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dc.contributorVall d'Hebron Barcelona Hospital Campus
dc.contributor.authorBarbour, Thomas
dc.contributor.authorScully, Marie
dc.contributor.authorAriceta Iraola, Gema
dc.contributor.authorCataland, Spero
dc.contributor.authorGarlo, Katherine
dc.contributor.authorHeyne, Nils
dc.date.accessioned2022-03-21T09:06:30Z
dc.date.available2022-03-21T09:06:30Z
dc.date.issued2021-03-24
dc.identifier.citationBarbour T, Scully M, Ariceta G, Cataland S, Garlo K, Heyne N, et al. Long-Term Efficacy and Safety of the Long-Acting Complement C5 Inhibitor Ravulizumab for the Treatment of Atypical Hemolytic Uremic Syndrome in Adults. Kidney Int Reports. 2021 Mar 24;6(6):1603–13.
dc.identifier.issn2468-0249
dc.identifier.urihttp://hdl.handle.net/11351/7217
dc.descriptionHemolytic uremic syndrome; Kidney failure; Ravulizumab
dc.description.abstractIntroduction Atypical hemolytic uremic syndrome (aHUS) is a rare, complex, multisystem disease of dysregulated complement activity, characterized by progressive thrombotic microangiopathy (TMA), acute kidney injury, and multiorgan dysfunction, which often progresses to chronic kidney disease. Results from the prospective clinical trial of ravulizumab (NCT02949128) reveal rapid resolution of TMA in patients with aHUS, with sustained efficacy and safety in a 26-week initial evaluation period. Methods The aim of this analysis was to characterize the long-term efficacy and the safety profile of ravulizumab in adults with aHUS who had completed the initial evaluation period of the trial. Complete TMA response, hematologic and kidney functions, and safety were evaluated for all patients available for follow-up in the extension period (median follow-up: 76.7 weeks; range: 0.6–118.3). This trial included a total of 58 patients, 49 of whom entered the extension period. Results A total of 4 additional patients achieved complete TMA response during the follow-up period. Normalization of platelet count, serum lactate dehydrogenase (LDH), and hemoglobin observed in the 26-week initial evaluation period was sustained until the last available follow-up, as were the improvements in the estimated glomerular filtration rate (eGFR) and patient quality of life. All efficacy endpoints were correlated with the sustained inhibition of complement C5. Most adverse events (AEs) occurred early during the initial evaluation period and decreased substantially during the extension period. No patient developed a meningococcal infection or died during the extension period. Conclusion This analysis reveals that ravulizumab administered every 8 weeks is efficacious with an acceptable safety profile for the long-term treatment of adults with aHUS and provides additional clinical benefit beyond 6 months of treatment.
dc.language.isoeng
dc.publisherElsevier
dc.relation.ispartofseriesKidney International Reports;6(6)
dc.rightsAttribution-NonCommercial-NoDerivatives 4.0 International
dc.rights.urihttp://creativecommons.org/licenses/by-nc-nd/4.0/
dc.sourceScientia
dc.subjectSíndrome hemolíticourèmica - Tractament
dc.subjectAvaluació de resultats (Assistència sanitària)
dc.subjectMalalties rares
dc.subject.meshAtypical Hemolytic Uremic Syndrome
dc.subject.mesh/drug therapy
dc.subject.meshTreatment Outcome
dc.subject.meshRare Diseases
dc.titleLong-Term Efficacy and Safety of the Long-Acting Complement C5 Inhibitor Ravulizumab for the Treatment of Atypical Hemolytic Uremic Syndrome in Adults
dc.typeinfo:eu-repo/semantics/article
dc.identifier.doi10.1016/j.ekir.2021.03.884
dc.subject.decssíndrome hemolítico urémico atípico
dc.subject.decs/farmacoterapia
dc.subject.decsresultado del tratamiento
dc.subject.decsenfermedades raras
dc.relation.publishversionhttps://doi.org/10.1016/j.ekir.2021.03.884
dc.type.versioninfo:eu-repo/semantics/publishedVersion
dc.audienceProfessionals
dc.contributor.organismesInstitut Català de la Salut
dc.contributor.authoraffiliation[Barbour T] Kidney Care, The Royal Melbourne Hospital, Melbourne, Australia. [Scully M] Department of Haematology, University College London Hospitals, London, UK. [Ariceta G] Servei de Nefrologia Pediàtrica, Vall d’Hebron Hospital Universitari, Barcelona, Spain. [Cataland S] Division of Hematology, The Ohio State University Medical Center, Columbus, Ohio, USA. [Garlo K] Clinical Development, Alexion Pharmaceuticals, Inc., Boston, Massachusetts, USA. [Heyne N] Section of Nephrology and Hypertension, Tübingen University Hospital, Tübingen, Germany
dc.identifier.pmid34169200
dc.identifier.wos000661053500016
dc.rights.accessrightsinfo:eu-repo/semantics/openAccess


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