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Kidney Week

Abstract: TH-PO800

Efficacy and Safety of the Long-Acting C5-Inhibitor Ravulizumab in Adult Patients with Atypical Hemolytic Uremic Syndrome (aHUS)

Session Information

Category: Genetic Diseases of the Kidneys

  • 1002 Genetic Diseases of the Kidneys: Non-Cystic


  • Rondeau, Eric, APHP; University Paris 6, PARIS, France
  • Scully, Marie, University College London Hospitals, London, United Kingdom
  • Ariceta, Gema, Hospital Universitari Vall d' Hebron, Barcelona, Spain
  • Barbour, Thomas D., Royal Melbourne Hospital, Parkville, Victoria, Australia
  • Cataland, Spero R., Ohio State University, Columbus, Ohio, United States
  • Heyne, Nils, Tübingen University Hospital, Tubingen, Germany
  • Miyakawa, Yoshitaka, Saitama Medical University, Saitama, Japan
  • Ortiz, Stephan, Alexion Pharmaceuticals, Inc., Boston, Massachusetts, United States
  • Swenson, Eugene Scott, Alexion Pharmaceuticals, Inc., Boston, Massachusetts, United States
  • Vallee, Marc, Alexion Pharmaceuticals, Inc., Boston, Massachusetts, United States
  • Yoon, Sung-Soo, Seoul National University, Seoul, Korea (the Republic of)
  • Kavanagh, David, National Renal Complement Therapeutics Centre, Newcastle upon Tyne, United Kingdom
  • Haller, Hermann G., Hannover Medical School, Hannover, Germany

Ravulizumab was engineered to achieve extended complement C5 inhibition, given every 8 weeks, while retaining the proven efficacy and safety of eculizumab. Here we evaluate the efficacy and safety of ravulizumab in adults with aHUS.


This was a phase 3, single arm study (NCT02949128). Complement inhibitor-naïve patients (pts) aged ≥18 years who fulfilled diagnostic criteria for aHUS (exclusion of ADAMTS13 <5% activity and Shiga toxin-producing Escherichia Coli) and active thrombotic microangiopathy (TMA) received ravulizumab at 8-week intervals during the maintenance phase. The primary endpoint was complete TMA response during the initial 183-day evaluation period. Secondary endpoints included time to complete TMA response, components of complete TMA response over time, CKD stage, dialysis-free status over time and time to dialysis-free status.


Fifty-six eligible pts were analyzed. Median age at baseline was 40 (range, 20–77) years and 36 (66%) were female. Complete TMA response was achieved in 30 pts (54%). 17/29 (59%) pts stopped dialysis (at a median time of 30 days). Primary endpoint and TMA parameter response over time is shown in the figure. Improvement in CKD stage from baseline was observed in 32/47 (68%) pts at Day 183. The most frequent serious adverse events were hypertension and pneumonia, each reported in 3 (5%) pts; 4 deaths not attributed to treatment occurred. No meningococcal infections were reported.


8-weekly ravulizumab dosing produced immediate, sustained and complete complement inhibition resulting in rapid hematologic and renal response with no unexpected safety concerns.


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