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Paroxysmal Nocturnal Hemoglobinuria: Unraveling Its Molecular Pathogenesis and Advancing Targeted Therapeutic

Elisavet Apostolidou1, Vasileios Georgoulis1, Dimitrios Leonardos1

  • 1Department of Haematology, Faculty of Medicine, School of Health Sciences, University of Ioannina, St. Niarchou Av., 45 500 Ioannina, Greece.

Diseases (Basel, Switzerland)
|September 26, 2025
PubMed

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired clonal hematologic disorder caused by somatic mutations in the PIGA gene of hematopoietic stem cells, leading to the absence of GPI-anchored proteins, including the complement regulators CD55 and CD59. This deficiency results in uncontrolled complement activation, causing intravascular and extravascular hemolysis, thrombosis, and bone marrow failure. Historically associated with substantial morbidity, PNH management has been transformed by the advent of complement inhibitors. Eculizumab, the first approved C5 inhibitor, significantly reduced thrombotic risk and improved survival but did not eliminate anemia due to extravascular hemolysis. Newer agents now target proximal complement components, offering broader control and improved convenience. This review summarizes the pathophysiology of PNH, evaluates established and emerging complement inhibitors, and discusses ongoing therapeutic challenges and future directions.

Keywords:
breakthrough hemolysiscomplement inhibitorsextravascular hemolysisintravascular hemolysisparoxysmal nocturnal hemoglobinuria

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