ReviewOphthalmic genetics2020
Patient-reported outcome measures in inherited retinal degeneration gene therapy trials.
Review in Ophthalmic genetics, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 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
19 citing papers in PubMed.
- Pilot Study of Patient-Reported Outcomes in Stargardt Disease: Correlation With Functional and Structural Findings.Investigative ophthalmology & visual science · 2026Article
- Article
- Addressing Challenges in Developing Treatments for Inherited Retinal Diseases: Recommendations From the Third Monaciano Symposium.Translational vision science & technology · 2025Review
- Quantitative Choroidal Analysis of Molecularly Characterized Retinitis Pigmentosa.Investigative ophthalmology & visual science · 2025Article
- Relationship between the full-field stimulus test and self-reported visual function in patients with retinitis pigmentosa: REPEAT Study report No. 3.Acta ophthalmologica · 2025Article
- Inherited Retinal Degenerations and Non-Neovascular Age-Related Macular Degeneration: Progress and Unmet Needs.Translational vision science & technology · 2024Review
- Patient-Reported Outcomes in RLBP1 Retinal Dystrophy: Longitudinal Assessment in a Prospective Natural History Study.Translational vision science & technology · 2024Article
- Update on Clinical Trial Endpoints in Gene Therapy Trials for Inherited Retinal Diseases.Journal of clinical medicine · 2024Review
- Self-Reported Functional Vision in USH2A-Associated Retinal Degeneration as Measured by the Michigan Retinal Degeneration Questionnaire.Investigative ophthalmology & visual science · 2024Observational
- From the Formation of Conceptual Framework to Regulatory Decision-Making: Considerations for the Developments of Patient-Reported Outcome Instruments.Drug design, development and therapy · 2024Review
- Choroideremia: The Endpoint Endgame.International journal of molecular sciences · 2023Review
- The validation of inherited retinal disease-specific patient-reported outcome measures in adolescent patients.Ophthalmic genetics · 2023Article
- Beyond the NEI-VFQ: Recent Experience in the Development and Utilization of Patient-Reported Outcomes for Inherited Retinal Diseases.Cold Spring Harbor perspectives in medicine · 2023Review
- Therapeutic landscape for inherited ocular diseases: Current and emerging therapies.Singapore medical journal · 2023Review
- The State of Patient-Reported Outcome Measures for Pediatric Patients with Inherited Retinal Disease.Ophthalmology and therapy · 2022Review
- Challenges of Cost-Effectiveness Analyses of Novel Therapeutics for Inherited Retinal Diseases.American journal of ophthalmology · 2022Review
- The Natural History of Leber's Hereditary Optic Neuropathy in an Irish Population and Assessment for Prognostic Biomarkers.Neuro-ophthalmology (Aeolus Press) · 2022Article
- The Michigan Vision-Related Anxiety Questionnaire: A Psychosocial Outcomes Measure for Inherited Retinal Degenerations.American journal of ophthalmology · 2021Article
- The Michigan Retinal Degeneration Questionnaire: A Patient-Reported Outcome Instrument for Inherited Retinal Degenerations.American journal of ophthalmology · 2021Article
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
Abstract
Patient-reported outcome (PRO) measures have the potential to uniquely capture patient experience and serve as an outcome measure in inherited retinal degeneration (IRD) gene therapy trials. An IRD-specific patient-reported outcome measure may yield valuable information that has not been obtained from inherited retinal dystrophy gene therapy trials published to-date. Existing PRO measures have inherent limitations for use in IRD gene therapy trials. Developing an applicable patient-reported outcome measure for such trials needs to incorporate patient input from the target population, demonstrate sound psychometric properties, and be made in accordance with U.S. Food and Drug Administration (FDA) guidelines. This review will discuss the currently available PRO instruments, their limitations for IRD therapeutic trials, and suggestions for future PRO development in IRD populations. The PRO instruments highlighted were identified in PubMed search of English-language journals and previously published review articles.
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.