Purpose This study assessed the responsiveness to change over time and theorized associations of Patient‐Reported Outcomes Measurement Information System (PROMIS) pediatric measures in children and adolescents in treatment for cancer to determine measure readiness for use in cancer clinical trials. Methods We administered eight PROMIS (three symptom, two psychological, and three performance) pediatric short‐form measures and the Symptom Distress Scale (SDS) to 96 pediatric oncology patients at three time points during a course of chemotherapy. We assessed responsiveness using paired t tests and generalized estimating equation (GEE) models, calculated standardized response mean (SRM) values for PROMIS measures, and examined scores over three data points (T1–T3). Guided by the theory of unpleasant symptoms (TOUS), we examined associations among the PROMIS measures, the SDS, and other variables using GEE. Results The paired t tests showed statistically significant changes in two psychological measures and one performance measure from T1 to T2; three symptom, two psychological and two performance measures from T2 to T3; and three symptom and two psychological measures from T1 to T3. Findings from GEE models indicate PROMIS pediatric measures had statistically significant short‐term and long‐term changes, controlling for demographic and clinical variables. One performance measure did not achieve significant change at any time point. We found positive support for theorized relationships in the TOUS. Conclusions Most of the PROMIS pediatric measures demonstrated changes over time and had significant relationships as theorized, thus supporting concurrent and construct validity of these measures when administered to pediatric oncology patients during a course of chemotherapy. This evidence supports the measures’ readiness for use in clinical trials.

Children experiencing troubling symptoms during cancer treatment are heterogeneous. With respect to the PROMIS symptom measures, two a priori unknown distinct latent profiles of patients were identified in a course of chemotherapy, and the transitions in the profile status were significantly predicted by a baseline single-item fatigue measure.

Background Pediatric participants on Phase I or Phase II clinical trials for incurable cancer are at risk of experiencing toxicities (adverse events, AEs) related to trial participation. Multiple AEs are subjective; thus, the real impact of trial treatment cannot be known unless patient subjective reports are solicited. Methods We assessed the feasibility and acceptability of soliciting symptom, function and quality of life (QoL) reports from 8- to 18-year-old participants enrolled on Phase I/II clinical trials at four cancer centers during the first course of chemotherapy. We also assessed the reliability and validity of six self-report PROMIS pediatric measures and four open-ended interview questions at two time points (trial enrollment, T1 and 3 to 4 weeks later, T2). Results The enrollment rate of 75.9% (n=20) exceeded our feasibility criterion, and missingness of measures by person, measure and items at T1 and T2 were lower than our acceptability criteria. New QoL themes were limited to the impact of treatment on families and being away from home, family and friends for treatment. All but one measure at T1 met the reliability criterion and all did at T2. Validity support was limited though as theorized, mobility decreased and fatigue increased as AEs increased. Conclusions Soliciting and documenting symptom, function and QoL reports from 8- to 18-year-olds enrolled on a Phase I/II clinical trial is feasible and acceptable to participants, particularly when embedded in trials. Reliable and valid findings can result, making patient self-reported outcomes a possible new trial endpoint.

Nurses have psychometrically strong options for measuring cancer-related fatigue in pediatric patients with cancer, but the PROMIS Pediatric Fatigue Short Form is applicable to more age groups.

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