Long-term natural history of ellipsoid zone width in USH2A-retinopathy.
Br J Ophthalmol
; 2024 Aug 05.
Article
em En
| MEDLINE
| ID: mdl-39103200
ABSTRACT
AIMS:
To investigate the long-term natural history of ellipsoid zone (EZ) width in USH2A-retinopathy.METHODS:
EZ width measurements from optical coherence tomography were retrospectively obtained from 110 eyes of 55 participants with molecularly confirmed biallelic USH2A-retinopathy. We used a hierarchical Bayesian method to construct and compare different mathematical models describing the long-term decline of EZ width.RESULTS:
Compared with linear and quadratic models, exponential decline best represented the long-term loss of EZ width based on the deviance information criterion score. Log-transformed EZ width declined linearly over 30 years of inferred disease duration (median 0.063 (IQR 0.040-0.086) log (µm)/year). Compared with the raw EZ width decline rate, the log-transformed EZ width decline rate required 48% fewer patients to achieve an identically powered 1-year trial (38 vs 73 participants). Log EZ width decline rate was uncoupled from baseline EZ width (Spearman ρ=-0.18, p=0.06) and age (ρ=-0.10, p=0.31). Eyes with Usher syndrome exhibited earlier median onset ages of macular EZ width loss (18.8 (IQR 13.1-24.7) vs 28.1 (IQR 18.5-35.8) years, p<0.001) but comparable log EZ width decline rates (0.060 (IQR 0.035-0.100) vs 0.065 (IQR 0.050-0.079) log (µm)/year; p=0.42).CONCLUSIONS:
EZ width follows an exponential decline in USH2A-retinopathy. Compared with raw EZ width decline rate, log-transformed EZ width decline rate may be a superior endpoint for clinical trials. Syndromic eyes exhibit an earlier onset of macular EZ width loss but progress at comparable rates to non-syndromic eyes.
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Base de dados:
MEDLINE
Idioma:
En
Revista:
Br J Ophthalmol
Ano de publicação:
2024
Tipo de documento:
Article
País de afiliação:
Estados Unidos