Hemolytic processes in PNH and its treatment: intravascular and extravascular hemolysis

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, life-threatening disease of the blood characterized by destruction of red blood cells (known as hemolysis) by the complement system, a part of the body's innate immune system.

Signs and symptoms of the disorder include fatigue, shortness of breath, abdominal pain, sudden blood clotting, or thrombosis, and—as the disorder’s name suggests—dark, hemoglobin-rich urine.

Current treatment methods involve either bone marrow transplantation or treatment with complement protein inhibitors such as eculizumab, which reduce mortality, improve quality of life and reduce need for transfusions, however the search for a definitive cure continues.