Real-World Effectiveness and Safety of Ravulizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria: Evidence From the International PNH Registry - PubMed
3 days ago
- #Ravulizumab
- #PNH
- #Real-world evidence
- Ravulizumab is a second-generation C5 inhibitor and standard-of-care for PNH.
- Study evaluates real-world effectiveness and safety using data from the International PNH Registry.
- Patients were stratified by prior eculizumab use (experienced vs. naive).
- LDH ratios remained near normal (<1.5× upper limit) in both groups.
- 86.8% of eculizumab-experienced and 76.5% of C5i-naive patients achieved transfusion independence.
- Low rates of major adverse vascular events were reported.
- Findings support ravulizumab as a first-line treatment for PNH.