Eculizumab for atypical haemolytic uraemic syndrome: what next?

Abstract

Effective treatments for atypical haemolytic uraemic syndrome (aHUS) have long been lacking, but the discovery that complement dysregulation is a major risk factor for this disease and the availability of the complement inhibitor eculizumab have improved the clinical picture. Legendre et al. have now published results from two prospective trials investigating eculizumab use in aHUS. Although we have come a long way, questions remain.