Assessing patient-reported outcomes (PROs) in paediatric oncology research: Which PRO would a pro pick, if a pro was picking PROs?

<h2>Abstract</h2> Patient-reported outcomes (PROs) enable the report of experiences that are only known to the patient, such as how an individual's symptoms, functioning, and quality of life are impacted by a health condition or treatment. The choice of PRO depends on the intended application and associated research questions, and structuring a rationale for the use of PRO data is key to deciding upon a PRO strategy. Rates of PRO use in paediatric oncology research remain low though the general use of PROs in clinical trials has been gradually increasing. This is likely to have risen due to increased emphasis by regulatory agencies to capture outcomes meaningful to patients. Despite increasing interest in PROs, a range of barriers to their use have been identified by researchers exploring their implementation and a pattern of under-reporting of PRO data has also been observed in general oncology trials. Research with children entails specific challenges, including the need for developmentally-appropriate outcome measures, the complexities of family involvement, and the adaptations required in research procedures and settings to accommodate children's physical, cognitive, and emotional development. With the nascent use of PROs in paediatric oncology, there are opportunities to benefit from learning from trials involving adult participants. The current paper outlines considerations for PRO selection for use in paediatric oncology research, describing five steps to determine the PRO study goals, the study outcomes, the reporter(s), measures, and study implementation. We discuss the breadth of applications of PROs in paediatric oncology research and potential for further learning and harmonisation with the aim of centering children's experiences.

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Publication Details

Journal
EJC Paediatric Oncology
Published
2025-04-24
DOI
https://doi.org/10.1016/j.ejcped.2025.100232
Citations
1
Primary Topic
Childhood Cancer Survivors' Quality of Life
Type
article
Field-Weighted Citation Impact
3.49
Controls
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article

Assessing patient-reported outcomes (PROs) in paediatric oncology research: Which PRO would a pro pick, if a pro was picking PROs?

Victoria Buenger, Jessica Roydhouse, Manuel Diezi, John Devin Peipert et al.
1 citations
EJC Paediatric Oncology
Childhood Cancer Survivors' Quality of Life
3.49
article

Assessing patient-reported outcomes (PROs) in paediatric oncology research: Which PRO would a pro pick, if a pro was picking PROs?

Victoria Buenger, Jessica Roydhouse, Manuel Diezi, John Devin Peipert, Bryce B. Reeve, Ameeta Retzer, Anne‐Sophie Darlington, Sarah Damery, Sarah Al-Jilaihawi, Shivani Bailey
article en
1 citations

Abstract

<h2>Abstract</h2> Patient-reported outcomes (PROs) enable the report of experiences that are only known to the patient, such as how an individual's symptoms, functioning, and quality of life are impacted by a health condition or treatment. The choice of PRO depends on the intended application and associated research questions, and structuring a rationale for the use of PRO data is key to deciding upon a PRO strategy. Rates of PRO use in paediatric oncology research remain low though the general use of PROs in clinical trials has been gradually increasing. This is likely to have risen due to increased emphasis by regulatory agencies to capture outcomes meaningful to patients. Despite increasing interest in PROs, a range of barriers to their use have been identified by researchers exploring their implementation and a pattern of under-reporting of PRO data has also been observed in general oncology trials. Research with children entails specific challenges, including the need for developmentally-appropriate outcome measures, the complexities of family involvement, and the adaptations required in research procedures and settings to accommodate children's physical, cognitive, and emotional development. With the nascent use of PROs in paediatric oncology, there are opportunities to benefit from learning from trials involving adult participants. The current paper outlines considerations for PRO selection for use in paediatric oncology research, describing five steps to determine the PRO study goals, the study outcomes, the reporter(s), measures, and study implementation. We discuss the breadth of applications of PROs in paediatric oncology research and potential for further learning and harmonisation with the aim of centering children's experiences.

EJC Paediatric OncologyVol. 5
Birmingham City University (GB), University of Tasmania (AU), Duke University (US), Patient-Centered Outcomes Research Institute (US), National Institute for Health and Care Research (GB), Cancer Research UK Clinical Trials Unit (GB), Coalition Against Childhood Cancer (US), NIHR Research Delivery Network (GB), NIHR Birmingham Biomedical Research Centre (GB), University of Southampton (GB), University of Birmingham (GB), University of Lausanne (CH)
Openalex Percentile: Top 10%
Childhood Cancer Survivors' Quality of Life
3.49
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