One-year outcomes from a phase 3 randomized trial of ravulizumab in adults with paroxysmal nocturnal hemoglobinuria who received prior eculizumab.

Autor: Kulasekararaj AG; 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., Hill A; Department of Haematology, Leeds Teaching Hospitals, Leeds, UK., Langemeijer S; Radboudumc, Nijmegen, the Netherlands., Wells R; Sunnybrook Health Sciences Centre, Toronto, ON, Canada., González Fernández FA; Department of Hematology, Hospital Clinico Universitario San Carlos, The Clınic Institute of Haematological and Oncological Diseases, Madrid, Spain., Gaya A; Department of Hematology, Hospital Clinic de Barcelona, Barcelona, Spain., Ojeda Gutierrez E; Hospital Universitario Puerta de Hierro Majadahonda, Madrid, Spain., Piatek CI; Jane Anne Nohl Division of Hematology, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA., Mitchell L; Monklands Hospital, Airdrie, Scotland, UK., Usuki K; NTT Medical Center Tokyo, Tokyo, Japan., Bosi A; Department of Experimental and Clinical Medicine, University of Florence, Florence, Italy., Brodsky RA; The John Hopkins University School of Medicine, Baltimore, MD, USA., Ogawa M; Alexion Pharmaceuticals, Inc., Boston, MA, USA., Yu J; Alexion Pharmaceuticals, Inc., Boston, MA, USA., Ortiz S; Alexion Pharmaceuticals, Inc., Boston, MA, USA., Röth A; Department of Hematology, West German Cancer Center, University Hospital Essen, Essen, Germany., Lee JW; Department of Hematology, Seoul St. Mary's Hospital, The Catholic University of Korea, Seoul, Republic of Korea., Peffault de Latour R; Bone Marrow Transplantation Unit, Saint Louis Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France.; French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Saint Louis Hospital and University Paris Diderot, Paris, France.; Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation, Leiden, the Netherlands.
Jazyk: angličtina
Zdroj: European journal of haematology [Eur J Haematol] 2021 Mar; Vol. 106 (3), pp. 389-397. Date of Electronic Publication: 2021 Jan 03.
DOI: 10.1111/ejh.13564
Abstrakt: Ravulizumab 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.)
Databáze: MEDLINE
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