Lookup NU author(s): Dr Edwin Wong,
Professor David Kavanagh
This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).
Atypical hemolytic uremic syndrome (aHUS), C3 glomerulopathy (C3G), and paroxysmal nocturnal hemoglobinuria (PNH) are prototypical disorders of complement dysregulation. Although complement overactivation is common to all, cell surface alternative pathway dysregulation (aHUS), fluid phase alternative pathway dysregulation (C3G), or terminal pathway dysregulation (PNH) predominates resulting in the very different phenotypes seen in these diseases. The mechanism underlying the dysregulation also varies with predominant acquired autoimmune (C3G), somatic mutations (PNH), or inherited germline mutations (aHUS) predisposing to disease. Eculizumab has revolutionized the treatment of PNH and aHUS although has been less successful in C3G. With the next generation of complement therapeutic in late stage development, these archetypal complement diseases will provide the initial targets.
Author(s): Wong EKS, Kavanagh D
Publication type: Article
Publication status: Published
Journal: Seminars in Immunopathology
Print publication date: 01/01/2018
Online publication date: 11/01/2018
Acceptance date: 01/11/2017
Date deposited: 11/01/2018
ISSN (print): 1863-2297
ISSN (electronic): 1863-2300
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