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

Updated: Nov 12, 2018
  • Author: Robert S Gillespie, MD, MPH; Chief Editor: Craig B Langman, MD  more...
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Eculizumab (Soliris) is the only treatment approved by the US Food and Drug Administration (FDA) (September, 2011) for adults and children with aHUS. Approval was based on data from adults and children who were resistant or intolerant to, or receiving, long-term plasma exchange/infusion. Data also included children (aged 2 mo to 17 y) who received eculizumab with or without prior plasma exchange/infusion. Eculizumab demonstrated significant improvement in platelet count from baseline (P = .0001). Thrombotic microangiopathy events were reduced, and maintained or improved kidney function was also reported. [25, 26, 27, 28]

Eculizumab is a humanized monoclonal antibody against C5, inhibiting progression of the complement cascade and blocking terminal complement activation. Specifically, eculizumab prevents cleavage of C5 into C5a and C5b, the latter being an essential component of the membrane attack complex.

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