Sofia Teixeira , Maria Franca , Nuno Gouveia , Bruno Teixeira , Rufino Silva , Joaquim Murta , João Pedro Marques
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Abstract
Purpose
Retinitis Pigmentosa (RP) is the most common inherited retinal disease (IRD). The Michigan Retinal Degeneration Questionnaire (MRDQ) is a validated patient-reported outcome (PRO) measure designed to assess visual function in IRDs. This study aims to explore the association between functional and structural clinician-reported outcomes (CROs) and PROs in RP.
Design
Cross-sectional study
Methods
Study conducted at a Portuguese IRD referral center, including genetically confirmed RP patients from the IRD-PT registry (retina.com.pt). Collected clinical data encompassed demographics, genetic results, best-corrected visual acuity (BCVA), mean deviation (MD) and foveal sensitivity (FS) from automated static perimetry, ultra-widefield fundus autofluorescence (UW-FAF) patterns, and ellipsoid zone area measurements from spectral-domain optical coherence tomography (SD-OCT). Participants completed the Portuguese version of the MRDQ.
Results
The study included 72 RP patients (50% female, mean age 46.96±17.24 years) with disease-causing variants across twelve different genes. Median BCVA of the better-seeing eye was 71.50 (61.00, 80.00) ETDRS letters. A significant negative correlation (p<0.001) was found between BCVA and all MRDQ domains. MD and FS also correlated negatively (p<0.002) with several MRDQ domains. In structural measures, ellipsoid zone (EZ) area showed significant negative correlations with all MRDQ parameters (p<0.05) except scotopic function and photosensitivity. UW-FAF patterns showed a significant association with scotopic (p=0.02) and mesopic peripheral functions (p=0.01).
Conclusion
This study reveals a strong correlation between PRO measures and both functional and structural CROs in RP. The MRDQ is a valuable tool for understanding self-perceived visual function in RP and should be used in therapeutic trials and personalized low vision rehabilitation.
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