NEPHROLOGY: Edited by Michel BaumEculizumab in the treatment of atypical haemolytic uraemic syndrome and other complement-mediated renal diseasesNester, Carla M.a,b,c; Brophy, Patrick D.b,c,d Author Information aDepartment of Internal Medicine bDepartment of Pediatrics cRare Renal Disease Clinic dDepartment of Surgery, University of Iowa, Iowa City, Iowa, USA Correspondence to Carla M. Nester, MD, Department of Pediatrics & Internal Medicine, Rare Renal Disease Clinic, University of Iowa, 200 Hawkins Dr 4036BT, Iowa City, IA 52242, USA. E-mail: [email protected] Current Opinion in Pediatrics: April 2013 - Volume 25 - Issue 2 - p 225-231 doi: 10.1097/MOP.0b013e32835df4a3 Buy Metrics Abstract Purpose of review This review considers the use of eculizumab in the treatment of atypical haemolytic uraemic syndrome (aHUS) as well as the other complement-mediated renal diseases, including dense deposit disease (DDD) and C3 glomerulonephritis (C3GN). In addition, a brief discussion of the effectiveness of eculizumab for the prevention of antibody-mediated rejection (AMR) in the setting of renal transplant and the treatment of shiga toxin associated haemolytic uraemic syndrome (STEC HUS) is also provided. Recent findings No randomized controlled trials exist to support the use of eculizumab in renal disease. The results of two unpublished, prospective adult and adolescent trials support its utility in aHUS, whereas retrospective data support the effectiveness in paediatric aHUS. These two data sets form the basis of the sole renal indication for eculizumab. One small, single-centre trial and a growing number of case reports support the use of eculizumab in C3 glomerulopathy (C3G). There are limited trial data in AMR and renal transplant. Finally, there are conflicting data for the use of eculizumab in STEC HUS. Summary The cumulative published data establish the effectiveness of eculizumab in a select group of renal diseases that have at the centre of their disease either abnormal complement control or maladaptive complement activation. © 2013 Lippincott Williams & Wilkins, Inc.