WIP 58 - Treatment Practice Pattern and Clinical Trajectory in Pediatric Patients with C3 Glomerulopathy

Publication Number: WIP 58.7459

Ana S. Solis Zavala, Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, United States; Bradley P.. Dixon, University of Colorado School of Medicine, Aurora, CO, United States; Meredith Harris, Lurie Children's Hospital, Chicago, IL, United States

Background: C3 Glomerulopathy (C3G) is characterized by dysregulation of the alternative complement pathway leading to deposition of C3 and its fragments in the glomeruli. C3G has variable clinical presentation, response to treatment, and clinical trajectory. Disease rarity has led to challenges in determining best diagnostic and treatment strategies. Therapy of choice is influenced by degree of kidney dysfunction or proteinuria, and in children, is mostly guided by small published series of patients and expert opinion.

Objective: Aim 1- We will describe clinical presentation, clinical trajectory, and treatment pattern in patients with C3G, hypothesizing that there is substantial variability in the clinical trajectory of patients and their treatment patterns. Aim 2- We will elucidate genotype-phenotype correlations regarding presentation, outcomes, and treatment patterns in patients with C3G, anticipating that the presence of genetic drivers of disease increases the risk of partial or no remission. Aim 3- We will assess the association between specific genotypes and immunological biomarker profiles with kidney related outcomes, hypothesizing that specific genotypes or immunological biomarker profiles are associated with progression to advanced chronic kidney disease. By describing a large dataset of patients with C3G, we will create an up-to-date dataset as groundwork for future studies focused on targeted therapies.

Design/Methods: Our center will serve as the pilot site with ~35 patients identified. We anticipate expansion to a multi-center retrospective study to be supported and funded by the Pediatric Nephrology Research Consortium. Inclusion Criteria: Patients ages 0-18 years diagnosed with C3G from January 2013 to December 2022 with confirmed diagnosis via kidney biopsy. We will perform descriptive statistics to characterize the clinical phenotype and treatment patterns and possible analysis with inference statistics to evaluate statistically significant factors associated with varying response to treatment. Study is IRB approved. Data analysis to be completed by 4/1/2025.