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Abstract: PO1851

Comparative Efficacy of Ravulizumab and Eculizumab in the Treatment of Atypical Hemolytic Uremic Syndrome: An Indirect Comparison Using Clinical Trial Data

Session Information

Category: Glomerular Diseases

  • 1203 Glomerular Diseases: Clinical, Outcomes, and Trials


  • Rondeau, Eric, Hôpital Tenon, APHP and Sorbonne Université, Paris, France
  • Hatswell, Anthony J., Delta Hat Limited, Nottingham, United Kingdom
  • Cataland, Spero R., Ohio State University, Columbus, Ohio, United States
  • Chen, Peter, Alexion Pharmaceuticals Inc., Boston, Massachusetts, United States
  • Freemantle, Nicholas, University College London, London, United Kingdom
  • Myren, Karl-Johan, Alexion Pharmaceuticals Inc., Boston, Massachusetts, United States
  • Lommele, Asa, Alexion Pharmaceuticals Inc., Boston, Massachusetts, United States
  • Wang, Yan, Alexion Pharmaceuticals Inc., Boston, Massachusetts, United States
  • Deighton, Kevin, Delta Hat Limited, Nottingham, United Kingdom
  • Knowles, Emma, Delta Hat Limited, Nottingham, United Kingdom
  • Sheerin, Neil S., Newcastle University, Newcastle, United Kingdom
  • Tomazos, Ioannis, Alexion Pharmaceuticals Inc., Boston, Massachusetts, United States

Atypical hemolytic uremic syndrome (aHUS) is a rare disease that can cause organ damage or death if not suitably treated. Eculizumab (ECU), a C5 inhibitor, was approved to treat aHUS in 2011. Ravulizumab (RAV), approved in 2019, was engineered from ECU to reduce dosing frequency (every 8 weeks [for patients weighing ≥20 kg] vs 2 weeks, respectively) and minimise treatment burden. Both drugs have established safety and efficacy via pivotal single armed studies. We indirectly compared the efficacy of RAV vs ECU using clinical trial data.


Patient-level data from a pivotal RAV trial (NCT02949128) and pivotal ECU trials (NCT00844428, NCT01194973) for adults with aHUS without kidney transplant were used. Propensity scores were calculated based on baseline characteristics (dialysis status, estimated glomerular filtration rate [eGFR], platelet count and serum lactate dehydrogenase), with stabilized inverse probability weighting used to balance groups while preserving sample size. Outcomes were changes in clinical characteristics at 26 weeks, and evaluated between groups using appropriate statistical tests at a 5% significance level.


In all, 85 patients (46 RAV, 39 ECU) were included for analysis. Baseline characteristics were balanced after weighting, with no significant difference between groups in any clinical or patient-reported characteristics. At 26 weeks, outcomes were improved from baseline in both groups, including reduced prevalence of dialysis, and increased mean eGFR and mean platelet count, with no significant differences between groups (Table).


After balancing patient characteristics between study groups, no significant differences were seen between outcomes for ECU and RAV at 26 weeks.

Representative clinical characteristics at baseline and 26 weeks.
Clinical characteristicPatients receiving ravulizumab (n=46)*Patients receiving eculizumab (n=39)P valueDifferences between groups (95% confidence interval)
Patients receiving dialysis (%)
At baseline
At 26 weeks
0 (-21 to 21)
-15 (-30 to 1)
eGFR (mL/min/1.73m2; mean [SD])
At baseline
At 26 weeks
16.7 (16.6)
55.4 (40.8)
16.6 (12.4)
51.4 (30.8)
0.0 (-6.3 to 6.3)
-4.0 (-19.8 to 11.8)
Patients experiencing an improvement of ≥15 mL/min/1.73m2 in eGFR from baseline at 26 weeks (%)59640.6625 (-16 to 26)
Platelet count (x109/L; mean [SD])
At baseline
At 26 weeks
118 (85)
243 (81)
118 (65)
244 (65)
0 (-33 to 32)
1 (-31 to 33)

*For eGFR and platelet count, n=43 at 26 weeks. eGFR defined as 10 mL/min/1.73m2 for those on dialysis.


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