Long-term outcomes of patients with paroxysmal nocturnal hemoglobinuria treated with eculizumab in a real-world setting.

Objective: Describe the real-world clinical profile of eculizumab-treated patients by characterizing their short- and long-term clinical and laboratory outcomes.

Methods: This retrospective study used preexisting medical records of eculizumab-treated patients with paroxysmal nocturnal hemoglobinuria (PNH) at the University Hospital Essen. Hematologic response, breakthrough hemolysis, transfusion dependence, and other outcomes were assessed.