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Incorporation of patient-reported outcomes in pediatric cancer clinical trials: design, implementation, and dissemination

Cette étude examine les stratégies optimales permettant d'intégrer des outils de reporting et des méthodologies analytiques solides dans les essais cliniques destinés aux enfants, adolescents et jeunes adultes

Understanding the patient experience of treatment toxicities and their impact on health-related quality of life (HRQoL) of cancer treatments requires asking patients themselves using patient-reported outcomes (PROs). Over the past twenty years, the National Institutes of Health (NIH) sponsored several tools, namely Patient-Reported Outcome Measurement Information System (PROMIS) measures and the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) for precisely this purpose: to ensure valid, reliable tools to collect and detect patient-reported toxicities or adverse events and their impact on HRQoL. These PRO measures have been widely incorporated in clinical trials for adults with cancer. Yet, despite considerable work developing and validating developmentally appropriate versions of these measures for pediatric and adolescent self-report, PRO inclusion in pediatric and adolescent and young adult (AYA) clinical trials has lagged. Here we discuss optimal strategies to integrate validated PRO tools and sound analytic methodologies in clinical trials for children and AYAs with cancer, highlighting lessons learned from recent successes and ongoing experiences developing and opening cross-network trials for children and AYAs through the Children’s Oncology Group (COG) for patients with classic Hodgkin lymphoma (cHL), osteosarcoma (OS), and acute lymphoblastic leukemia (ALL).

Journal of the National Cancer Institute , article en libre accès, 2025

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