Complement C3-targeted therapy in C3 glomerulopathy, a prototype of complement-mediated kidney diseases

Legendre, 2013, Terminal complement inhibitor eculizumab in atypical hemolytic-uremic syndrome, N. Engl. J. Med., 368, 2169, 10.1056/NEJMoa1208981 Jayne, 2021, Avacopan for the treatment of ANCA-associated vasculitis, N. Engl. J. Med., 384, 599, 10.1056/NEJMoa2023386 Smith, 2019, C3 glomerulopathy — understanding a rare complement-driven renal disease, Nat. Rev. Nephrol., 15, 129, 10.1038/s41581-018-0107-2 Pickering, 2013, C3 glomerulopathy: consensus report, Kidney Int., 84, 1079, 10.1038/ki.2013.377 Pickering, 2013, C3 glomerulopathy: consensus report, Kidney Int., 84, 1079, 10.1038/ki.2013.377 Hou, 2014, Toward a working definition of C3 glomerulopathy by immunofluorescence, Kidney Int., 85, 450, 10.1038/ki.2013.340 Servais, 2012, Acquired and genetic complement abnormalities play a critical role in dense deposit disease and other C3 glomerulopathies, Kidney Int., 82, 454, 10.1038/ki.2012.63 Ravindran, 2018, C3 Glomerulopathy: ten years’ experience at mayo clinic, Mayo Clin. Proc., 93, 991, 10.1016/j.mayocp.2018.05.019 Bomback, 2018, C3 glomerulonephritis and dense deposit disease share a similar disease course in a large United States cohort of patients with C3 glomerulopathy, Kidney Int., 93, 977, 10.1016/j.kint.2017.10.022 Medjeral-Thomas, 2014, C3 Glomerulopathy: clinicopathologic features and predictors of outcome, CJASN, 9, 46, 10.2215/CJN.04700513 Bomback, 2018, C3 glomerulonephritis and dense deposit disease share a similar disease course in a large United States cohort of patients with C3 glomerulopathy, Kidney Int, 93, 977, 10.1016/j.kint.2017.10.022 Iatropoulos, 2016, Complement gene variants determine the risk of immunoglobulin-associated MPGN and C3 glomerulopathy and predict long-term renal outcome, Mol. Immunol., 71, 131, 10.1016/j.molimm.2016.01.010 Medjeral-Thomas, 2014, C3 glomerulopathy: clinicopathologic features and predictors of outcome, Clin. J. Am. Soc. Nephrol., 9, 46, 10.2215/CJN.04700513 Goodship, 2017, Atypical hemolytic uremic syndrome and C3 glomerulopathy: conclusions from a “Kidney Disease: Improving Global Outcomes” (KDIGO) Controversies Conference, Kidney Int., 91, 539, 10.1016/j.kint.2016.10.005 Le Quintrec, 2015, Eculizumab for treatment of rapidly progressive C3 glomerulopathy, Am. J. Kidney Dis., 65, 484, 10.1053/j.ajkd.2014.09.025 Bomback, 2012, Eculizumab for dense deposit disease and C3 glomerulonephritis, Clin. J. Am. Soc. Nephrol., 7, 748, 10.2215/CJN.12901211 Regunathan-Shenk, 2019, Kidney transplantation in C3 glomerulopathy: a case series, Am. J. Kidney Dis., 73, 316, 10.1053/j.ajkd.2018.09.002 Zand, 2014, Clinical findings, pathology, and outcomes of C3GN after kidney transplantation, J. Am. Soc. Nephrol., 25, 1110, 10.1681/ASN.2013070715 Marinozzi, 2017, C5 nephritic factors drive the biological phenotype of C3 glomerulopathies, Kidney Int., 92, 1232, 10.1016/j.kint.2017.04.017 Marinozzi, 2017, Anti-Factor B and anti-C3b autoantibodies in C3 glomerulopathy and Ig-associated membranoproliferative GN, J. Am. Soc. Nephrol., 28, 1603, 10.1681/ASN.2016030343 Blom, 2016, Testing the activity of complement convertases in serum/plasma for diagnosis of C4NeF-mediated C3 glomerulonephritis, J. Clin. Immunol., 36, 517, 10.1007/s10875-016-0290-5 Iatropoulos, 2016, Complement gene variants determine the risk of immunoglobulin-associated MPGN and C3 glomerulopathy and predict long-term renal outcome, Mol. Immunol., 71, 131, 10.1016/j.molimm.2016.01.010 Caravaca-Fontán F., Lucientes L., Serra N., Cavero T., Rodado R., Ramos N., et al. C3 glomerulopathy associated with monoclonal gammopathy: impact of chronic histologic lesions and beneficial effects of clone-targeted therapies. Nephrol Dial Transplant. 2021 Oct 22;gfab302. Wong E.K.S., Marchbank K.J., Lomax-Browne H., Pappworth I.Y., Denton H., Cooke K., et al. C3 Glomerulopathy and Related Disorders in Children: Etiology-Phenotype Correlation and Outcomes. Clin J Am Soc Nephrol. 