ReviewCold Spring Harbor perspectives in medicine2023
Beyond the NEI-VFQ: Recent Experience in the Development and Utilization of Patient-Reported Outcomes for Inherited Retinal Diseases.
Review in Cold Spring Harbor perspectives in medicine, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
9 citing papers in PubMed.
- Recovery of cone-mediated vision in Lebercilin associated retinal ciliopathy after gene therapy: One-year results of a phase I/II trial.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Trial
- Pilot Study of Patient-Reported Outcomes in Stargardt Disease: Correlation With Functional and Structural Findings.Investigative ophthalmology & visual science · 2026Article
- Approval of retinal gene therapies in the US and Europe based on visual acuity and microperimetry.Expert review of ophthalmology · 2025Article
- Article
- Inherited Retinal Degenerations and Non-Neovascular Age-Related Macular Degeneration: Progress and Unmet Needs.Translational vision science & technology · 2024Review
- Update on Clinical Trial Endpoints in Gene Therapy Trials for Inherited Retinal Diseases.Journal of clinical medicine · 2024Review
- Report From the Second Global Scientific Conference on Clinical Trial Design and Outcome Measures for RDH12-Associated Inherited Retinal Degeneration.Translational vision science & technology · 2024Article
- Self-Reported Functional Vision in USH2A-Associated Retinal Degeneration as Measured by the Michigan Retinal Degeneration Questionnaire.Investigative ophthalmology & visual science · 2024Observational
- Change in Cone Structure Over 24 Months in USH2A-Related Retinal Degeneration.American journal of ophthalmology · 2023Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Patient-reported outcome measures (PROMs) are tools designed to capture how a patient feels or functions, without the input or interpretation of anyone else. The earliest PROMs used in studies of inherited retinal diseases (IRDs) lack the validity required for therapy development today. The NEI-VFQ was one of the earliest PROMs developed using concept elicitation and cognitive debriefing of patients, but it lacks items that are common to patients with IRDs and it has poor measurement properties. Recent advances in PROM development include the Michigan Retinal Degeneration Questionnaire (MRDQ) and the ViSIO-PRO for nonsyndromic retinitis pigmentosa (RP), both of which have been qualitatively and quantitatively validated. As these new tools are used in clinical studies, they will generate additional evidence about their measurement characteristics. With the latest advances in PROM development for IRDs, it is now possible to move beyond the NEI-VFQ to measure what is truly important to patients.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.