Real-World Effectiveness and Safety of Ravulizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria: Evidence From the International PNH Registry

Abstract

Ravulizumab, a second-generation complement component 5 inhibitor (C5i) derived from eculizumab, with improved pharmacokinetics, is the current standard-of-care treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH), where available. Pivotal trials have demonstrated durable long-term efficacy, safety, and improved survival, and increasing real-world evidence is required. This analysis utilized data from the International PNH Registry (NCT01374360) to evaluate the real-world effectiveness and safety of ravulizumab, and to assess baseline characteristics at treatment initiation. Adults (aged 18–65 years) enrolled in the registry as of July 1, 2024, who were treated with ravulizu..