Case report: benefits and challenges of long-term eculizumab in atypical hemolytic uremic syndrome

N Cullinan, KM Gorman, M Riordan, M Waldron… - …, 2015 - publications.aap.org
Atypical hemolytic uremic syndrome (aHUS) is caused by dysregulation of the complement
system, leading to complement overactivation. A humanized anti-C5 monoclonal antibody,
eculizumab, has been available for the treatment of aHUS since 2011. The long-term safety
and efficacy of this novel drug in the pediatric population remain under review. We present a
child with a hybrid CFH/CFHR3 gene who, having had multiple disease relapses despite
optimal treatment with plasma exchange, commenced eculizumab therapy in August 2010 …