Journal article
Ravulizumab demonstrates long-term efficacy, safety and favorable patient survival in patients with paroxysmal nocturnal hemoglobinuria
A Kulasekararaj, R Brodsky, H Schrezenmeier, M Griffin, A Röth, C Piatek, M Ogawa, J Yu, AS Patel, Y Patel, R Notaro, K Usuki, A Kulagin, S Gualandro, W Füreder, R Peffault de Latour, J Szer, JW Lee
Annals of Hematology | Springer Science and Business Media LLC | Published : 2025
Abstract
Ravulizumab is a second-generation complement component 5 (C5) inhibitor (C5i) approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) following positive results from two pivotal trials in patients with PNH originally naive to C5i treatment and eculizumab-experienced patients with PNH. In both trials, after the 26week primary evaluation period, all patients received ravulizumab for up to 6 years. To report ravulizumab treatment outcomes in patients with PNH originally naive to C5i treatment and eculizumab-experienced patients with PNH treated for up to 6 years. Originally C5i-naive (N = 244) and eculizumab-experienced (N = 191) patients with PNH continued ravulizumab treatmen..
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