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Shiga Toxin producing Escherichia coli (STEC)-associated hemolytic uremic syndrome (HUS), defined as typical, is the most common cause of acute renal failure in children. Atypical HUS (aHUS) is usually caused by uncontrolled complement activation, secondary to a dysregulation of the alternative pathway most frequently due to genetic variants in complement regulating proteins.
Recent studies have proven an activation of the alternative complement system also during STEC-associated-HUS, leading to the hypothesis of using Eculizumab, a monoclonal antibody directed against C5.
The aim of our study is to assess the efficacy and safety of Eculizumab in a cohort of pediatric patients compared with a group of patients receiving only supportive therapy, including dialysis, blood transfusion and plasmapheresis.
From 2010 to date, 80 patients with STEC-associated-HUS were admitted to the Bambino Gesù Children's Hospital. The prevalence of the serotype identified was E.coli 026. A total of 11 patients received Eculizumab infusion, 64% for severe neurological involvement and 36% in suspicion of an atypical form of HUS.
11 children with STEC-associated-HUS treated with conservative therapy alone, were selected as controls.
In our center, the administration of Eculizumab in patients with typical HUS was limited to cases with severe extrarenal involvement or in suspicion of atypical forms. Apart from a single patient with liver toxicity, the drug was safe and well tolerated.
However, a one-year comparison between the group of patients treated with Eculizumab and the group of patients treated with supportive therapy alone did not show significant differences in the outcome between the 2 groups.
Randomized controlled trials (RCT) are necessary to assess the efficacy and safety of the use of Eculizumab in STEC-HUS patients