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Pegcetacoplan in idiopathic and familial pediatric C3 glomerulopathy
Elena Román Ortiz1, Marina Sáez Bello2, Andrea Reparaz Suevos3
1Unidad de Nefrología Pediátrica, Hospital Universitario Dr. Peset, Valencia, Spain. eroman@comv.es.
Insights
Pegcetacoplan effectively treated pediatric C3 glomerulopathy (C3G) and immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN). This C3/C3b inhibitor improved kidney function and achieved remission in young patients with these rare kidney diseases.
Area of Science:
- Nephrology
- Complement System Biology
- Pediatric Rare Diseases
Background:
- C3 glomerulopathy (C3G) and IC-MPGN are complement-driven kidney diseases with poor prognosis.
- Pediatric C3G/IC-MPGN lacks approved targeted therapies.
- These conditions involve C3 deposition, alone or with immunoglobulins.
Purpose of the Study:
- To evaluate the efficacy of pegcetacoplan in pediatric C3G/IC-MPGN.
- To assess pegcetacoplan's impact on proteinuria, kidney function, and nephrotic syndrome remission.
Main Methods:
- Observational study of three pediatric cases with diverse C3G/IC-MPGN presentations.
- Treatment with the C3/C3b inhibitor pegcetacoplan.
- Monitoring of C3 levels, proteinuria, and kidney function.
Main Results:
- Pegcetacoplan inhibited C3, reduced C3 consumption, and normalized C3 levels.
- Significant proteinuria reduction within one month.
- Complete remission of nephrotic syndrome and improved kidney function after six months with no serious adverse events.
Conclusions:
- Pegcetacoplan shows potential for treating pediatric C3G/IC-MPGN.
- C3 inhibition is a promising therapeutic strategy for refractory or genetic C3G in children.
Background:
C3 glomerulopathy (C3G) and immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN) represent a continuous spectrum of a glomerular disease driven by dysregulation of the complement and characterized by C3 deposition alone or associated with immunoglobulins. Despite the significant burden and poor prognosis associated with these conditions, no therapies have been approved for their treatment in children.
Methods:
In this observational study, we present three pediatric cases that span the clinical and pathogenic spectrum of C3G/IC-MPGN, including multi-resistant nephrotic syndrome with clear terminal pathway activation, familial genetic C3G with early anti-C3 intervention, and multi-resistant nephrotic syndrome of unclear etiology, likely related to IC.
Results:
All three patients were successfully treated with the C3/C3b inhibitor pegcetacoplan, that inhibited C3, blocked C3 consumption, and restored physiological C3 levels, leading to significant proteinuria reduction within the first month. After six months, patients experienced notable improvements in kidney function, a complete remission of nephrotic syndrome, and normalized proteinuria levels. There were no treatment-related adverse events, only mild infections that resolved with standard oral therapy.
Conclusions:
These findings support the potential of C3 inhibition with pegcetacoplan in pediatric patients with refractory or genetic C3G.
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