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Published on: February 15, 2022
Treatment options for C3 glomerulopathy
Carla M Nester1, Richard J Smith
1Departments of Internal Medicine and Pediatrics, University of Iowa, Iowa City, Iowa, USA. carla-nester@uiowa.edu
Insights
C3 glomerulopathy (C3G) shows limited response to traditional treatments. Emerging research suggests terminal complement blockade, like eculizumab, may offer therapeutic benefits for this complement-mediated renal disease.
Area of Science:
- Nephrology
- Immunology
- Complement System
Background:
- C3 glomerulopathy (C3G) is a rare kidney disease characterized by complement deposition.
- Understanding the alternative complement pathway's role is crucial for C3G pathogenesis.
Observation:
- Traditional immunosuppression and plasma therapy demonstrate limited efficacy in C3G.
- Emerging data suggests eculizumab, an anticomplement drug, may benefit some C3G patients.
Findings:
- C3G exhibits minimal responsiveness to conventional immunosuppressive therapies.
- Randomized controlled trials supporting C3G therapies are currently absent.
- Animal studies and case reports indicate terminal complement blockade may be advantageous.
Implications:
- Further research is needed to validate pathogenic mechanisms and disease spectrum in C3G.
- Genetic and biomarker research are essential for guiding rigorous clinical trials.
- Identifying effective therapies for C3G requires a deeper understanding of complement-mediated renal disease.
Purpose Of Review:
The purpose of this review is to discuss emerging nomenclature, review the salient clinicopathological features and describe the therapeutic options available for the treatment of C3 glomerulopathy (C3G).
Recent Findings:
C3G is minimally responsive to traditional immune suppression and randomized controlled trials to support therapy are absent. The burgeoning understanding of the role of the alternative complement pathway in C3G combined with animal data supporting the use of terminal complement blockade and a few reports suggesting that the anticomplement drug eculizumab may offer a therapeutic advantage have triggered great interest in the field of complement-mediated renal disease.
Summary:
Anticellular immune suppression and plasma therapy have limited efficacy in C3G. Data suggest that eculizumab may ameliorate disease in some C3G patients. The limited, recently published cohort data highlight crucial aspects of this group of diseases and support the need for extensive genetic and biomarker research to validate the pathologic mechanisms, delineate the spectrum of disease and guide the design of the rigorous trials to identify effective therapies for the treatment of C3G.
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