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Kidney Week

Abstract: PO2358

Efficacy and Safety of Ravulizumab in Pediatric Patients with Atypical Hemolytic Uremic Syndrome Naïve to Complement Inhibitor Treatment: 26-Week and 1-Year Data

Session Information

Category: Pediatric Nephrology

  • 1700 Pediatric Nephrology


  • Ariceta, Gema, Hospital Vall d'Hebron, Barcelona, Catalunya, Spain
  • Dixon, Bradley P., University of Colorado School of Medicine, Aurora, Colorado, United States
  • Kim, Seong heon, Pusan National University Children's Hospital, Seoul, Korea (the Republic of)
  • Kapur, Gaurav, Children's Hospital of Michigan, Detroit, Michigan, United States
  • Mauch, Teri Jo, University of Nebraska Medical Center, Omaha, Nebraska, United States
  • Ortiz, Stephan, Alexion Pharmaceuticals Inc, Boston, Massachusetts, United States
  • Vallee, Marc, Alexion Pharmaceuticals Inc, Boston, Massachusetts, United States
  • Denker, Andrew E., Alexion Pharmaceuticals Inc, Boston, Massachusetts, United States
  • Kang, Hee Gyung, Seoul National University College of Medicine, Seoul, Korea (the Republic of)
  • Greenbaum, Larry A., Emory University School of Medicine and Children's Healthcare of Atlanta, Atlanta, Georgia, United States

Ravulizumab is a complement C5 inhibitor derived from eculizumab, with an increased half–life extending the maintenance dosing schedule from every 2–3 to every 4–8 weeks. We evaluated the efficacy and safety of ravulizumab to resolve active thrombotic microangiopathy (TMA) in children with atypical hemolytic uremic syndrome (aHUS) naïve to complement inhibitors.


ALXN1210-aHUS-312 (NCT03131219) is a phase 3, single–arm trial in children with TMA due to aHUS. Patients received ravulizumab every 4–8 weeks, depending on bodyweight. The primary endpoint was complete TMA response (platelet count and lactate dehydrogenase normalization and ≥25% improvement in serum creatinine from baseline at 2 visits ≥28 days apart) through 26 weeks. Key secondary endpoints included eGFR and dialysis requirement. Patients were then followed in an ongoing extension period. Here we report efficacy outcomes through 1 year and safety from all available follow–up (median 82.6 weeks).


Eighteen patients (mean [SD] age 6.4 [4.5] years; 55.6% female) were enrolled; 14 (77.8%) achieved complete TMA response by 26 weeks, and 3 more patients (94.4%) by 50 weeks. Further improvements in TMA parameters occurred with longer-term treatment (Table). Mean (SD) increase in eGFR from baseline was 85.4 (54.3) mL/min/1.73m2 at 26 weeks, and 94.1 (50.7) at 50 weeks. Of 6 patients on dialysis at baseline, 5 (83.3%) discontinued dialysis by week 26, and the last patient by week 50. Complete free C5 inhibition was sustained through the trial. No unexpected adverse events, deaths, or meningococcal infections occurred.


Ravulizumab administration every 4–8 weeks improved hematologic and renal outcomes in 94% of patients, with no unexpected safety concerns. Renal function improved with longer-term treatment.

Complete TMA response components over time
VariableInitial evaluation period (26 weeks) n=18All available follow-up n=18
Complete TMA response14 (77.8)17 (94.4)
Platelet count normalization17 (94.4)17 (94.4)
LDH normalization16 (88.9)17 (94.4)
25% improvement in serum creatinine from baseline15 (83.3)17 (94.4)
Hematologic normalization16 (88.9)17 (94.4)

Data shown as n (%). LDH, lactate dehydrogenase; TMA, thrombotic microangiopathy.


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