Progession of ABCA4-related retinopathy: prognostic value of demographic, functional, genetic, and imaging parameters
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 longitudin...
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| Hauptverfasser: | , |
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| Dokumenttyp: | Article (Journal) |
| Sprache: | Englisch |
| Veröffentlicht: |
December 2020
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| In: |
Retina
Year: 2020, Jahrgang: 40, Heft: 12, Pages: 2343-2356 |
| ISSN: | 1539-2864 |
| DOI: | 10.1097/IAE.0000000000002747 |
| Online-Zugang: | Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1097/IAE.0000000000002747 Verlag, lizenzpflichtig, Volltext: https://journals.lww.com/retinajournal/Abstract/2020/12000/PROGRESSION_OF_ABCA4_RELATED_RETINOPATHY_.12.aspx 
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| Verfasserangaben: | 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 |
| Zusammenfassung: | 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. |
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| Beschreibung: | Gesehen am 26.11.2021 |
| Beschreibung: | Online Resource |
| ISSN: | 1539-2864 |
| DOI: | 10.1097/IAE.0000000000002747 |