Long-term follow-up shows ravulizumab effective in paroxysmal nocturnal hemoglobinuria
In this study, 441 patients from 2 pivotal phase 3 studies comparing ravulizumab to eculizumab in complement inhibitor-naïve (n = 301) and complement inhibitor-experienced (n = 302) patients with paroxysmal nocturnal hemoglobinuria after 26 weeks of treatment, continued treatment with ravulizumab throughout the extension period.
During the extension period, improvements in lactate dehydrogenase were maintained in each group.
Transfusion avoidance was maintained in 81.8% in the complement inhibitor-naïve group and 85.6% in the complement inhibitor-experienced group. FACIT-F scores remained stable.
Adverse events rates were similar in both studies and serious adverse events related to treatment with ravulizumab was 3%. Overall, ravulizumab was considered well tolerated.
Kulasekararaj AG, Griffin M, Langemeijer S, et al. Long-term safety and efficacy of ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: 2-year results from two pivotal phase 3 studies. Eur J Haematol. 2022;doi: 10.1111/ejh.13783. Epub ahead of print. PMID: 35502600.
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