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Patient-reported outcome measures for retinoblastoma: a scoping review
Ana Janic1,2,3,4, Sylvie Bowden1,2, Sarah Levy2,3
1Faculty of Medicine, University of Toronto, 1 King's College Circle, Toronto, M5S 1A8, Canada.
Insights
This study reviewed patient-reported outcome measures (PROMs) for retinoblastoma, finding many PROMs lack sufficient validation. Future development should focus on creating a validated, retinoblastoma-specific PROM in partnership with patients.
Area of Science:
- Pediatric Oncology
- Ophthalmology
- Health Outcomes Research
Background:
- Retinoblastoma, a childhood eye cancer, has long-term effects including vision loss and secondary cancer risk.
- Patient-reported outcome measures (PROMs) assess patient-reported health outcomes.
- This study evaluated the scope, characteristics, and quality of PROMs in retinoblastoma and related pediatric fields.
Purpose of the Study:
- To determine the scope of PROMs used in retinoblastoma research.
- To characterize the identified PROMs by construct measured and domains assessed.
- To assess the methodological quality of PROMs relevant to retinoblastoma patients.
Main Methods:
- Searched MEDLINE and Embase for studies on PROMs in retinoblastoma, pediatric ophthalmology, and oncology.
- Excluded grey literature and studies on PROM developmental phases.
- Assessed PROM methodological quality using the Consensus-based standard for the Selection of health Measurement INstruments (COSMIN) strategy.
Main Results:
- Identified 143 PROMs from 110 studies; only one was retinoblastoma-specific and unvalidated.
- Health-related quality of life and emotional well-being were the most common constructs and domains.
- Few PROMs met COSMIN standards for measurement properties; PROMIS Pediatric Profile-25 scored highest.
Conclusions:
- Several PROMs from pediatric ophthalmology and oncology may apply to retinoblastoma, but validation is often limited.
- Developing a retinoblastoma-specific PROM with patient input is recommended.
- Prioritize adapting and validating existing high-quality PROMs for the retinoblastoma population.
Background:
Retinoblastoma is a childhood retinal cancer with lifelong consequences such as vision loss and increased risk of second cancer. Patient-reported outcome measures (PROMs) are instruments that measure outcomes related to health directly reported by patients. The purpose of this study was to determine the scope, characteristics and quality of PROMs used in retinoblastoma and related fields of pediatric ophthalmology and pediatric oncology.
Methods:
Databases MEDLINE and Embase were searched for studies in the English language that reported on PROMs used in retinoblastoma, pediatric oncology, or pediatric ophthalmology; grey literature and studies reporting on developmental PROM phases were excluded. PROMs were grouped by the construct measured and domains assessed, and classified as condition-specific or generic. A subsequent search was then conducted in MEDLINE and Embase for studies assessing measurement properties of the identified PROMs. PROMs with associated studies were assessed for their methodologic quality using the COnsensus-based standard for the Selection of health Measurement INstruments (COSMIN) strategy.
Results:
Among 110 eligible studies uncovered by the database searches, 143 PROMs were identified: one retinoblastoma-specific, 56 ophthalmology- and 86 oncology-related. The most common construct measured was 'health-related quality of life' and the most common domain assessed was emotional well-being. Of the 143 PROMs, 100 had associated validation studies; the one retinoblastoma-specific PROM was not validated. Quality assessment revealed 34/100 PROMs received a score of sufficient quality in both subcategories of 'overall content validity'; 3/100 received a score of sufficient quality in both subcategories of 'internal structure'; 0/100 received a score of sufficient quality in all three subcategories of 'remaining measurement properties'. The Patient-Reported Outcome Measure Information System (PROMIS) Pediatric Profile-25 was the highest-scoring PROM identified, meeting COSMIN standards for 2/3 measurement property categories (and 5/7 subcategories). Eleven additional PROMs were identified which had sufficient scores in 1/3 measurement property categories (and 5/7 subcategories).
Conclusion:
The study identified several PROMs from the pediatric ophthalmology and pediatric oncology literature that could be relevant to the retinoblastoma population, but many have limits to their validation. Future development of a retinoblastoma-specific PROM, performed in partnership with retinoblastoma patients to support optimal content validity, could first focus on the selection and definition of the optimal construct to measure, followed potentially by adaptation and further validation of the relevant PROMs with strong methodologic quality identified in this study.
