Abstract
Purpose :
To provide insights on GA treatment characteristics and high medical need phenotypes based on retina specialists’ (RS) opinions.
Methods :
GA, an advanced form of age-related macular degeneration (AMD), is a leading and currently untreatable cause of blindness. Despite multiple clinical trials, there is no consensus report on the smallest clinically-meaningful effect size of an intervention designed to slow the growth rate of GA. Retrospective data extracted from a broader-topic questionnaire were analyzed, comprising 90 RS located in 27 US states (90% private practice, 10% hospital or academic). RS were polled on (a) the smallest clinically meaningful effect size (SMES) of a treatment designed to slow progression of GA, (b) which GA phenotypes would benefit from treatment, and (c) preferred route of administration. Descriptive statistics are presented.
Results :
Median SMES slowing of GA growth rate was 20% (SD: 0.12, Range: 1-50%) (Figure 1). 87%, 80% and 63% of RS agreed that patients with “Bilateral GA with foveal sparing in 1+ eyes”, “GA with visual acuity >20/100”, or “Unilateral GA with CNV in fellow eye,” respectively, would most benefit from the slowing of GA by SMES. 73% and 53% of RS agreed that patients with “GA with visual acuity <20/200” and “GA without foveal sparing,” respectively, would not benefit from a treatment that slows GA by SMES (Figure 2). 57% of RS expressed that they would favor a single treatment modality independent of GA phenotype; of those RS and assuming equal safety and efficacy profiles, 86% preferred an oral treatment, while 9% and 5% preferred an intraocular device or monthly intravitreal injections, respectively.
Conclusions :
Data presented here may be used to understand RS' opinions on treatment efficacy and to optimize clinical trial design. RS responses reveal that what appears to be a numerically modest slowing of GA progression is nonetheless considered clinically meaningful. Despite the fact that GA is not strongly correlated with visual acuity, slowing GA growth, especially near the fovea, may benefit patients due to the threat posed to visual acuity by juxtafoveal atrophic lesions.
This abstract was presented at the 2019 ARVO Annual Meeting, held in Vancouver, Canada, April 28 - May 2, 2019.