Structural and functional clinician-reported outcomes show strong correlations with patient-reported outcomes in Retinitis Pigmentosa

AJO International Pub Date : 2025-02-09 DOI:10.1016/j.ajoint.2025.100105

Sofia Teixeira , Maria Franca , Nuno Gouveia , Bruno Teixeira , Rufino Silva , Joaquim Murta , João Pedro Marques

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引用次数: 0

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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临床报告的结构和功能结果与色素性视网膜炎患者报告的结果有很强的相关性

目的色素性视网膜炎（RP）是最常见的遗传性视网膜疾病。密歇根视网膜变性问卷（MRDQ）是一种经过验证的患者报告结果（PRO）测量，旨在评估ird的视觉功能。本研究旨在探讨RP的功能和结构临床报告结果（cro）与PROs之间的关系。设计横断面研究方法在葡萄牙IRD转诊中心进行的研究，包括来自IRD- pt登记处（retina.com.pt）的遗传确诊RP患者。收集的临床数据包括人口统计学、遗传结果、最佳矫正视力（BCVA）、平均偏差（MD）和中央凹灵敏度（FS），这些数据来自自动静态视野测量、超宽视场眼底自身荧光（UW-FAF）模式，以及光谱域光学相干断层扫描（SD-OCT）的椭球区面积测量。参与者完成了葡语版的MRDQ。结果本研究纳入72例RP患者（50%为女性，平均年龄46.96±17.24岁），12种不同基因的致病变异。视力较好的眼的中位BCVA为71.50 (61.00,80.00)ETDRS字母。BCVA与所有MRDQ域呈显著负相关（p<0.001）。MD和FS也与多个MRDQ域呈负相关（p<0.002）。在结构测量中，除暗函数和光敏性外，椭球区（EZ）面积与所有MRDQ参数均呈显著负相关（p < 0.05）。UW-FAF模式与暗观（p=0.02）和介观外周功能（p=0.01）有显著相关性。结论本研究揭示了PRO测量与RP的功能性和结构性cro之间的相关性。MRDQ是了解RP患者自我感知视觉功能的一个有价值的工具，应用于治疗试验和个性化的低视力康复。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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AJO International

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