![]() Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired disease characterized by chronic intravascular hemolysis caused by uncontrolled complement activation. This international registry will provide an ongoing, valuable resource to further the clinical understanding of paroxysmal nocturnal hemoglobinuria. Patients suffered from poor quality of life 23% of patients had been hospitalized due to paroxysmal nocturnal hemoglobinuria-related complications and 17% stated that paroxysmal nocturnal hemoglobinuria was the reason they were not working or were working less. Frequently reported symptoms included fatigue (80%), dyspnea (64%), hemoglobinuria (62%), abdominal pain (44%), and chest pain (33%). Therapies included anticoagulation (31%), immunosuppression (19%), and eculizumab (25%). Overall, 16% of patients had a history of thrombotic events and 14% a history of impaired renal function. Median granulocyte paroxysmal nocturnal hemoglobinuria clone size was 68.1% (range 0.01–100%). Here we report the characteristics of the first 1610 patients enrolled. In addition to evaluating the long-term safety and effectiveness of eculizumab in a global population, the registry aims to improve diagnosis, optimize patient management and outcomes, and enhance the understanding of the natural history of paroxysmal nocturnal hemoglobinuria. The International PNH Registry is a worldwide, observational, non-interventional study collecting safety, effectiveness, and quality-of-life data from patients with a confirmed paroxysmal nocturnal hemoglobinuria diagnosis or detectable paroxysmal nocturnal hemoglobinuria clone, irrespective of treatment. ![]() Paroxysmal nocturnal hemoglobinuria is a rare, acquired disease associated with hemolytic anemia, bone marrow failure, thrombosis, and, frequently, poor quality of life.
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