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Asymmetric Inter-Eye Progression in Stargardt Disease
Stanley Lambertus1, Nathalie M Bax1, Joannes M M Groenewoud2
1Department of Ophthalmology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, Nijmegen, The Netherlands.
Investigative Ophthalmology & Visual Science
|December 22, 2016
Summary
Asymmetry in Stargardt disease (STGD1) progression varies by age of onset and ABCA4 variant severity. For therapeutic trials, select early-onset STGD1 patients with severe ABCA4 variants and no baseline asymmetry.
Area of Science:
- Ophthalmology
- Genetics
- Retinal Diseases
Background:
- Stargardt disease (STGD1) can exhibit asymmetric progression between eyes.
- This asymmetry complicates the design of therapeutic trials, particularly those using fellow-eye controls.
- Understanding inter-eye discordance is crucial for optimizing trial design and interpreting results.
Purpose of the Study:
- To investigate the inter-eye discordance in best-corrected visual acuity (BCVA) and retinal pigment epithelium (RPE) atrophy progression in STGD1.
- To identify factors influencing this discordance, including age of onset and ABCA4 variant pathogenicity.
- To provide recommendations for selecting patient cohorts in STGD1 therapeutic trials.
Main Methods:
- Retrospective cohort study of 68 STGD1 patients (136 eyes) with ABCA4 variants and follow-up data.
- Analysis of inter-eye correlations of RPE atrophy progression across different onset groups (early, intermediate, late).
- Statistical comparison of discordant baseline BCVA and RPE atrophy with discordant progression using odds ratios.
Main Results:
- Moderate correlation in RPE atrophy progression between eyes (ρ = 0.766), decreasing with later onset and lower ABCA4 variant pathogenicity.
- 17.6% of patients showed discordant inter-eye RPE atrophy progression.
- Discordant progression was associated with baseline RPE atrophy asymmetry (OR, 6.50), but not baseline BCVA asymmetry (OR, 0.33).
Conclusions:
- Inter-eye correlation of STGD1 progression is lower in late-onset cases and those with less pathogenic ABCA4 variants.
- Minimizing inter-eye discordance in early trials requires selecting early-onset STGD1 patients with severe ABCA4 variants and no baseline asymmetry.
- This approach can maximize statistical power in therapeutic interventions for STGD1.
