Abstract: SA-PO806
Design of an Observational, Longitudinal Data Platform of Patients with C3 Glomerulopathy (C3G) and Idiopathic Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN) in the United States
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
- Poyan-Mehr, Ali, The Permanente Medical Group Inc, San Francisco, California, United States
- Khairnar, Rahul, Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, United States
- Bose, Anirban, Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, United States
- Tang, Jackson, Asclepius Analytics, New York, New York, United States
- Buchan, Claire, Asclepius Analytics, New York, New York, United States
- Buchan, Tayler, Asclepius Analytics, New York, New York, United States
- Ndife, Briana C., Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, United States
Background
C3G and IC-MPGN are rare renal diseases characterized by overactivation of the complement system. The absence of a comprehensive United States (US) real-world data (RWD) platform for these diseases limits our understanding of the challenges faced by patients (pts) and their healthcare providers. Here, we describe the development of a national RWD platform to capture, harmonize, and report comprehensive information for pts with C3G and idiopathic IC-MPGN.
Methods
This living data platform will include longitudinal data from prospective and retrospective cohorts of US-based pts of all ages with a diagnosis of C3G or idiopathic IC-MPGN, compiled using coordinated recruitment strategies. Site-based recruitment in collaboration with The GlomCon Foundation (a non-profit consortium of medical centers, clinicians, and pathologists overcoming challenges in glomerular disease through research partnerships) will generate retrospective and prospective cohorts of harmonized patient-level data from various single-center cohorts and patient registries. A patient cohort will also be generated from data collected via retrospective chart review. Variables collected will include demographic and clinical characteristics, comorbidities, biopsy findings, genetic studies, treatment patterns, genetic testing, laboratory values, and key outcomes of interest, including dialysis, transplant, and kidney failure. A data model will harmonize retrospective cohorts for pooled analyses. In a patient-centric recruitment approach, we will complement these center-specific, patient-level data with patient-reported outcomes, including the Functional Assessment of Chronic Illness Therapy–Fatigue Scale and the EuroQol-5D Measure of Health-Related Quality of Life.
Results
The site and patient recruitment are ongoing. The first analyses from this data platform are anticipated in 2025.
Conclusion
The development of an RWD platform for pts with C3G and idiopathic IC-MPGN will improve our understanding of the epidemiology, clinical burden, and determinants of clinical outcomes in these rare diseases, across a geographically diverse population in multiple centers and healthcare settings.
Funding
- Commercial Support – Novartis Pharmaceutical Corporation