Eculizumab is a safe and effective treatment in pediatric patients with atypical hemolytic uremic syndrome

Kidney Int. 2016 Mar;89(3):701-11. doi: 10.1016/j.kint.2015.11.026. Epub 2016 Jan 28.


Atypical hemolytic uremic syndrome (aHUS) is caused by alternative complement pathway dysregulation, leading to systemic thrombotic microangiopathy (TMA) and severe end-organ damage. Based on 2 prospective studies in mostly adults and retrospective data in children, eculizumab, a terminal complement inhibitor, is approved for aHUS treatment. Here we prospectively evaluated efficacy and safety of weight-based dosing of eculizumab in eligible pediatric patients with aHUS in an open-label phase II study. The primary end point was complete TMA response by 26 weeks. Twenty-two patients (aged 5 months-17 years) were treated; 16 were newly diagnosed, 12 had no prior plasma exchange/infusion during current TMA symptomatology, 11 received baseline dialysis and 2 had prior renal transplants. By week 26, 14 achieved a complete TMA response, 18 achieved hematologic normalization, and 16 had 25% or better improvement in serum creatinine. Plasma exchange/infusion was discontinued in all, and 9 of the 11 patients who required dialysis at baseline discontinued, whereas none initiated new dialysis. Eculizumab was well tolerated; no deaths or meningococcal infections occurred. Bone marrow failure, wrist fracture, and acute respiratory failure were reported as unrelated severe adverse events. Thus, our findings establish the efficacy and safety of eculizumab for pediatric patients with aHUS and are consistent with proposed immediate eculizumab initiation following diagnosis in children.

Keywords: aHUS; children; eculizumab; kidney disease; pediatric; thrombotic microangiopathy.

Publication types

  • Clinical Trial, Phase II
  • Multicenter Study

MeSH terms

  • Adolescent
  • Age Factors
  • Antibodies, Monoclonal, Humanized / adverse effects
  • Antibodies, Monoclonal, Humanized / pharmacokinetics
  • Antibodies, Monoclonal, Humanized / therapeutic use*
  • Atypical Hemolytic Uremic Syndrome / diagnosis
  • Atypical Hemolytic Uremic Syndrome / drug therapy*
  • Atypical Hemolytic Uremic Syndrome / immunology
  • Australia
  • Child
  • Child, Preschool
  • Complement Activation / drug effects*
  • Complement Inactivating Agents / adverse effects
  • Complement Inactivating Agents / pharmacokinetics
  • Complement Inactivating Agents / therapeutic use*
  • Europe
  • Female
  • Humans
  • Infant
  • Male
  • North America
  • Plasma Exchange
  • Prospective Studies
  • Renal Dialysis
  • Time Factors
  • Treatment Outcome


  • Antibodies, Monoclonal, Humanized
  • Complement Inactivating Agents
  • eculizumab