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

Understanding the patient experience of treatment toxicities and their impact on health-related quality of life from cancer treatments requires asking patients using patient-reported outcomes. Over the past 20 years, the National Institutes of Health has sponsored several tools - namely, Patient-Reported Outcomes Measurement Information System measures and the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events - for precisely this purpose: to ensure valid, reliable tools to collect and detect patient-reported toxicities or adverse events and their impact on health-related quality of life. These patient-reported outcomes 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, patient-reported outcomes inclusion in pediatric and adolescent and young adult clinical trials has lagged. Here, we discuss optimal strategies to integrate validated patient-reported outcomes tools and sound analytic methodologies in clinical trials for children and adolescent and young adults with cancer, highlighting lessons learned from recent successes and ongoing experiences developing and opening cross-network trials for children and adolescent and young adults through the Children's Oncology Group for patients with classic Hodgkin lymphoma, osteosarcoma, and acute lymphoblastic leukemia.