Related Experiment Video
Updated: Aug 26, 2025
07:35
In Vivo Vascular Injury Readouts in Mouse Retina to Promote Reproducibility
Published on: April 21, 2022
2.2K
Beyond the NEI-VFQ: Recent Experience in the Development and Utilization of Patient-Reported Outcomes for Inherited
Todd Durham1, Judit Banhazi2, Francesco Patalano2
1Foundation Fighting Blindness, Columbia, Maryland 21045, USA.
Cold Spring Harbor Perspectives in Medicine
|October 11, 2022
Summary
New patient-reported outcome measures (PROMs) like the MRDQ and ViSIO-PRO offer validated tools for inherited retinal diseases (IRDs). These advancements enable better measurement of patient experiences beyond older, less suitable instruments.
Area of Science:
- Ophthalmology and Vision Science
- Patient-Reported Outcome Measures (PROMs)
- Inherited Retinal Diseases (IRDs)
Background:
- Patient-reported outcome measures (PROMs) are crucial for assessing patient well-being and function in clinical research.
- Early PROMs for inherited retinal diseases (IRDs), such as the NEI-VFQ, exhibit limitations in relevance and measurement properties for current therapy development.
- The NEI-VFQ, despite early development using patient input, lacks specific items pertinent to IRDs and demonstrates suboptimal psychometric characteristics.
Purpose of the Study:
- To highlight the evolution and current state of PROMs specifically designed for inherited retinal diseases (IRDs).
- To introduce and validate newer, more appropriate PROMs for IRDs, moving beyond the limitations of earlier instruments.
- To emphasize the importance of using validated PROMs that capture patient-centered outcomes in IRD research and therapy development.
Main Methods:
- Review of existing literature on PROMs used in inherited retinal disease (IRD) research.
- Identification and description of recently developed and validated PROMs, including the Michigan Retinal Degeneration Questionnaire (MRDQ) and ViSIO-PRO.
- Emphasis on the qualitative and quantitative validation processes undertaken for these new instruments.
Main Results:
- The Michigan Retinal Degeneration Questionnaire (MRDQ) and ViSIO-PRO have been developed and validated for nonsyndromic retinitis pigmentosa (RP).
- These newer PROMs demonstrate improved relevance and measurement properties compared to earlier tools like the NEI-VFQ for IRDs.
- Ongoing use in clinical studies will further establish the measurement characteristics of MRDQ and ViSIO-PRO.
Conclusions:
- Recent advancements in PROM development allow for the use of more suitable and validated tools in IRD research.
- The MRDQ and ViSIO-PRO represent significant progress, enabling better assessment of patient-reported outcomes in IRDs.
- It is now feasible to transition from outdated instruments to modern PROMs that accurately reflect patient experiences and priorities in IRDs.
