Abstract
The targeted terminal complement inhibitor eculizumab (Soliris) reduces intravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria (PNH), as well as stabilizing hemoglobin levels, improving fatigue and health-related quality of life, and reducing the requirement for packed red cell transfusions. Eculizumab is generally well tolerated in patients with PNH, although the risk of Neisseria meningitidis infection is increased with eculizumab, meaning that patients must be vaccinated.
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