Abstract: SA-PO802
Efficacy of Pegcetacoplan in Children with C3 Glomerulopathy
Session Information
- C3G, TMA, MGRS, Amyloidosis, and More
October 26, 2024 | Location: Exhibit Hall, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Glomerular Diseases
- 1402 Glomerular Diseases: Clinical, Outcomes, and Therapeutics
Authors
- Mancuso, Maria Cristina, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Lombardia, Italy
- Mastrangelo, Antonio, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Lombardia, Italy
- Dato, Letizia, Università del Piemonte Orientale, Novara, Novara, Italy
- Verdesca, Simona, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Lombardia, Italy
- Cugno, Massimo, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Lombardia, Italy
- Ardissino, Gianluigi, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Lombardia, Italy
Background
C3 glomerulopathy (C3G) is a rare chronic kidney disease caused by a dysregulation of the alternative complement pathway leading to glomerular deposition of complement component 3 (C3) followed by inflammation and tissue damage. Pegcetacoplan is an inhibitor of both C3 and of its active fragment C3b, that can prevent their glomerular deposition. A phase III registration protocol is currently ongoing in patients (adolescents and adults) with C3G, with promising results. Herein we describe our recent experience with Pegcetacoplan in 5 pediatric patients with C3G.
Methods
This retrospective, observational study presents the efficacy and safety of Pegcetacoplan in pediatric patients with biopsy proven C3G over a 12-week treatment period. The drug was administered subcutaneously, twice a week for the first month, then weekly. The primary endpoint was the change in urinary protein-to-urinary creatinine ratio evaluated by the mean of 3 samples collected on different days before each visit at baseline, 8 and 12 weeks. The changes in serum C3, albumine, creatinine and urinary erythrocytes (number/µL) were also evaluated.
Results
Detailed results are shown in Fig.1. Median C3 level increased of more than 600% while proteinuria decreased to 30% of baseline value. Some of the patients with decreased renal function exhibited an improvement of eGFR. No adverse event has been recorded except for some transient discomfort at the injection site.
Conclusion
All our patients showed a rapid over-normalization of the C3 levels, a significant reduction of proteinuria and a significant increase in albuminemia. We think that the present case series, although small and with a short follow up period, may be important to support other physicians to consider this treatment as an opportunity for their C3G patients.