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Paroxysmal nocturnal hemoglobinuria (PNH) is a rare life-threatening and debilitating disorder of hematopoiesis. It is characterized by the clinical triad of Coombs-negative acquired extracorpuscular hemolytic anemia, thrombophilia, and bone marrow failure. The sequelae of intravascular hemolysis drastically affect the quality of life of patients. Cytopenia and in particular thrombophilia can cause life-threatening complications. Thromboembolic events are the major cause of death. The only curative treatment for PNH is allogeneic stem cell transplantation from a related or unrelated stem cell donor. Recent reports demonstrated favorable outcomes. Other treatments were generally supportive in nature. Recently, eculizumab, a humanized monoclonal antibody that inhibits complement factor C5, was approved. It is a targeted, disease-modifying treatment of PNH. This article will summarize major advances in the management of PNH during the last year, and in particular will discuss new results of eculizumab, treatment of thrombosis, and stem cell transplantation. Given the new advances there is hope for improved survival of these patients.

The Management of Paroxysmal Nocturnal Hemoglobinuria—Recent Advances in Diagnosis and Treatment, and New Hope for Patients