Methodological resources to guide the collection of health-related quality of life data in paediatric cancer clinical trials: Scoping review

In paediatric oncology clinical trials, health-related quality of life data through patient-reported outcomes (PROs) provide valuable data on symptoms, functioning and quality of life during treatment. PROs can provide evidence on treatment tolerability and efficacy, facilitate regulatory review and cost-benefit profiling, and inform clinical decision making, treatment selection and post-treatment follow-up. Despite this, PROs are rarely implemented in paediatric oncology trials. This scoping review aimed to identify and describe the relevant methodological guidance available to support PRO implementation in paediatric oncology research. This included resources relating to adults who are less able to self-report (such as those with impaired capacity) where methodologies may apply to a paediatric population. Systematic searches were performed in Medline, Embase, PsycInfo and Web of Science from 2005 to 4th November 2024, alongside grey literature and citation hand-searching. Screening was performed independently by pairs of reviewers and data were summarised narratively. Twenty-nine articles were included. Eight articles (28%) provided a full methodological framework, whereas 21 articles (72%) provided a partial framework, addressing one or more aspects of PRO implementation. Fifteen articles (52%) specifically addressed a paediatric population; five (17%) concerned adults who were less able to self-report and nine (31%) were conducted in the general population with applicability to the paediatric oncology setting. Six methodological themes were identified: stakeholder involvement, measure selection, use of proxies, data collection methods, data management, and statistical analysis. Findings are presented and summarised. There is a need for a consensus-based approach to support PRO implementation in paediatric cancer clinical trials.