Long-Term Efficacy and Safety of the Long-Acting Complement C5 Inhibitor Ravulizumab for the Treatment of Atypical Hemolytic Uremic Syndrome in Adults - Sorbonne Université
Journal Articles Kidney International Reports Year : 2021

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

Katherine Garlo
  • Function : Author
Jan Menne
  • Function : Author
Yoshitaka Miyakawa
  • Function : Author
Sunil Babu
  • Function : Author
Nilufer Broeders
  • Function : Author
Nicole Lietar
  • Function : Author
Fiona Brown
Philip Campbell
  • Function : Author
Josep Campistol
  • Function : Author
Paramit Chowdhury
  • Function : Author
Theo Kasimatis
  • Function : Author
Lino Cirami
  • Function : Author
Leonardo Caroti
  • Function : Author
Guilia Antognoli
  • Function : Author
Yahsou Delmas
  • Function : Author
Vladimir Dobronravov
  • Function : Author
Anja Gaeckler
  • Function : Author
Cyril Garrouste
Gregory Greenwood
  • Function : Author
Siân Griffin
  • Function : Author
Chiu-Ching Huang
  • Function : Author
I-Ru Chen
  • Function : Author
Susan Huang
  • Function : Author
Jin Seok Kim
  • Function : Author
Gaetano La Manna
  • Function : Author
Guillaume Jeantet
  • Function : Author
Iino Fumie
  • Function : Author
Eric Rondeau
  • Function : Author
  • PersonId : 835505
Hermann Haller
  • Function : Author
Johan Morelle
  • Function : Author
Eric Goffin
  • Function : Author
Anja Muhlfeld
  • Function : Author
Shashi Nagaraj
  • Function : Author
Gowthami Arepally
  • Function : Author
Doyeun Oh
  • Function : Author
Masayoshi Okumi
  • Function : Author
Manuel Praga Terente
  • Function : Author
Francois Provot
  • Function : Author
Ulf Schönermarck
  • Function : Author
Michael Fischereder
  • Function : Author
Natalia Ramos Terrada
  • Function : Author
Barbara Seitz-Polski
  • Function : Author
Guillaume Favre
  • Function : Author
Sonia Boyer-Suavet
  • Function : Author
Maria Vinogradova
  • Function : Author
Tatiana Kirsanova
  • Function : Author
Edwin K.S. Wong
  • Function : Author

Abstract

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.
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Dates and versions

hal-03278504 , version 1 (05-07-2021)

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Thomas Barbour, Marie Scully, Gema Ariceta, Spero Cataland, Katherine Garlo, 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 International Reports, 2021, 6 (6), pp.1603-1613. ⟨10.1016/j.ekir.2021.03.884⟩. ⟨hal-03278504⟩
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