One-year outcomes from a phase 3 randomized trial of ravulizumab in adults with paroxysmal nocturnal hemoglobinuria who received prior eculizumab
| العنوان: | One-year outcomes from a phase 3 randomized trial of ravulizumab in adults with paroxysmal nocturnal hemoglobinuria who received prior eculizumab |
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| المؤلفون: | Austin G. Kulasekararaj, Emilio Gutierrez, Stephan Ortiz, F. Ataúlfo González Fernández, Régis Peffault de Latour, Ji Yu, Saskia Langemeijer, Kensuke Usuki, Anita J. Hill, Anna Gaya, Robert A. Brodsky, Alberto Bosi, Richard A. Wells, Lindsay Mitchell, Caroline I. Piatek, Alexander Röth, Masayo Ogawa, Jong Wook Lee |
| المصدر: | European Journal of Haematology, 106, 3, pp. 389-397 European Journal of Haematology European Journal of Haematology, 106, 389-397 |
| سنة النشر: | 2021 |
| مصطلحات موضوعية: | Adult, Male, paroxysmal nocturnal hemoglobinuria, Hemoglobinuria, Paroxysmal, Medizin, Antibodies, Monoclonal, Humanized, Hemolysis, law.invention, 03 medical and health sciences, chemistry.chemical_compound, Complement inhibitor, Cancer development and immune defence Radboud Institute for Health Sciences [Radboudumc 2], 0302 clinical medicine, Randomized controlled trial, Quality of life, breakthrough hemolysis, law, Lactate dehydrogenase, Medicine, Humans, In patient, Blood Transfusion, Molecular Targeted Therapy, Adverse effect, transfusion, business.industry, complement inhibitor, Complement C5, Hematology, General Medicine, Original Articles, Eculizumab, hemoglobin, medicine.disease, Combined Modality Therapy, Complement Inactivating Agents, Treatment Outcome, chemistry, quality of life, 030220 oncology & carcinogenesis, Anesthesia, Retreatment, Paroxysmal nocturnal hemoglobinuria, Female, Original Article, business, 030215 immunology, medicine.drug |
| الوصف: | Contains fulltext : 245091.pdf (Publisher’s version ) (Open Access) 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 |
| وصف الملف: | application/pdf |
| اللغة: | English |
| تدمد: | 0902-4441 |
| URL الوصول: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6a2a0e5023a2735533b062dba1a8be86 https://www.ncbi.nlm.nih.gov/pubmed/33301613 |
| Rights: | OPEN |
| رقم الانضمام: | edsair.doi.dedup.....6a2a0e5023a2735533b062dba1a8be86 |
| قاعدة البيانات: | OpenAIRE |
| تدمد: | 09024441 |
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