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Diseases of complement dysregulation—an overview

Lookup NU author(s): Dr Edwin Wong, Professor David KavanaghORCiD



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.

Publication metadata

Author(s): Wong EKS, Kavanagh D

Publication type: Article

Publication status: Published

Journal: Seminars in Immunopathology

Year: 2018

Volume: 40

Issue: 1

Pages: 49-64

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

Publisher: Springer


DOI: 10.1007/s00281-017-0663-8


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