Long-Term Efficacy and Safety of the Long-Acting Complement C5 Inhibitor Ravulizumab for the Treatment of Atypical Hemolytic Uremic Syndrome in Adults

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Introduction: 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.

Original languageEnglish
Pages (from-to)1603-1613
Number of pages11
JournalKidney International Reports
Issue number6
Publication statusPublished - 2021 Jun

Bibliographical note

Funding Information:
Medical writing support was funded by Alexion Pharmaceuticals , Inc., Boston, Massachusetts, USA, and was provided by Leia Silvagnoli-Compston, MSc, and Alexander T. Hardy, PhD, of Bioscript, Macclesfield, UK. The authors acknowledge Åsa Lommelé, PhD, and Jonathan Mathias, PhD, of Alexion Pharmaceuticals, Inc. for their critical review of the manuscript. The authors also acknowledge Marc Vallee and Jimmy Wang for their assessment of all data and statistical analyses and Guillermo del Angel, PhD, of Alexion Pharmaceuticals, Inc. for review of the genetic analysis.

Publisher Copyright:
© 2021

All Science Journal Classification (ASJC) codes

  • Nephrology


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