2021 Nov;16(11):1639–51. Zhang, 2014, Defining the complement biomarker profile of C3 glomerulopathy, Clin. J. Am. Soc. Nephrol., 9, 1876, 10.2215/CJN.01820214 Marinozzi, 2017, C5 nephritic factors drive the biological phenotype of C3 glomerulopathies, Kidney Int., 92, 1232, 10.1016/j.kint.2017.04.017 Sethi, 2012, C3 glomerulonephritis: clinicopathological findings, complement abnormalities, glomerular proteomic profile, treatment, and follow-up, Kidney Int., 82, 465, 10.1038/ki.2012.212 Sethi, 2009, Glomeruli of dense deposit disease contain components of the alternative and terminal complement pathway, Kidney Int, 75, 952, 10.1038/ki.2008.657 Sethi, 2017, Characterization of C3 in C3 glomerulopathy, Nephrol. Dial. Transpl., 32, 459 Medjeral-Thomas, 2019, Glomerular complement factor H-related protein 5 (FHR5) is highly prevalent in c3 glomerulopathy and associated with renal impairment, Kidney Int Rep., 4, 1387, 10.1016/j.ekir.2019.06.008 Zhang, 2012, Causes of alternative pathway dysregulation in dense deposit disease, Clin. J. Am. Soc. Nephrol., 7, 265, 10.2215/CJN.07900811 Pickering, 2002, Uncontrolled C3 activation causes membranoproliferative glomerulonephritis in mice deficient in complement factor H, Nat. Genet., 31, 424, 10.1038/ng912 Devalaraja-Narashimha, 2021, Humanized C3 mouse: a novel accelerated model of C3 glomerulopathy, J. Am. Soc. Nephrol., 32, 99, 10.1681/ASN.2020050698 Wessels, 1995, Studies of group B streptococcal infection in mice deficient in complement component C3 or C4 demonstrate an essential role for complement in both innate and acquired immunity, Proc. Natl. Acad. Sci. U. S. A., 92, 11490, 10.1073/pnas.92.25.11490 Zhang, 2014, Defining the complement biomarker profile of C3 glomerulopathy, Clin. J. Am. Soc. Nephrol., 9, 1876, 10.2215/CJN.01820214 Pickering, 2006, Prevention of C5 activation ameliorates spontaneous and experimental glomerulonephritis in factor H-deficient mice, Proc. Natl. Acad. Sci. U. S. A, 103, 9649, 10.1073/pnas.0601094103 Goodship, 2017, Atypical hemolytic uremic syndrome and C3 glomerulopathy: conclusions from a “Kidney Disease: Improving Global Outcomes” (KDIGO) Controversies Conference, Kidney Int., 91, 539, 10.1016/j.kint.2016.10.005 Herlitz, 2012, Pathology after eculizumab in dense deposit disease and C3 GN, J. Am. Soc. Nephrol., 23, 1229, 10.1681/ASN.2011121186 Le Quintrec, 2018, Patterns of clinical response to eculizumab in patients with C3 glomerulopathy, Am. J. Kidney Dis., 72, 84, 10.1053/j.ajkd.2017.11.019 Mannes, 2021, Complement inhibition at the level of C3 or C5: mechanistic reasons for ongoing terminal pathway activity, Blood, 137, 443, 10.1182/blood.2020005959 Gao, 2020, The complement C3a and C3a receptor pathway in kidney diseases, Front Immunol., 11, 1875, 10.3389/fimmu.2020.01875 Bao, 2011, Distinct roles for C3a and C5a in complement-induced tubulointerstitial injury, Kidney Int, 80, 524, 10.1038/ki.2011.158 Tang, 2009, C3a mediates epithelial-to-mesenchymal transition in proteinuric nephropathy, J. Am. Soc. Nephrol., 20, 593, 10.1681/ASN.2008040434 Haddad, 2021, Altered glycosylation of IgG4 promotes lectin complement pathway activation in anti-PLA2R1-associated membranous nephropathy, J. Clin. Invest., 131, 10.1172/JCI140453 Sahu, 1996, Inhibition of human complement by a C3-binding peptide isolated from a phage-displayed random peptide library, J. Immunol., 157, 884, 10.4049/jimmunol.157.2.884 Sahu, 2003, Compstatin, a peptide inhibitor of complement, exhibits species-specific binding to complement component C3, Mol. Immunol., 39, 557, 10.1016/S0161-5890(02)00212-2 Janssen, 2007, Structure of compstatin in complex with complement component C3c reveals a new mechanism of complement inhibition, J. Biol. Chem., 282, 29241, 10.1074/jbc.M704587200 Nilsson, 1998, Compstatin inhibits complement and cellular activation in whole blood in two models of extracorporeal circulation, Blood, 92, 1661, 10.1182/blood.V92.5.1661 Zhang, 2015, Compstatin analog Cp40 inhibits complement dysregulation in vitro in C3 glomerulopathy, Immunobiology, 220, 993, 10.1016/j.imbio.2015.04.001 Servais, 2012, Acquired and genetic complement abnormalities play a critical role in dense deposit disease and other C3 glomerulopathies, Kidney Int., 82, 454, 10.1038/ki.2012.63 Dempsey, 1996, C3d of complement as a molecular adjuvant: bridging innate and acquired immunity, Science, 271, 348, 10.1126/science.271.5247.348 Michels, 2021, Long-term follow-up including extensive complement analysis of a pediatric C3 glomerulopathy cohort, Pedia Nephrol. Schubart, 2019, Small-molecule factor B inhibitor for the treatment of complement-mediated diseases, Proc. Natl. Acad. Sci. U. S. A., 116, 7926, 10.1073/pnas.1820892116