| Title: |
PROGRESSION OF ABCA4-RELATED RETINOPATHY ; Prognostic value of demographic, functional, genetic, and imaging parameters |
| Authors: |
Müller, Philipp L.; Pfau, Maximilian; Treis, Tim; Pascual-Camps, Isabel; Birtel, Johannes; Lindner, Moritz; Herrmann, Philipp; Holz, Frank G. |
| Source: |
Retina ; volume 40, issue 12, page 2343-2356 ; ISSN 0275-004X |
| Publisher Information: |
Ovid Technologies (Wolters Kluwer Health) |
| Publication Year: |
2020 |
| Description: |
Purpose: To investigate the prognostic value of demographic, functional, genetic, and imaging parameters on retinal pigment epithelium atrophy progression secondary to ABCA4 -related retinopathy. Methods: Patients with retinal pigment epithelium atrophy secondary to ABCA4 -related retinopathy were examined longitudinally with fundus autofluorescence imaging. Lesion area, perimeter, circularity, caliper diameters, and focality of areas with definitely decreased autofluorescence were determined. A model was used to predict the lesion enlargement rate based on baseline variables. Sample size calculations were performed to model the power in a simulated interventional study. Results: Sixty-eight eyes of 37 patients (age range, 14–78 years) with a follow-up time of 10 to 100 months were included. The mean annual progression of retinal pigment epithelium atrophy was 0.89 mm 2 . The number of atrophic areas, the retina-wide functional impairment, and the age-of-onset category constituted significant predictors for future retinal pigment epithelium atrophy growth, explaining 25.7% of the variability. By extension of a simulated study length and/or specific patient preselection based on these baseline characteristics, the required sample size could significantly be reduced. Conclusion: Trial design based on specific shape-descriptive factors and patients' baseline characteristics and the adaption of the trial duration may provide potential benefits in required cohort size and absolute number of visits. |
| Document Type: |
article in journal/newspaper |
| Language: |
English |
| DOI: |
10.1097/iae.0000000000002747 |
| DOI: |
10.1097/IAE.0000000000002747 |
| Availability: |
https://doi.org/10.1097/iae.0000000000002747; https://journals.lww.com/10.1097/IAE.0000000000002747 |
| Accession Number: |
edsbas.B1B3F054 |
| Database: |
BASE |