Erschienen in:
24.02.2017 | Original Article
C3 glomerulopathy and eculizumab: a report on four paediatric cases
verfasst von:
Célia Lebreton, Justine Bacchetta, Frédérique Dijoud, Lucie Bessenay, Véronique Fremeaux-Bacchi, Anne Laure Sellier-Leclerc
Erschienen in:
Pediatric Nephrology
|
Ausgabe 6/2017
Einloggen, um Zugang zu erhalten
Abstract
Background
Eculizumab may be used to treat C3-glomerulopathy (C3G), a rare but severe glomerular disease.
Diagnosis and Treatment
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 Proteinuria relapsed when eculizumab was withdrawn, but the re-introduction of eculizumab normalized proteinuria.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 Eculizumab was withdrawn due to multiple viral reactivations, but the re-introduction of the drug a few months later enabled a moderate decrease in proteinuria.
Conclusion
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.