Abstract
Purpose :
To evaluate four patient-reported outcome (PRO) measures of patient symptoms, limitations, distress, and health-related quality of life (HRQOL) among patients with EYS-related retinal degeneration in a worldwide multicenter study (Pro-EYS) sponsored by the Foundation Fighting Blindness (FFB).
Methods :
Adult patients (N=103) with a diagnosis of EYS-related retinal degeneration were recruited prospectively and categorized into three cohorts based on disease severity (1: BCVA ≥20/80 and ≥10 degrees of visual field, 2: either 20/100≤BCVA≤20/400 or both BCVA≥20/80 and <10 degrees of visual field, 3: BCVA≤20/500). All patients completed three PROs: Michigan Retinal Degeneration Questionnaire (MRDQ), Patient-Reported Outcomes Measurement Information System 29 (PROMIS-29), and Visual System and Impact Outcomes PRO (ViSIO-PRO). Cohorts 1 and 2 completed the Veterans Affairs Low-Vision Visual Functioning Questionnaire-48 (VALVVFQ48). PROs were completed within 6 months of visual acuity, microperimetry, static perimetry, and rod isolated electroretinogram (ERG) testing for 70 patients. One-way ANOVA was used to compare the severity cohorts for mean domain scores of the PROs. Spearman correlations were performed between PRO domains and visual/retinal function assessments.
Results :
Of the study sample, 51% were female and the median age was 45 years. Domains of the MRDQ, ViSIO-PRO, and VALVVFQ48 were sensitive to differences among severity cohorts, while those of the PROMIS-29 were not (Table 1). MRDQ rod/peripheral retinal function domains correlated with static perimetry, full-field stimulus testing (FST) and rod isolated ERG, while macular/cone domains correlated with BCVA. The VALVVFQ48 visual motor domain correlated highly with BCVA (Table 2). Six of 7 PROMIS-29 domains exhibited floor effects/right skew.
Conclusions :
In this landmark study evaluating the utility of four PROs in a single retinal disease, domains pertaining to visual symptoms, visual function, and HRQOL were assessed for distribution characteristics and correlations with severity grading and visual/retinal function testing to assess construct validity. The study further supports the selection of specific PROs and their domains based on a priori hypotheses for inherited retinal disease clinical trials and natural history studies.
This abstract was presented at the 2023 ARVO Annual Meeting, held in New Orleans, LA, April 23-27, 2023.