Use children’s feedback on symptoms, adverse events to improve cancer treatment

A new federal requirement in oncology is the inclusion of patient-reported drug- and device-related outcomes during oncology treatment. Federal agencies want evidence of the effect of cancer treatment directly from patients — their assessment of the symptoms and adverse events experienced during treatment. The patient reports are not designed to replace the traditional reporting of symptoms and adverse events by clinicians, but this new expectation signals a striking change: Federal agencies expect our increased attention to the patient’s experience of any given treatment.

At first consideration, this may seem to pose special challenges in pediatric oncology. However, the strong clinical and research consensus in pediatric oncology is that patients aged younger than 21 years (children and adolescents), who do indeed experience multiple symptoms and adverse events secondary to cancer and its treatment, are able to describe the symptoms and adverse events using qualitative and/or quantitative indicators. Although there are instances of clinicians and researchers questioning the ability of very young patients (5 to 6 years of age or younger) to validly report their experiences using standard measures, these instances are impressively rare and indeed may be related to the nature of the measures used and not the ability of the child to report.

Pamela S. Hinds

In multiple cross-sectional and longitudinal descriptive, in a limited number of interventional studies, children as young as 5 years of age and adolescents have demonstrated their ability to report their symptoms during cancer treatment.

Child and adolescent reports are now embedded as aims in clinical trials approved by the NCI and the Children’s Oncology Group or embedded in companion studies.

Part of our standard approach

Given that children and adolescents experience symptoms and adverse events and are able to report them to us, one issue we face is how to incorporate this information into our formal assessments of overall treatment impact. Currently, child and adolescent reports are not routinely collected except in clinical trials that specifically require these to be documented, nor are these reports systematically incorporated into treatment assessments, treatment decisions, or treatment summaries submitted to federal agencies or product companies. This important information remains primarily at the level of research data. Consequently, because clinicians do not routinely ask children and adolescents about their symptom and adverse event experiences and this population tends not to spontaneously report them, these troubling and occasionally debilitating experiences remain underreported and undertreated.

Johanna C. Menard

We in pediatric oncology must commit to routinely solicit symptom and adverse event reports from our patients, not for research purposes only, but as part of our standard approach to care and treatment impact reporting. In so doing, we would demonstrate our respect for their experiences and directly convey that their symptoms and adverse events are of significant importance as we actively engage in determining the best management approach of their disease. Finally, patients, even the young ones, must realize that their symptoms and adverse events contribute to our specialty’s understanding of their treatment and its outcomes in ways that will ultimately contribute to improved treatments for future patients.

Translate data into useful clinical guides

Important steps are under way in pediatric oncology to facilitate collection and documentation of patient-reported outcomes. We must achieve consensus on, and routinely use, a core set of measures that are age-appropriate and that bridge in valid and reliable ways with corresponding adult measures. Where necessary, we should complete testing of certain pediatric patient-report measures and make findings readily available for the scrutiny of scientific and clinical peer review. This will bring the child and adolescent voices directly into the offices of federal agencies that influence future treatment options for this age population.

Max J. Coppes

Subsequently, the pediatric oncology community will need to help the FDA, the NCI and industry to interpret the child and adolescent reports. We must provide evidence regarding when the child or adolescent’s report is an adjunct to the clinician report, when the two reports could be considered of equal importance, and when the ill child or adolescent’s report should be considered primary. Our evidence will be informed by the nature of the symptom or adverse event (eg, those who have a subjective dimension such as nausea, altered sleep or fatigue as compared with those who are laboratory-based, such as neutropenia or hypokalemia). We will also need to recommend whether patient age should be a consideration (eg, patient reports from adolescents could be considered primary, not adjunct, under certain circumstances, whereas the reports of younger children might be considered adjunct).

In our view, we have not taken patient reports far enough in pediatric oncology. Researchers have convincing data regarding the ability of children and adolescents, when asked, to validly report on their treatment experiences. Researchers and clinicians must jointly translate these data into useful clinical guides for actual care and examine how care outcomes change as a result of the data-based guidelines being available. And then, we must report these data in a standard fashion to federal agencies that sponsor clinical trials or that monitor the effect of pediatric oncology treatment. We ask and expect a great deal of children and adolescents during their cancer treatment. Now, let’s ask for and seriously consider their symptom and adverse event reports. Let’s take their reports much further in our science and in our care — indeed, as far as possible.

Max J. Coppes, MD, PhD, MBA, is senior vice president, Center for Cancer and Blood Disorders at Children’s National Medical Center; professor of oncology, medicine and pediatrics, Georgetown University; and clinical professor of pediatrics, George Washington University. He is also section editor for Childhood Cancer on the HemOnc Today Editorial Board.

Pamela S. Hinds, PhD, RN, FAAN, is director, Department of Nursing Research and Quality Outcomes; associate director of the Clinical and Community Research Center at Children’s National Medical Center; and professor of pediatrics, George Washington University.

Johanna C. Menard, BSN, RN, CPN, is a research nurse coordinator in the Department of Nursing Research and Quality Outcomes at Children’s National Medical Center, Washington, D.C.

For more information:

• FDA. Guidance for industry — Patient-reported outcomes measures: Use in medical product development to support labeling claims. Available at: www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM193282.pdf. Accessed June 6, 2011.
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