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Semin Immunopathol. 2018 Jan;40(1):49-64. doi: 10.1007/s00281-017-0663-8. Epub 2018 Jan 11.

Diseases of complement dysregulation-an overview.

Author information

1
The National Renal Complement Therapeutics Centre, aHUS Service, Building 26, Royal Victoria Infirmary, Queen Victoria Road, Newcastle upon Tyne, NE1 4LP, UK.
2
Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne, UK.
3
The National Renal Complement Therapeutics Centre, aHUS Service, Building 26, Royal Victoria Infirmary, Queen Victoria Road, Newcastle upon Tyne, NE1 4LP, UK. david.kavanagh@ncl.ac.uk.
4
Institute of Cellular Medicine, Newcastle University, Newcastle upon Tyne, UK. david.kavanagh@ncl.ac.uk.

Abstract

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.

KEYWORDS:

C3G, aHUS; Complement; PNH

PMID:
29327071
PMCID:
PMC5794843
DOI:
10.1007/s00281-017-0663-8
[Indexed for MEDLINE]
Free PMC Article

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