Long-term safety and efficacy of ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: 2-year results from two pivotal phase 3 studies

Kulasekararaj, Austin GAustin GKulasekararajGriffin, MoragMoragGriffinLangemeijer, SaskiaSaskiaLangemeijerUsuki, KensukeKensukeUsukiKulagin, AlexanderAlexanderKulaginOgawa, MasayoMasayoOgawaYu, JiJiYuMujeebuddin, ArshadArshadMujeebuddinNishimura, Jun-IchiJun-IchiNishimuraLee, Jong WookJong WookLeePeffault de Latour, RégisRégisPeffault de LatourCHIEN-CHIN LIN301/302 Study Group2023-05-222023-05-222022-090902-44411600-0609https://scholars.lib.ntu.edu.tw/handle/123456789/631248The complement component 5 (C5) inhibitor ravulizumab demonstrated non-inferiority to eculizumab following 26 weeks of treatment in complement inhibitor-naïve and complement inhibitor-experienced patients with paroxysmal nocturnal hemoglobinuria (PNH; studies 301 and 302, respectively). This study aims to describe the results of both studies from 27 weeks to 2 years.enbreakthrough hemolysis; complement inhibitor; lactate dehydrogenase; paroxysmal nocturnal hemoglobinuria; ravulizumabLong-term safety and efficacy of ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: 2-year results from two pivotal phase 3 studiesjournal article10.1111/ejh.13783355026002-s2.0-85133664985WOS:000811637300001https://api.elsevier.com/content/abstract/scopus_id/85133664985