Visual Acuity and Quality of Life Outcomes With Pegcetacoplan Treatment: A Post Hoc Analysis From the OAKS and DERBY Trials.

OBJECTIVE

To evaluate the effect of pegcetacoplan on visual function and patient quality of life (QoL) measures based on distance of geographic atrophy (GA) lesions from the center of the fovea.

DESIGN

Post hoc analysis from OAKS and DERBY, two global, 24-month, multicenter, randomized, double-masked, sham-controlled phase 3 studies evaluating pegcetacoplan treatment for GA in age-related macular degeneration (AMD).

PARTICIPANTS

888 study patients with GA for whom all data necessary for the post hoc analysis to be performed had been collected were included.

METHODS

Best-corrected visual acuity (BCVA) and 25-item National Eye Institute Visual Functioning Questionnaire (NEI VFQ-25) were assessed at baseline and every 4 and 6 months, respectively, to completion at month 24 in both pegcetacoplan-treated eyes and sham (observed) eyes. Eyes were stratified based on optical coherence tomography‒derived GA lesion location ≥250 µm (n = 192) or <250 µm (n = 696) from the foveal center.

MAIN OUTCOME MEASURES

Change from baseline in BCVA and NEI VFQ-25 was evaluated over 24 months in GA lesions based on distance (≥250 or <250 µm) from the foveal center. Adjustment was conducted via propensity score weighting, where model specification and baseline covariate selection were done a priori based on clinical rationale.

RESULTS

Pegcetacoplan-treated eyes with GA lesion margins ≥250 µm from the foveal center demonstrated directionally slower decline in visual acuity (mean +5.6 [SE 3.2] [P = .0785]) and QoL (mean +4.0 [SE 2.4] [P = .0905]) from baseline at 24 months compared with sham. The change in visual acuity (BCVA, mean -1.6 [SE 1.1] [P = .1522]) and QoL (NEI VFQ-25, -2.3 [1.1] [P = .0284]) in pegcetacoplan-treated eyes with GA lesion margins <250 µm from the foveal center were within the limits of variability compared with sham.

CONCLUSIONS

Pegcetacoplan treatment demonstrated a potentially meaningful slower decline in visual acuity and QoL for patients with GA lesions ≥250 µm from the foveal center. The change in visual acuity and QoL seen with pegcetacoplan treatment for patients with GA lesions <250 µm from the foveal center were within the limits of variability.