Terminal complement inhibitor eculizumab in atypical hemolytic-uremic syndrome
| Autor: | Richard R. Furman, Bruno Moulin, Timothy H.J. Goodship, Sunil Babu, Maria Herthelius, Jens Nürnberger, Neil S. Sheerin, Petra Muus, C. Bedrosian, Rebecca Sberro-Soussan, C. Bingham, Christoph Licht, Yvon Lebranchu, Jan Menne, Christophe Mariat, B. Severino, Frank Eitner, Y. Delmas, Osama Gaber, T. Richard, David J. Cohen, Thorsten Feldkamp, Denis Fouque, Kenneth W. Douglas, Diana Karpman, Chantal Loirat, Maryvonne Hourmant, Masayo Ogawa, Antonella Trivelli, Giuseppe Remuzzi, Ch. Legendre, L.B. Zimmerhackl, Laurence Greenbaum |
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| Rok vydání: | 2013 |
| Předmět: |
Adult
Male medicine.medical_specialty Thrombotic microangiopathy Adolescent medicine.medical_treatment Medizin Renal function Antibodies Monoclonal Humanized Gastroenterology Pediatrics Complement inhibitor Young Adult Translational research [ONCOL 3] Internal medicine Atypical hemolytic uremic syndrome medicine Humans Adverse effect Dialysis Aged Plasma Exchange business.industry Platelet Count Thrombotic Microangiopathies Complement C5 General Medicine Eculizumab Middle Aged medicine.disease Combined Modality Therapy Surgery Clinical trial Hemolytic-Uremic Syndrome Mutation Quality of Life Female Kidney Diseases business medicine.drug |
| Zdroj: | The New England Journal of Medicine, 368, 23, pp. 2169-81 New England Journal of Medicine; 368(23), pp 2169-2181 (2013) The New England Journal of Medicine, 368, 2169-81 |
| ISSN: | 1533-4406 0028-4793 |
| Popis: | Item does not contain fulltext BACKGROUND: Atypical hemolytic-uremic syndrome is a genetic, life-threatening, chronic disease of complement-mediated thrombotic microangiopathy. Plasma exchange or infusion may transiently maintain normal levels of hematologic measures but does not treat the underlying systemic disease. METHODS: We conducted two prospective phase 2 trials in which patients with atypical hemolytic-uremic syndrome who were 12 years of age or older received eculizumab for 26 weeks and during long-term extension phases. Patients with low platelet counts and renal damage (in trial 1) and those with renal damage but no decrease in the platelet count of more than 25% for at least 8 weeks during plasma exchange or infusion (in trial 2) were recruited. The primary end points included a change in the platelet count (in trial 1) and thrombotic microangiopathy event-free status (no decrease in the platelet count of >25%, no plasma exchange or infusion, and no initiation of dialysis) (in trial 2). RESULTS: A total of 37 patients (17 in trial 1 and 20 in trial 2) received eculizumab for a median of 64 and 62 weeks, respectively. Eculizumab resulted in increases in the platelet count; in trial 1, the mean increase in the count from baseline to week 26 was 73x10(9) per liter (P |
| Databáze: | OpenAIRE |
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