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Aim <p>To determine which Patient-Reported Outcome Measures (PROM) tools were used in studies regarding Inherited Retinal Diseases (IRDs) in the last decade.</p> Subject and methods <p>A scoping review was conducted in PubMed and clinicaltrials.gov from 1st January 2015 to 20th May 2025. Reference lists of relevant studies were also looked at. Both interventional and observational studies regarding patients with IRDs were included. Studies not using a PROM tool were excluded.</p> Results <p>Eighty-one primary studies, 52 interventional and 29 observational, were reviewed, mostly reports of protocols from clinicaltrials.gov and only 12 completed study publications. An IRD-specific tool was used in fourteen of 81 (17.3%) studies. The most frequently used was the Michigan Retinal Degeneration Questionnaire (MRDQ) tool in 10 of 14 studies, followed by the Michigan Vision-related Anxiety Questionnaire (MVAQ) in 4 of 14 studies and the Visual Symptom and Impact Outcomes patient-reported outcome and observer-reported outcome (Visio-PRO/ ObsRO) in one study. The PROM tool most widely used was the National Eye Institute Visual Functioning Questionnaire-25 (NEI VFQ-25), in 38 of 81 (46.9%) studies. In 10 of 81 studies the outcome measure used was not specified. Thirty-four studies were initiated after 2023, when the publication of IRD-specific tools was available, yet 58% of them still used a non-IRD specific tool.</p> Conclusion <p>In the last decade the majority of interventional and observational studies regarding IRD patients, did not use an IRD-specific PROM tool. These tools, designed to describe the particular outcomes in IRD patients, are yet to become accepted and adopted by researchers.</p>

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Use of Patient-Reported Outcome Measures (PROMs) in interventional clinical trials and observational studies regarding Inherited Retinal Diseases (IRDs) -A scoping review

  • 期刊论文

  • 发表日期：

    2026年5月1日

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  • Ariadni Gavriilidou,
  • Theodora Gianni,
  • Athina Ververi,
  • Anna-Bettina Haidich,
  • Nikolaos Ziakas,
  • Zoi Tsimtsiou,
  • Asimina Mataftsi

摘要

Aim

To determine which Patient-Reported Outcome Measures (PROM) tools were used in studies regarding Inherited Retinal Diseases (IRDs) in the last decade.

Subject and methods

A scoping review was conducted in PubMed and clinicaltrials.gov from 1st January 2015 to 20th May 2025. Reference lists of relevant studies were also looked at. Both interventional and observational studies regarding patients with IRDs were included. Studies not using a PROM tool were excluded.

Results

Eighty-one primary studies, 52 interventional and 29 observational, were reviewed, mostly reports of protocols from clinicaltrials.gov and only 12 completed study publications. An IRD-specific tool was used in fourteen of 81 (17.3%) studies. The most frequently used was the Michigan Retinal Degeneration Questionnaire (MRDQ) tool in 10 of 14 studies, followed by the Michigan Vision-related Anxiety Questionnaire (MVAQ) in 4 of 14 studies and the Visual Symptom and Impact Outcomes patient-reported outcome and observer-reported outcome (Visio-PRO/ ObsRO) in one study. The PROM tool most widely used was the National Eye Institute Visual Functioning Questionnaire-25 (NEI VFQ-25), in 38 of 81 (46.9%) studies. In 10 of 81 studies the outcome measure used was not specified. Thirty-four studies were initiated after 2023, when the publication of IRD-specific tools was available, yet 58% of them still used a non-IRD specific tool.

Conclusion

In the last decade the majority of interventional and observational studies regarding IRD patients, did not use an IRD-specific PROM tool. These tools, designed to describe the particular outcomes in IRD patients, are yet to become accepted and adopted by researchers.