TY - JOUR
T1 - Retinal Pigment Epithelium and Outer Retinal Atrophy (RORA) in Retinitis Pigmentosa: Functional, Structural, and Genetic Evaluation
AU - Savastano, Maria Cristina
AU - Placidi, Giorgio
AU - Fossataro, Claudia
AU - Giannuzzi, Federico
AU - D'Onofrio, Nicola Claudio
AU - Hu, Lorenzo
AU - Cestrone, Valentina
AU - D’Agostino, Elena
AU - Biagini, Ilaria
AU - Paris, Leonardo
AU - Coppa, Gabriele
AU - Rizzo, Clara
AU - Kilian, Raphael
AU - Chiurazzi, Pietro
AU - Bertelli, Matteo
AU - Maltese, Paolo Enrico
AU - Falsini, Benedetto
AU - Rizzo, Stanislao
PY - 2024
Y1 - 2024
N2 - Purpose: To examine whether the extension of retinal pigment epithelium (RPE) and outer retinal atrophy (RORA) and various other morphofunctional parameters correlate with the genetic assessment and severity of retinitis pigmentosa (RP). Methods: Thirty-eight patients (76 eyes) with RP were prospectively enrolled and underwent full ophthalmic examination, including visual field testing, full-field electroretinography (ERG), and optical coherence tomography angiography. The severity of the disease was calculated using the RP stage scoring system, and the area of RORA was assessed using the automatically calculated area of sub-RPE illumination. Blood or saliva samples were collected from subjects, and DNA extraction was performed to evaluate genetic mutations and nucleotide and amino acid variations. Results: There was a statistically significant correlation between the extent of RORA and patient age, best-corrected visual acuity, ellipsoid zone extension, and disease severity in both eyes (each, P < 0.05). In contrast, RORA did not correlate with either the visual field or the ERG amplitude. Cumulative score and grade severity were both significantly correlated with superficial and deep capillary plexus density (both, P < 0.001) in both eyes. Evaluating RORA, we found genes with an overall less severe phenotype, such as EYS, PCDH15,andPRPF31, and those with a worse phenotype, such as RPGR. Conclusions: The correlation of RORA with structural, functional, and genetic assessment in RP disease leads us to consider RORA as a potential biomarker for prediction of disease stage. Multicenter studies are needed to confirm our findings. Translational Relevance: The morphofunctional and genetic correlations suggest a role for RORA in RP diagnosis and follow-up.
AB - Purpose: To examine whether the extension of retinal pigment epithelium (RPE) and outer retinal atrophy (RORA) and various other morphofunctional parameters correlate with the genetic assessment and severity of retinitis pigmentosa (RP). Methods: Thirty-eight patients (76 eyes) with RP were prospectively enrolled and underwent full ophthalmic examination, including visual field testing, full-field electroretinography (ERG), and optical coherence tomography angiography. The severity of the disease was calculated using the RP stage scoring system, and the area of RORA was assessed using the automatically calculated area of sub-RPE illumination. Blood or saliva samples were collected from subjects, and DNA extraction was performed to evaluate genetic mutations and nucleotide and amino acid variations. Results: There was a statistically significant correlation between the extent of RORA and patient age, best-corrected visual acuity, ellipsoid zone extension, and disease severity in both eyes (each, P < 0.05). In contrast, RORA did not correlate with either the visual field or the ERG amplitude. Cumulative score and grade severity were both significantly correlated with superficial and deep capillary plexus density (both, P < 0.001) in both eyes. Evaluating RORA, we found genes with an overall less severe phenotype, such as EYS, PCDH15,andPRPF31, and those with a worse phenotype, such as RPGR. Conclusions: The correlation of RORA with structural, functional, and genetic assessment in RP disease leads us to consider RORA as a potential biomarker for prediction of disease stage. Multicenter studies are needed to confirm our findings. Translational Relevance: The morphofunctional and genetic correlations suggest a role for RORA in RP diagnosis and follow-up.
KW - disease staging
KW - electroretinography
KW - multimodal imaging
KW - subretinal illumination
KW - retinal degeneration
KW - retinitis pigmentosa
KW - RORA
KW - OCT
KW - disease staging
KW - electroretinography
KW - multimodal imaging
KW - subretinal illumination
KW - retinal degeneration
KW - retinitis pigmentosa
KW - RORA
KW - OCT
UR - http://hdl.handle.net/10807/298987
U2 - 10.1167/tvst.13.8.44
DO - 10.1167/tvst.13.8.44
M3 - Article
SN - 2164-2591
VL - 13
SP - 1
EP - 9
JO - Translational Vision Science and Technology
JF - Translational Vision Science and Technology
ER -