Pegcetacoplan-induced remission in pediatric immune-complex membranoproliferative glomerulonephritis with comorbid autosomal recessive polycystic kidney disease: a case report.
PMID 41884136 | PMCID PMC13010528 | DOI 10.3389/fmed.2026.1668375 · Frontiers in medicine · 2026
BACKGROUND: Membranoproliferative glomerulonephritis (MPGN) is a rare glomerular disorder characterized by immune complex or complement-mediated injury, often leading to nephrotic syndrome, hypertension, and progressive renal dysfunction. Its management remains challenging, particularly in pediatric patients with coexisting renal pathologies. CASE PRESENTATION: We report a case of an 11-years-old girl who presented with nephrotic syndrome, severe hypertension, and impaired renal function. Renal biopsy confirmed immune complex-mediated MPGN (IC-MPGN), and genetic testing revealed a homozygous, pathogenic missense PKHD1 variant (NM_138694.3:c.4870C > T; p.(Arg1624Trp), leading to a diagnosis of autosomal recessive polycystic kidney disease (ARPKD). Initial treatment with prednisolone and mycophenolate mofetil failed to halt disease progression, and the patient became dialysis dependent. Pegcetacoplan, a complement C3 inhibitor, was subsequently initiated. After 11 weeks of pegcetacoplan therapy, the patient achieved renal recovery and was successfully weaned from dialysis. Proteinuria decreased from nephrotic to sub-nephrotic levels without significant adverse effects. CONCLUSION: To our knowledge, this is the first pediatric case of IC-MPGN with genetically confirmed ARPKD successfully treated with pegcetacoplan. The case illustrates that renal recovery occurred following initiation of proximal complement inhibition with pegcetacoplan.
Validated evidence
| Type | Entity | Source evidence | Confidence | Extractor |
|---|---|---|---|---|
| gene | PKHD1 | “Renal biopsy confirmed immune complex-mediated MPGN (IC-MPGN), and genetic testing revealed a homozygous, pathogenic missense PKHD1 variant (NM_138694.3:c.4870C > T; p.(Arg1624Trp), leading to a diagnosis of autosomal recessive polycystic kidney disease (ARPKD).” | 0.98 | hgnc_dict_v1 |
| phenotype | polycystic kidney disease | “Pegcetacoplan-induced remission in pediatric immune-complex membranoproliferative glomerulonephritis with comorbid autosomal recessive polycystic kidney disease: a case report.” | 0.98 | phenotype_alias_lexicon_v2 |
| phenotype | hypertension | “BACKGROUND: Membranoproliferative glomerulonephritis (MPGN) is a rare glomerular disorder characterized by immune complex or complement-mediated injury, often leading to nephrotic syndrome, hypertension, and progressive renal dysfunction.” | 0.98 | phenotype_alias_lexicon_v2 |
| phenotype | nephrotic syndrome | “BACKGROUND: Membranoproliferative glomerulonephritis (MPGN) is a rare glomerular disorder characterized by immune complex or complement-mediated injury, often leading to nephrotic syndrome, hypertension, and progressive renal dysfunction.” | 0.98 | phenotype_alias_lexicon_v2 |
| variant | p.Arg1624Trp | “Renal biopsy confirmed immune complex-mediated MPGN (IC-MPGN), and genetic testing revealed a homozygous, pathogenic missense PKHD1 variant (NM_138694.3:c.4870C > T; p.(Arg1624Trp), leading to a diagnosis of autosomal recessive polycystic kidney disease (ARPKD).” | 0.95 | hgvs_regex_v1 |