• Austin G Kulasekararaj, Anita Hill, Saskia Langemeijer, Richard Wells, F Ataúlfo González Fernández, Anna Gaya, Ojeda GutierrezEmilioEHospital Universitario Puerta de Hierro Majadahonda, Madrid, Spain., Caroline I Piatek, Lindsay Mitchell, Kensuke Usuki, Alberto Bosi, Robert A Brodsky, Masayo Ogawa, Ji Yu, Stephan Ortiz, Alexander Röth, Jong Wook Lee, and Régis Peffault de Latour.
• Department of Haematological Medicine, King's College Hospital, National Institute of Health Research/Wellcome King's Clinical Research Facility and King's College London, London, UK.
• Eur. J. Haematol. 2021 Mar 1; 106 (3): 389-397.

AbstractRavulizumab every 8 weeks showed non-inferiority to eculizumab every 2 weeks in a 26-week, phase 3, randomized controlled trial in adults with paroxysmal nocturnal hemoglobinuria (PNH) who were clinically stable on eculizumab (NCT03056040). We report results from the first 26 weeks of the extension period in which patients continued ravulizumab (n = 96) or switched from eculizumab to ravulizumab (n = 95). At week 52, mean (SD) lactate dehydrogenase levels increased 8.8% (29%) with ravulizumab-ravulizumab and 5.8% (27%) with eculizumab-ravulizumab from primary evaluation period baseline. During the extension period, four patients (ravulizumab-ravulizumab, n = 3; eculizumab-ravulizumab, n = 1) experienced breakthrough hemolysis, but none associated with serum free C5 ≥ 0.5 μg/mL. Mean Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scores remained stable through week 52. During the extension period, proportions of patients avoiding transfusion remained stable (ravulizumab-ravulizumab, 86.5%; eculizumab-ravulizumab, 83.2%); 81.2% and 81.1%, respectively, had stabilized hemoglobin. All patients maintained serum free C5 levels < 0.5 μg/mL. Adverse events were generally similar between groups, and rates were lower in the extension period. Adults with PNH on stable eculizumab therapy who received ravulizumab over 52 weeks experienced durable efficacy, with consistent efficacy in patients who received eculizumab during the primary evaluation period and then switched to ravulizumab. Ravulizumab was well tolerated.© 2020 The Authors. European Journal of Haematology published by John Wiley & Sons Ltd.

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