C3 glomerulopathy — understanding a rare complement-driven renal disease

The C3 glomerulopathies are a group of rare kidney diseases characterized by complement dysregulation occurring in the fluid phase and in the glomerular microenvironment, which results in prominent complement C3 deposition in kidney biopsy samples. The two major subgroups of C3 glomerulopathy — dense deposit disease (DDD) and C3 glomerulonephritis (C3GN) — have overlapping clinical and pathological features suggestive of a disease continuum. Dysregulation of the complement alternative pathway is fundamental to the manifestations of C3 glomerulopathy, although terminal pathway dysregulation is also common. Disease is driven by acquired factors in most patients — namely, autoantibodies that target the C3 or C5 convertases. These autoantibodies drive complement dysregulation by increasing the half-life of these vital but normally short-lived enzymes. Genetic variation in complement-related genes is a less frequent cause. No disease-specific treatments are available, although immunosuppressive agents and terminal complement pathway blockers are helpful in some patients. Unfortunately, no treatment is universally effective or curative. In aggregate, the limited data on renal transplantation point to a high risk of disease recurrence (both DDD and C3GN) in allograft recipients. Clinical trials are underway to test the efficacy of several first-generation drugs that target the alternative complement pathway.

Appel, Gerald B.Columbia University (författare)
Blom, Anna M.Lund University,Lunds universitet,Proteinkemi, Malmö,Forskargrupper vid Lunds universitet,Protein Chemistry, Malmö,Lund University Research Groups(Swepub:lu)klke-abl (författare)
Cook, H. TerenceImperial College London (författare)
D’Agati, Vivette D.Columbia University (författare)
Fakhouri, FadiNantes University Hospital (författare)
Fremeaux-Bacchi, VéroniqueParis Descartes University,Necker-Enfants Malades Hospital (författare)
Józsi, MihálySemmelweis University,Eötvös Loránd University (författare)
Kavanagh, DavidUniversity of Newcastle upon Tyne (författare)
Lambris, John D.University of Pennsylvania (författare)
Noris, MarinaMario Negri Institute for Pharmacological Research (författare)
Pickering, Matthew C.Imperial College London (författare)
Remuzzi, GiuseppeMario Negri Institute for Pharmacological Research,Azienda Ospedaliera Papa Giovanni XXIII,University of Milan (författare)
de Córdoba, Santiago RodriguezBiological Research Center (CIB), Madrid,Biomedical Network on Rare Diseases (CIBERER) (författare)
Sethi, SanjeevMayo Clinic Minnesota (författare)
Van der Vlag, JohanRadboud University Nijmegen (författare)
Zipfel, Peter F.Leibniz Institute for Natural Product Research and Infection Biology,Friedrich Schiller University Jena (författare)
Nester, Carla M.University of Iowa (författare)
University of IowaColumbia University (creator_code:org_t)

Ingår i:Nature Reviews Nephrology: Springer Science and Business Media LLC1759-50611759-507X

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