Kidney Week

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

• AKI Clinical: Outcomes and Biomarkers
  November 03, 2017 | Location: Hall H, Morial Convention Center
  Abstract Time: 10:00 AM - 10:00 AM

Category: Acute Kidney Injury

• 003 AKI: Clinical and Translational

Authors

• Menne, Jan, Medical School Hannover, Hannover, Germany

Jan Menne,

• Delmas, Yahsou, CHU Bordeaux, Bordeaux, France

Yahsou Delmas,

• Kincaid, John F., Alexion Pharmaceuticals, Inc., New Haven, Connecticut, United States

John F. Kincaid,

• Licht, Christoph, The Hospital for Sick Children, Toronto, Ontario, Canada

Christoph Licht,

• Minetti, Enrico E., Careggi University Hospital - Florence, Firenze, Italy

Enrico E. Minetti,

• Mix, Chris, Alexion Pharmaceuticals, Inc., New Haven, Connecticut, United States

Chris Mix,

• Provot, Francois, CHU de Lille, Lille, France

Francois Provot,

• Rondeau, Eric, APHP; University Paris 6, PARIS, France

Eric Rondeau,

• Sheerin, Neil S., Newcastle University, Newcastle upon Tyne, United Kingdom

Neil S. Sheerin,

• Dain, Bradley, Alexion Pharmaceuticals, Inc., New Haven, Connecticut, United States

Bradley Dain,

• Weekers, Laurent E., CHU ULg, Esneux, Belgium

Laurent E. Weekers,

• Greenbaum, Larry A., Emory University, Atlanta, Georgia, United States

Larry A. Greenbaum,

Background

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.

Methods

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

Results

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.

Conclusion

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.

Funding

• Commercial Support – Alexion Pharmaceuticals, Inc.