Use of eculizumab for atypical haemolytic uraemic syndrome and C3 glomerulopathies
Top Cited Papers
- 2 October 2012
- journal article
- review article
- Published by Springer Nature in Nature Reviews Nephrology
- Vol. 8 (11), 643-657
- https://doi.org/10.1038/nrneph.2012.214
Abstract
In the past decade, a large body of evidence has accumulated in support of the critical role of dysregulation of the alternative complement pathway in atypical haemolytic uraemic syndrome (aHUS) and C3 glomerulopathies. These findings have paved the way for innovative therapeutic strategies based on complement blockade, and eculizumab, a monoclonal antibody targeting the human complement component 5, is now widely used to treat aHUS. In this article, we review 28 case reports and preliminary data from 37 patients enrolled in prospective trials of eculizumab treatment for episodes of aHUS involving either native or transplanted kidneys. Eculizumab may be considered as an optimal first-line therapy when the diagnosis of aHUS is unequivocal and this treatment has the potential to rescue renal function when administered early after onset of the disease. However, a number of important issues require further study, including the appropriate duration of treatment according to an individual's genetic background and medical history, the optimal strategy to prevent post-transplantation recurrence of aHUS and a cost-efficacy analysis. Data regarding the efficacy of eculizumab in the control of C3 glomerulopathies are more limited and less clear, but several observations suggest that eculizumab may act on the most inflammatory forms of this disorder.Keywords
This publication has 105 references indexed in Scilit:
- Eculizumab as rescue therapy for atypical hemolytic uremic syndrome with normal platelet countPediatric Nephrology, 2012
- Insufficient protection by Neisseria meningitidis vaccination alone during eculizumab therapyPediatric Nephrology, 2011
- Substrate recognition by complement convertases revealed in the C5-cobra venom factor complexThe EMBO Journal, 2011
- Structures of C3b in Complex with Factors B and D Give Insight into Complement Convertase FormationScience, 2010
- Infections of People with Complement Deficiencies and Patients Who Have Undergone SplenectomyClinical Microbiology Reviews, 2010
- Treatment with human complement factor H rapidly reverses renal complement deposition in factor H-deficient miceKidney International, 2010
- Structural and functional implications of the alternative complement pathway C3 convertase stabilized by a staphylococcal inhibitorNature Immunology, 2009
- Translational Mini-Review Series on Complement Factor H: Genetics and disease associations of human complement factor HClinical and Experimental Immunology, 2007
- Spontaneous hemolytic uremic syndrome triggered by complement factor H lacking surface recognition domainsThe Journal of Experimental Medicine, 2007
- Prevention of C5 activation ameliorates spontaneous and experimental glomerulonephritis in factor H-deficient miceProceedings of the National Academy of Sciences, 2006