Janic Ana, Bowden Sylvie, Levy Sarah, Stinson Jennifer, Dimaras Helen
Faculty of Medicine, University of Toronto, 1 King's College Circle, Toronto, M5S 1A8, Canada.
Department of Ophthalmology & Vision Sciences, The Hospital for Sick Children, 555 University Ave, Toronto, M5G 2R3, Canada.
J Patient Rep Outcomes. 2020 Aug 8;4(1):66. doi: 10.1186/s41687-020-00232-7.
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.
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.
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).
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.
视网膜母细胞瘤是一种儿童期视网膜癌症，会带来诸如视力丧失和患第二种癌症风险增加等终身后果。患者报告结局测量工具（PROMs）是用于测量患者直接报告的与健康相关结局的工具。本研究的目的是确定视网膜母细胞瘤以及小儿眼科和小儿肿瘤学相关领域中使用的PROMs的范围、特征和质量。
在MEDLINE和Embase数据库中检索以英文发表的、报告了视网膜母细胞瘤、小儿肿瘤学或小儿眼科中使用的PROMs的研究；排除灰色文献和报告PROMs开发阶段的研究。PROMs按所测量的结构和评估的领域进行分组，并分为特定疾病或通用型。随后在MEDLINE和Embase中进行进一步检索，以查找评估已识别PROMs测量属性的研究。使用基于共识的健康测量工具选择标准（COSMIN）策略对有相关研究的PROMs进行方法学质量评估。
在数据库检索中发现的110项符合条件的研究中，识别出143种PROMs：1种是视网膜母细胞瘤特异性的，56种与眼科相关，86种与肿瘤学相关。所测量的最常见结构是“健康相关生活质量”，所评估的最常见领域是情绪健康。在这143种PROMs中，100种有相关的验证研究；1种视网膜母细胞瘤特异性PROM未经验证。质量评估显示，100种PROMs中有34种在“总体内容效度”的两个子类别中均获得了足够质量的评分；100种中有3种在“内部结构”的两个子类别中获得了足够质量的评分；100种中没有一种在“其余测量属性”的所有三个子类别中获得足够质量的评分。患者报告结局测量信息系统（PROMIS）儿科概况-25是所识别的得分最高的PROM，在2/3的测量属性类别（以及5/7的子类别）中符合COSMIN标准。另外还识别出11种PROMs，它们在1/3的测量属性类别（以及5/7的子类别）中有足够的评分。
该研究从小儿眼科和小儿肿瘤学文献中识别出几种可能与视网膜母细胞瘤患者相关的PROMs，但许多PROMs在验证方面存在局限性。与视网膜母细胞瘤患者合作开发特定于视网膜母细胞瘤的PROM，以支持最佳的内容效度，未来的发展可以首先专注于选择和定义要测量的最佳结构，随后可能对本研究中确定的具有强大方法学质量的相关PROMs进行改编和进一步验证。
