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

Katie A. Greenzang, Department of Pediatric Oncology, Dana-Farber Cancer Institute; Boston, MA; USA.
Kathleen E. Montgomery, UW-Madison School of Nursing; Madison, WI; USA.
Adam DuVall, Department of Medicine, University of Chicago, Chicago, IL; USA.
Michael E. Roth, Department of Pediatrics Patient Care, Division of Pediatrics, The University of Texas MD Anderson Cancer Center, Houston, TX; USA.
Mark Krailo, Department of Population and Public Health Sciences, University of Southern California, Los Angeles, CA; USA.
Michelle M. Nuño, Department of Population and Public Health Sciences, University of Southern California, Los Angeles, CA; USA.
Lindsay Renfro, Department of Population and Public Health Sciences, University of Southern California, Los Angeles, CA; USA.
Natalie DelRocco, Department of Population and Public Health Sciences, University of Southern California, Los Angeles, CA; USA.
John Doski, Departments of Surgery and Pediatrics, University of Texas Health Science Center, San Antonio; San Antonio, TX; USA.
Kara Kelly, Department of Pediatric Oncology, Roswell Park Comprehensive Cancer Center; Buffalo, NY; USA.
Sharon M. Castellino, Department of Pediatrics; Emory School of Medicine; Atlanta GA; USA.
Jennifer McNeer, Department of Pediatrics, University of Utah/Primary Children's Hospital, Salt Lake City, UT; USA.
Maureen M. O'Brien, Department of Pediatrics, Children's Hospital Colorado; University of Colorado Anschutz School of Medicine; Denver, CO; USA.
Damon Reed, Department of Pediatrics, Memorial Sloan Kettering Cancer Center; New York, NY; USA.
Katherine Janeway, Department of Pediatric Oncology, Dana-Farber Cancer Institute; Boston, MA; USA.
Pamela S. Hinds, Department of Nursing Science, Children's National Hospital, Washington, DC; USA.
Sue Zupanec, Department of Pediatric Oncology, The Hospital for Sick Children; Toronto, ON; Canada.
Susan K. Parsons, Tufts University School of Medicine; Boston, MA; USA.

Document Type

Journal Article

Publication Date

3-31-2025

Journal

Journal of the National Cancer Institute

DOI

10.1093/jnci/djaf083

Abstract

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).

Department

Pediatrics

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