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Real-World Analysis of Patients With C3 Glomerulopathy in the United States
Briana C Ndife1, Carolina Aldworth1, Jennifer Nguyen1
1Health Economics and Outcomes Research, Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA.
Insights
C3 glomerulopathy (C3G) is a rare kidney disease. This study found C3G patients in the US often have advanced disease and rapid CKD progression, indicating a need for new treatments.
Area of Science:
- Nephrology
- Immunology
Background:
- C3 glomerulopathy (C3G) is a rare kidney disease driven by overactivation of the alternative complement pathway (AP).
- Understanding the clinical course and progression of C3G is crucial for patient management.
Purpose of the Study:
- To describe the demographic and clinical characteristics of C3G patients in the US.
- To evaluate the progression of chronic kidney disease (CKD) in this population.
Main Methods:
- Retrospective cohort study using electronic health records (EHRs).
- Identified patients aged ≥12 years with C3G diagnosis and ≥12 months baseline and ≥6 months follow-up data.
- Stratified patients by transplant status, CKD progression, and baseline C3 levels; assessed CKD progression in stages 1-4.
Main Results:
- 260 C3G patients identified; 51.9% female, mean age 47.8 years, median follow-up 2.1 years.
- At diagnosis, most had CKD stage ≥3 (56.6%) and proteinuria (54.4%).
- Of those assessed, 59.0% progressed to a higher CKD stage, and 26.0% progressed to stage 5 or kidney failure within a median of 14.9 months.
Conclusions:
- Patients with C3G in the US present with advanced kidney disease at diagnosis.
- High rates of CKD stage progression observed, emphasizing the need for effective treatments.
- Findings highlight the urgent need for novel therapeutic strategies to improve outcomes for C3G patients.
Introduction:
C3 glomerulopathy (C3G) is a rare kidney disease caused by alternative pathway (AP) overactivation. This analysis describes demographic and clinical characteristics of patients with C3G in the United States (US) and evaluates chronic kidney disease (CKD) stage progression.
Methods:
This retrospective cohort study used electronic health records (EHRs) to identify patients aged ≥ 12 years with a C3G diagnosis (index) and with continuous clinical activity ≥ 12 months before (baseline) and ≥ 6 months after index (follow-up; until death or data end). Patients were stratified by prior kidney transplant status, CKD stage progression during follow-up, and baseline serum C3 level. CKD stage progression was assessed in patients with CKD stages 1 to 4 (at index) and adequate data to assess progression during follow-up.
Results:
Overall, 260 patients with C3G were identified: 51.9% were female; mean (SD) age was 47.8 (20.6) years; median follow-up was 2.1 years. Of those assessed, most had CKD stage ≥ 3 (56.6%) and proteinuria (54.4%) at index. Posttransplant recurrent C3G was reported in 27 patients (10.4%). Of those assessed for CKD stage progression, 102 (59.0%) and 45 (26.0%) progressed to a higher CKD stage or CKD stage 5 or kidney failure, respectively. Median time to progression of CKD stage or to stage 5 or kidney failure was 14.9 months and not estimable (NE), respectively.
Conclusion:
In a real-world US population of patients with C3G, we identified a population with both advanced kidney disease around the time of diagnosis and high rates of CKD stage progression, highlighting the need to utilize novel treatments to improve patient outcomes.
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