Progession of ABCA4-related retinopathy

• Verfasst von: Müller, Philipp Lothar [VerfasserIn]
• Verfasst von: Treis, Tim [VerfasserIn]
• Titel: Progession of ABCA4-related retinopathy
• Titelzusatz: prognostic value of demographic, functional, genetic, and imaging parameters
• Verf.angabe: Müller, Philipp L. MD; Pfau, Maximilian MD; Treis, Tim BSc; Pascual-Camps, Isabel MD; Birtel, Johannes MD; Lindner, Moritz MD; Herrmann, Philipp MD, PhD; Holz, Frank G. MD
• E-Jahr: 2020
• Jahr: December 2020
• Umfang: 14 S.
• Fussnoten: Gesehen am 26.11.2021
• Titel Quelle: Enthalten in: Retina
• Ort Quelle: Philadelphia, Pa. [u.a.] : Lippincott, Williams & Wilkins, 1981
• Jahr Quelle: 2020
• Band/Heft Quelle: 40(2020), 12 vom: Dez., Seite 2343-2356
• ISSN Quelle: 1539-2864
• DOI: doi:10.1097/IAE.0000000000002747
• Datenträger: Online-Ressource
• Sprache: eng
• K10plus-PPN: 1751180530

Abstract

To investigate the prognostic value of demographic, functional, genetic, and imaging parameters on retinal pigment epithelium atrophy progression secondary to ABCA4-related retinopathy. - 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. - 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 mm2. 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.