title: C3 glomerulopathy and eculizumab: a report on four paediatric cases.
abstract: Eculizumab may be used to treat C3-glomerulopathy (C3G), a rare but severe glomerular disease.
  
  
  
  Patients 1, 2 and 3 were diagnosed with nephritic syndrome with alternative complement pathway activation (low C3, C3Nef-positive) and C3G at the age of 9, 13 and 12 years, respectively. Treatment with eculizumab normalized proteinuria within 1, 2 and 7 months, respectively. Proteinuria relapsed when eculizumab was withdrawn, but the re-introduction of eculizumab normalized proteinuria. Patient 4 was diagnosed with C3G at 9 years of age, with progression to end-stage renal disease within 2 years, followed by a first renal transplantation (R-Tx) with early disease recurrence and graft loss within 39 months. After a second R-Tx, she rapidly presented with biological and histological recurrence: therapy with eculizumab was started, with no effect on proteinuria after 5 months, in a complex clinical setting (i.e. association of C3G recurrence, humoral rejection and BK nephritis). Eculizumab was withdrawn due to multiple viral reactivations, but the re-introduction of the drug a few months later enabled a moderate decrease in proteinuria.
  
  
  
  These cases illustrate the efficacy of eculizumab, at least on native kidneys, in paediatric C3G. However, larger international studies are warranted to confirm the benefit and safety of eculizumab therapy.
authors.affiliation: Service de Néphrologie Rhumatologie Dermatologie Pédiatriques, Centre de Référence des Maladies Rénales Rares Hôpital Femme Mère Enfant, Hospices Civils de Lyon, 59 boulevard Pinel, 69677, Bron, France.
authors.forename: Célia
authors.identifier: 0000-0002-0566-2658
authors.initials: C
authors.lastname: Lebreton
authors.affiliation: Service de Néphrologie Rhumatologie Dermatologie Pédiatriques, Centre de Référence des Maladies Rénales Rares Hôpital Femme Mère Enfant, Hospices Civils de Lyon, 59 boulevard Pinel, 69677, Bron, France.|Faculté de Médecine Lyon Est, Université Lyon 1, Lyon, France.
authors.forename: Justine
authors.initials: J
authors.lastname: Bacchetta
authors.affiliation: Faculté de Médecine Lyon Est, Université Lyon 1, Lyon, France.|Laboratoire d'Anatomopathologie, Groupe Hospitalier Est, Hospices Civils de Lyon, Bron, France.
authors.forename: Frédérique
authors.initials: F
authors.lastname: Dijoud
authors.affiliation: Service de Pédiatrie, CHU Clermont Ferrand, Clermont Ferrand, France.
authors.forename: Lucie
authors.initials: L
authors.lastname: Bessenay
authors.affiliation: Laboratoire d'Immunologie, Hôpital Georges Pompidou, Assistance publique-Hôpitaux de Paris (AP-HP), Paris, France.
authors.forename: Véronique
authors.initials: V
authors.lastname: Fremeaux-Bacchi
authors.affiliation: Service de Néphrologie Rhumatologie Dermatologie Pédiatriques, Centre de Référence des Maladies Rénales Rares Hôpital Femme Mère Enfant, Hospices Civils de Lyon, 59 boulevard Pinel, 69677, Bron, France. anne-laure.sellier-leclerc@chu-lyon.fr.
authors.forename: Anne Laure
authors.initials: AL
authors.lastname: Sellier-Leclerc
mesh_terms: D000293:Adolescent; D061067:Antibodies, Monoclonal, Humanized / Q000627:therapeutic use*; D002648:Child; D003176:Complement C3 / Q000037:antagonists & inhibitors / Q000276:immunology; D003178:Complement C3 Nephritic Factor; D003170:Complement Pathway, Alternative / Q000187:drug effects; D005260:Female; D015432:Glomerulonephritis, Membranoproliferative / Q000150:complications / Q000188:drug therapy* / Q000276:immunology / Q000652:urine; D006084:Graft Rejection / Q000188:drug therapy / Q000276:immunology; D006801:Humans; D056724:Immunity, Humoral / Q000187:drug effects; D007668:Kidney / Q000473:pathology; D007676:Kidney Failure, Chronic / Q000209:etiology / Q000628:therapy*; D016030:Kidney Transplantation / Q000009:adverse effects; D008297:Male; D056687:Off-Label Use; D011507:Proteinuria / Q000188:drug therapy*; D012008:Recurrence; D016896:Treatment Outcome
chemical_list: D061067:Antibodies, Monoclonal, Humanized; D003176:Complement C3; D003178:Complement C3 Nephritic Factor; C481642:eculizumab
keywords: C3 glomerulonephritis; Children; Eculizumab; Paediatrics; Recurrence post-transplantation