What is the role of eculizumab in the treatment of pediatric hemolytic uremic syndrome (HUS)?

Updated: Nov 12, 2018
  • Author: Robert S Gillespie, MD, MPH; Chief Editor: Craig B Langman, MD  more...
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Eculizumab is a monoclonal antibody against the C5 component of the complement system. Its use in aHUS is well established and is described below under Special considerations for aHUS.

The role of eculizumab in STEC-HUS is uncertain and has not been studied in a controlled clinical trial. Existing data are limited to case reports, retrospective studies, and registry data. [49] During an outbreak of STEC-HUS in Germany, a letter to the editor reporting experience with three pediatric patients suggested that eculizumab might be beneficial in STEC-HUS patients with neurologic involvement. [15] Subsequent larger reviews of adult [16, 17] and pediatric [5] patients from the same outbreak did not find a benefit to eculizumab.

These were uncontrolled, open-label studies, and many patients received other therapies such as plasma exchange and/or antibiotics (despite the lack of evidence to support their use in STEC-HUS). This outbreak was due to an unusual serotype of E coli, O104:H4, not the more common O157:H7 serotype. One group of reviewers has noted: “It is very difficult to draw definitive conclusions about the efficacy of eculizumab treatment for STEC-HUS from any of the published reports based on its emergent use on a compassionate basis. . .because of potential biases in patient selection and because of the impact of concomitant therapy.” [18]

A 2-year follow-up on two phase 2 studies found that the earlier clinical benefits achieved by eculizumab treatment of aHUS were maintained and that the estimated glomerular filtration rate (eGFR) was significantly improved compared with baseline and year 1. [19]

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