Abstract: FR-PO0935
Use of Pegcetacoplan in a Pediatric Patient with ESRD to Prevent Recurrence of C3 Glomerulonephritis After Kidney Transplantation
Session Information
- Pediatric Nephrology: Genetic Diseases, Development, Neonatal Nephrology, Glomerular Diseases, and More
October 23, 2026 | Location: Exhibit Hall A, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Pediatric Nephrology
- 1800 Pediatric Nephrology
Authors
- Huang, Elliott, Columbia University Vagelos College of Physicians and Surgeons, New York, New York, United States
- Fernandez, Hilda E., Columbia University Vagelos College of Physicians and Surgeons, New York, New York, United States
- Lieberman, Kenneth V., Hackensack Meridian Joseph M Sanzari Children's Hospital, Hackensack, New Jersey, United States
Introduction
C3 glomerulonephritis (C3GN) is a rare cause of kidney disease characterized by high incidence of end stage renal disease and early recurrence after renal transplant. We report the first case of the use of pegcetacoplan, a C3 inhibitor, in a pediatric patient with ESRD due to C3GN undergoing renal transplant.
Case Description
A 10-year-old female with a history of chronic anemia presented to the ED with abdominal pain, decreased appetite, emesis and dark urine for one month. Initial labs were notable for a BUN of 111 mg/dL and serum creatinine of 11 mg/dL (BUN and creatinine from one month prior were 22 mg/dL and 0.89 mg/dL respectively). C3 was low, and soluble C5b-9 was elevated. Renal biopsy showed MPO-ANCA associated necrotizing and crescentic glomerulonephritis (Figure 1A) and diffuse C3 deposits consistent with C3 glomerulopathy (Figure 1B). Genetic testing showed a heterozygous C3 variant associated with gain of function autosomal dominant atypical HUS. She started hemodialysis and received pegcetacoplan through the Apellis compassionate use program; while C3 levels and sC5b-9 normalized, renal function did not recover, so pegcetacoplan was discontinued. She restarted pegcetacoplan about one month prior to renal transplant and then underwent living unrelated kidney transplant with thymoglobulin induction and rituximab infusions. She continued pegcetacoplan after transplant, and sC5b-9 and C3 remained normal. Renal biopsy of the transplant kidney about 4 months post-transplant showed no recurrence of the C3GN on light microscopy or immunofluorescence (Figure 1C and 1D), and she has remained disease free 8 months post-transplant.
Discussion
C3 inhibitors such as pegcetacoplan have been shown to reduce proteinuria and disease activity in patients with C3GN, but had not previously been used to prevent disease recurrence post-transplant. We demonstrate that pegcetacoplan may help prevent disease recurrence in patients with C3GN requiring renal transplantation.
Acknowledgment
We would like to thank Michael Barry Stokes, MD Professor of Pathology and Cell Biology, Columbia University Vagelos College of Physicians and Surgeons, for providing the pathology images.
We would like to thank Apellis Pharmaceuticals for providing pegcetacoplan through their Compassionate Use program.
(A) and (B): Native kidney tissue. (C) and (D): Transplant kidney tissue.