Patient-reported outcome measures for retinoblastoma: a scoping review

Abstract

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.

Health-Related Quality of Life in Adolescent and Young Adult Retinoblastoma Survivors

“…The Multidimensional Scale of Perceived Social Support (MSPSS) is a 12-item questionnaire measuring an individual's perception of their social support (Gregory D. Zimet, Dahlem, Zimet, & Farley, 1988). The MSPSS has three subscales for social support (friends, family, and significant other) and uses a seven-point Likert-type scale ranging from Very Strongly Disagree (1) to Very Strongly Agree (7). The four items for each subscale are summed and divided by four.…”

“…However, sensitivity to the specific HRQOL effects of the disease may be lacking. There is no disease targeted HRQOL measure that has been psychometrically evaluated for use in the RB population [7]. Limitations in the methodologic quality of pediatric ophthalmology and oncology measures and applicability of content to the RB population have been identified [7].…”

Vision-related quality of life compared to generic measures in retinoblastoma survivors

“…The NIH Toolbox® VRQOL is a newer measure and has not been previously used in adolescent or the RB population. Only one previous study has assessed VRQOL in the adult RB population using the NEI–VFQ [ 5 ]. The NIH Toolbox® VRQOL measure was chosen due to the advantages of its IRT developmental basis over classical test theory [ 24 ] as well as the applicability of the questions to the adolescent RB population.…”

“…However, sensitivity to specific HRQOL effects of the disease may be lacking. There is no disease-targeted HRQOL measure that has been psychometrically evaluated for use in the RB population [ 5 ]. Limitations in the methodologic quality of pediatric ophthalmology and oncology measures and applicability of content to the RB population have been identified [ 5 ].…”

“…There is no disease-targeted HRQOL measure that has been psychometrically evaluated for use in the RB population [ 5 ]. Limitations in the methodologic quality of pediatric ophthalmology and oncology measures and applicability of content to the RB population have been identified [ 5 ]. However, it is uncertain how well a generic HRQOL measure captures the impact of RB compared to a VRQOL measure.…”