Abstract
Purpose :
Outer retinal tubulation (ORT) has been identified as a biomarker for advanced Age-Related Macular Degeneration (AMD); however, the clinical implications are not well understood. This analysis examined the prevalence of ORT over time and association with GA lesion growth among patients in FILLY, a Phase 2 clinical trial investigating the C3 inhibitor pegcetacoplan in GA secondary to AMD.
Methods :
The inclusion criteria for the analysis were patients randomized to sham treatment in the FILLY trial with a baseline Spectralis optical coherence tomography (OCT) measurement from which the presence of a fully formed ORT could be established, and at least one post-baseline GA lesion assessment via fundus autofluorescence (FAF) for which the lesion area could be measured (n=62). The analysis included calculation of the prevalence of ORT at baseline, Month 12, and Month 18 for patients that had suitable Spectralis OCT images available and descriptive summaries of the change in GA lesion size (mm2) as measured by FAF over 18 months.
Results :
Patients with a fully formed ORT at baseline and/or Month 12 had a mean change from baseline GA lesion area (mm2) of 2.73 (SE: 0.34; 95% CI [2.02,3.43]) and a mean percentage change from baseline of 37.4% (SE:6.85%; 95% CI [23.26, 51.54]) at Month 18. Patients without an ORT present at both baseline and Month 12 had a mean change from baseline GA lesion area (mm2) of 3.61 (SE: 0.470; 95% CI [2.65,4.58]) and a mean percentage change from baseline of 50.1% (SE:6.27%; 95% CI [37.23, 62.98]) at Month 18. ORT prevalence rates were 24.19% (15/62) at baseline, 42.86% (24/56) at Month 12, and remained 42.86% (24/56) at Month 18.
Conclusions :
The results indicate a trend toward slower lesion growth in patients with a fully formed ORT present at baseline and/or 12 months compared to patients without the presence of an ORT. Clinicians and clinical trialists evaluating patients with GA may consider these findings when assessing progression risk.
This abstract was presented at the 2023 ARVO Annual Meeting, held in New Orleans, LA, April 23-27, 2023.