms, with or without signs and symptoms of bone marrow failure. Advances in understanding the underlying pathophysiology, along with the development and use of complement inhibition therapy, have significantly improved the prognosis and quality of life of patients. Nonetheless, managing PNH remains challenging due to its rarity and the heterogeneous nature of its presentation. To provide local physicians with practical guidance and promote high-quality care, the Hematology Society of Taiwan developed a consensus on the diagnosis, screening, assessment, monitoring, and treatment for PNH patients, incorporating the latest evidence and local context.