ArticleActa ophthalmologica2025
Artificial intelligence-quantified schisis volume as a structural endpoint for gene therapy clinical trials in X-linked retinoschisis.
Article in Acta ophthalmologica, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
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Who cites it
2 citing papers in PubMed.
- Randomised Clinical Trial to Evaluate the Efficacy of Acetazolamide for the Treatment of Cystoid Fluid Collections in X-Linked Retinoschisis: The AXIS Trial.Clinical & experimental ophthalmologyTrial
- Artificial intelligence-quantified schisis volume as a structural endpoint for gene therapy clinical trials in X-linked retinoschisis.Acta ophthalmologica · 2025Article
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11 authors.
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Abstract
purposeTo use artificial intelligence (AI) for quantifying schisis volume (ASV) in X-linked retinoschisis (XLRS) for use as a structural endpoint in gene therapy clinical trials.
methodsWe used data from Singapore, the United Kingdom, the Netherlands, and the United States. The AI model was developed on 250 optical coherence tomography (OCT) slices, with human annotation of schisis cavities (Dataset 1). ASV was quantified on Dataset 2 - 16 OCT scans from 8 eyes with XLRS at two time points, and Dataset 4 - 62 OCT scans from 31 eyes at two time points before and after carbonic anhydrase inhibitor (CAI) treatment. A clinical trial was simulated comparing CAI treatment against control. Changes in ASV, central subfield thickness (CST) and central foveal thickness (CFT) were compared. Effect size (Cohen's d) of the three structural endpoints was determined and used in sample size calculations for a future XLRS gene therapy clinical trial, at a 0.05 significance level and 80% power.
resultsIn the simulated clinical trial, all structural metrics showed greater reductions with intervention than with control, but only change in ASV reached statistical significance (p = 0.004). Cohen's d for ASV, CST and CFT were 0.972, 0.685 and 0.521, respectively. For the future gene therapy clinical trial, sample sizes required in each arm for ASV, CST and CFT were 18, 35 and 59 participants, respectively.
conclusionsASV measurements can track changes in schisis volume in response to treatment. As an endpoint, ASV has a greater statistical effect size than CST/CFT, which reduces sample size requirements for future XLRS gene therapy clinical trials.
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