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Systemic complement activation levels in Stargardt disease.
Patty P A Dhooge1,2, Esmee H Runhart1,2, Catherina H Z Li1,2
1Department of Ophthalmology, Radboud University Medical Center, Nijmegen, The Netherlands.
Plos One
|June 25, 2021
Summary
Systemic complement activation was not elevated in Stargardt disease (STGD1) patients compared to controls, suggesting complement
Area of Science:
- Ophthalmology
- Immunology
- Genetics
Background:
- Preclinical research suggests the complement system is a common pathway in Stargardt disease (STGD1) and age-related macular degeneration (AMD), potentially causing retinal pigment epithelium (RPE) loss.
- However, systemic complement activation has not been previously assessed in STGD1 patients.
Purpose of the Study:
- To assess systemic complement activation in STGD1 patients.
- To investigate the association between systemic complement activation and STGD1 disease severity.
Main Methods:
- A cross-sectional case-control study compared systemic complement component C3 and its degradation product C3d levels between 80 STGD1 patients and 80 controls.
- The C3d/C3 ratio was used to measure systemic complement activation.
- Associations between the C3d/C3 ratio, demographic factors, and disease severity measures were analyzed within the STGD1 cohort.
Main Results:
- No significant difference in the C3d/C3 ratio was observed between STGD1 patients and controls.
- Elevated C3d/C3 ratios were found in three patients with concomitant inflammatory conditions.
- Within the STGD1 cohort, C3 levels were associated with sex and BMI.
Conclusions:
- Systemic complement levels are not elevated in STGD1 patients and do not correlate with disease severity.
- Complement activation in STGD1 may be a localized ocular process rather than systemic.
- Further research measuring intraocular complement levels in STGD1 patients is warranted, especially with the development of complement-targeted therapies.
