C3 glomerulopathy and eculizumab: a report on four paediatric cases
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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 Proteinuria relapsed when eculizumab was withdrawn, but the re-introduction of eculizumab normalized proteinuria.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 Eculizumab was withdrawn due to multiple viral reactivations, but the re-introduction of the drug a few months later enabled a moderate decrease in proteinuria.
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.
KeywordsC3 glomerulonephritis Paediatrics Eculizumab Recurrence post-transplantation Children
The authors would like to thank Dr Marie-Nathalie Sarda (Department of Biology, Hospices Civils de Lyon) for her help in local complement assessment, and also Dr Moglie Le Quintrec, Pr Eric Rondeau, Pr Chantal Loirat, Pr Fadi Fakhouri and Dr Sophie Chauvet for their expertise during the French multidisciplinary review of the first two cases.
Compliance with ethical standards
The local ethical committee (CPP Lyon Sud Est II) approved this retrospective review of cases (IRB 00009118, session 1/27/2016).
Disclosure of interest
JB received travel grants from Alexion. ALL received travel grants from Alexion and honararia from Alexion (2013, 2014). VFB acts as a scientific advisor for Alexion.
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