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.