Long-term safety and efficacy of ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: 2-year results from two pivotal phase 3 studies.
Austin G KulasekararajMorag GriffinSaskia LangemeijerKensuke UsukiAlexander Dmitrievich KulaginMasayo OgawaJi YuArshad MujeebuddinJun-Ichi NishimuraJong Wook LeeRégis Peffault de Latournull nullPublished in: European journal of haematology (2022)
This study reports, to date, the longest period of follow-up in over 400 patients with PNH treated with ravulizumab (662 patient-years). Long-term, ravulizumab demonstrated durable efficacy and was well tolerated, highlighting the importance of C5 inhibitors as the mainstay of PNH treatment.