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Eculizumab Safety: Five-Year Experience From the Global Atypical Hemolytic Uremic Syndrome Registry

INTRODUCTION: Eculizumab has transformed outcomes for patients with atypical hemolytic uremic syndrome (aHUS). Its efficacy and safety profile was well characterized in the clinical trial program. The long-term safety profile was not previously assessed or compared against nontreated patients in an...

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Autores principales: Rondeau, Eric, Cataland, Spero R., Al-Dakkak, Imad, Miller, Benjamin, Webb, Nicholas J.A., Landau, Daniel
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6933459/
https://www.ncbi.nlm.nih.gov/pubmed/31890998
http://dx.doi.org/10.1016/j.ekir.2019.07.016
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author Rondeau, Eric
Cataland, Spero R.
Al-Dakkak, Imad
Miller, Benjamin
Webb, Nicholas J.A.
Landau, Daniel
author_facet Rondeau, Eric
Cataland, Spero R.
Al-Dakkak, Imad
Miller, Benjamin
Webb, Nicholas J.A.
Landau, Daniel
author_sort Rondeau, Eric
collection PubMed
description INTRODUCTION: Eculizumab has transformed outcomes for patients with atypical hemolytic uremic syndrome (aHUS). Its efficacy and safety profile was well characterized in the clinical trial program. The long-term safety profile was not previously assessed or compared against nontreated patients in an observational registry setting. METHODS: The Global aHUS Registry recruits patients with clinical diagnoses of aHUS. This analysis includes baseline characteristics and targeted safety events from adult and pediatric patients who were “ever treated” versus “never treated” with eculizumab in the first 5 years of the registry, through January 26, 2017. RESULTS: Overall, 1321 patients (adult, n = 842; pediatric, n = 479; ever treated, n = 865; never treated, n = 456) were enrolled. A higher proportion of ever-treated versus never-treated adult and pediatric patients had renal, cardiovascular, pulmonary, central nervous system, gastrointestinal symptoms, and hepatic impairment. No differences in safety event rates between ever-treated and never-treated patients were observed, except serious infections in pediatric patients (5.15 versus 1.12 events/100 patient-years for ever- and never-treated patients, respectively). Deaths were more frequent in adult (4.7% and 9.9% of ever- and never-treated patients) compared with pediatric patients (1.8% of ever-treated patients; no deaths in never-treated patients).Three meningococcal infections were reported in ever-treated patients; 1 infection led to a fatal outcome. CONCLUSION: In this large observational dataset covering 5 years of registry enrollment, no new safety concerns were identified for adult or pediatric eculizumab-treated patients with aHUS, confirming a positive benefit−risk profile in a real-world setting.
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spelling pubmed-69334592019-12-30 Eculizumab Safety: Five-Year Experience From the Global Atypical Hemolytic Uremic Syndrome Registry Rondeau, Eric Cataland, Spero R. Al-Dakkak, Imad Miller, Benjamin Webb, Nicholas J.A. Landau, Daniel Kidney Int Rep Clinical Research INTRODUCTION: Eculizumab has transformed outcomes for patients with atypical hemolytic uremic syndrome (aHUS). Its efficacy and safety profile was well characterized in the clinical trial program. The long-term safety profile was not previously assessed or compared against nontreated patients in an observational registry setting. METHODS: The Global aHUS Registry recruits patients with clinical diagnoses of aHUS. This analysis includes baseline characteristics and targeted safety events from adult and pediatric patients who were “ever treated” versus “never treated” with eculizumab in the first 5 years of the registry, through January 26, 2017. RESULTS: Overall, 1321 patients (adult, n = 842; pediatric, n = 479; ever treated, n = 865; never treated, n = 456) were enrolled. A higher proportion of ever-treated versus never-treated adult and pediatric patients had renal, cardiovascular, pulmonary, central nervous system, gastrointestinal symptoms, and hepatic impairment. No differences in safety event rates between ever-treated and never-treated patients were observed, except serious infections in pediatric patients (5.15 versus 1.12 events/100 patient-years for ever- and never-treated patients, respectively). Deaths were more frequent in adult (4.7% and 9.9% of ever- and never-treated patients) compared with pediatric patients (1.8% of ever-treated patients; no deaths in never-treated patients).Three meningococcal infections were reported in ever-treated patients; 1 infection led to a fatal outcome. CONCLUSION: In this large observational dataset covering 5 years of registry enrollment, no new safety concerns were identified for adult or pediatric eculizumab-treated patients with aHUS, confirming a positive benefit−risk profile in a real-world setting. Elsevier 2019-08-02 /pmc/articles/PMC6933459/ /pubmed/31890998 http://dx.doi.org/10.1016/j.ekir.2019.07.016 Text en © 2019 International Society of Nephrology. Published by Elsevier Inc. http://creativecommons.org/licenses/by-nc-nd/4.0/ This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).
spellingShingle Clinical Research
Rondeau, Eric
Cataland, Spero R.
Al-Dakkak, Imad
Miller, Benjamin
Webb, Nicholas J.A.
Landau, Daniel
Eculizumab Safety: Five-Year Experience From the Global Atypical Hemolytic Uremic Syndrome Registry
title Eculizumab Safety: Five-Year Experience From the Global Atypical Hemolytic Uremic Syndrome Registry
title_full Eculizumab Safety: Five-Year Experience From the Global Atypical Hemolytic Uremic Syndrome Registry
title_fullStr Eculizumab Safety: Five-Year Experience From the Global Atypical Hemolytic Uremic Syndrome Registry
title_full_unstemmed Eculizumab Safety: Five-Year Experience From the Global Atypical Hemolytic Uremic Syndrome Registry
title_short Eculizumab Safety: Five-Year Experience From the Global Atypical Hemolytic Uremic Syndrome Registry
title_sort eculizumab safety: five-year experience from the global atypical hemolytic uremic syndrome registry
topic Clinical Research
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6933459/
https://www.ncbi.nlm.nih.gov/pubmed/31890998
http://dx.doi.org/10.1016/j.ekir.2019.07.016
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