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Abstract: FR-PO136

Ongoing Eculizumab (ECU) Prevents Thrombotic Microangiopathy (TMA) in Patients (Pts) with Atypical Hemolytic Uremic Syndrome (aHUS): Final Long-Term Observational Study Data

Session Information

Category: Acute Kidney Injury

  • 003 AKI: Clinical and Translational


  • Menne, Jan, Medical School Hannover, Hannover, Germany
  • Delmas, Yahsou, CHU Bordeaux, Bordeaux, France
  • Kincaid, John F., Alexion Pharmaceuticals, Inc., New Haven, Connecticut, United States
  • Licht, Christoph, The Hospital for Sick Children, Toronto, Ontario, Canada
  • Minetti, Enrico E., Careggi University Hospital - Florence, Firenze, Italy
  • Mix, Chris, Alexion Pharmaceuticals, Inc., New Haven, Connecticut, United States
  • Provot, Francois, CHU de Lille, Lille, France
  • Rondeau, Eric, APHP; University Paris 6, PARIS, France
  • Sheerin, Neil S., Newcastle University, Newcastle upon Tyne, United Kingdom
  • Dain, Bradley, Alexion Pharmaceuticals, Inc., New Haven, Connecticut, United States
  • Weekers, Laurent E., CHU ULg, Esneux, Belgium
  • Greenbaum, Larry A., Emory University, Atlanta, Georgia, United States

Pts from previous ECU trials in aHUS could enroll in this observational long-term follow-up study (NCT01522170; enrollment/follow-up concluded), regardless of ongoing ECU use. The objective was to evaluate TMA rates and safety off and on ECU.


Endpoints included TMA event rate (primary) and targeted serious adverse events (TSAEs).


In 93 pts (42 with off-, 82 with on-treatment periods), the TMA event rate was 3-fold higher off vs on ECU. Rates were stratified by baseline characteristics (Table). TSAE rates were higher off vs on ECU. Four pts had meningococcal infections on ECU in the current study; all recovered with treatment and remained on ECU without dosing changes.


Final data from the largest prospective cohort with aHUS, with some pts receiving ECU for >5 yrs, demonstrate clinical benefits with ongoing ECU.
Acknowledgment: Medical writing support provided by Peloton Advantage with funding from Alexion.


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