Abstract
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease with the most prominent symptoms being hemolytic anemia, hemoglobinuria, thrombosis and fatigue. Complement C5 inhibition with eculizumab (Ecu) or ravulizumab (Ravu) is the current standard of care. Despite improvements in morbidity and mortality, some patients continue experiencing hemolysis and suboptimal quality of life (QoL). This study aimed at investigating the symptom burden of PNH in patients currently treated with a C5 inhibitor, globally.
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