Does early phase study enrollment for pediatric oncology patients have an impact on symptom burden and quality of life (QOL)? A national Canadian study report.

10023 Background: There are few therapeutic options for pediatric patients with relapsed or refractory cancers. The impact of early phase trial enrolment on the quality of life (QOL) and symptom burden in this patient population is unknown. We hypothesized that this information could inform future clinical trial design and would be of value to clinicians, patients and their families. The primary aim was to determine if those enrolled on a phase I or II trial had improved QOL measured using the PedsQL 3.0 Acute cancer Module compared to those eligible, not not enrolled on a trial. Methods: In this Canadian multi-site study, we included patients 2-18 years of age who were enrolled on a phase I/II clinical trial and those who would have been eligible but did not enroll. The PedsQL 3.0 Acute Cancer Module was completed at baseline, week 4 and week 8 by guardians and by patients, for those age 5 and older. The PedsQL 3.0 Acute Cancer Module total scores were compared separately for patients and guardians using a mixed linear registration model with a random effect for a patient. Results: Of the 80 patients, 31 (39%) patients were enrolled on an early phase trial and 49 (61%) were not enrolled. The majority of patients had a brain tumor (63.3% of those enrolled; 32.7% of those not), followed by a bone tumor (16.7% of those enrolled; 24.5% of those not). In both groups, the majority of patient were included at the time of their first relapse (64.5% of those enrolled and 69.9% of those not). No significant difference was found between PedsQL3.0 patients or guardians; p = 0.09). The PedsQL3.0 total scores were significantly higher for patients enrolled on an early phase trial compared to those patients not enrolled (p = 0.01). However, after adjusting for baseline levels, the difference between enrolled patients vs not was not statistically significant (p = 0.8). Conclusions: Being enrolled on an early phase trial does not appear to have a negative effect on one's quality of life. The results have also demonstrated that it is feasible to evaluate patients enrolled and not enrolled on early phase trials and compare their symptom experience using the PedsQL 3.0 Acute cancer Module. Continued efforts will focus on more recruitment and using SSPedi to further investigate differences in QOL. This research is supported through C17 Council, Kindred Foundation and the CHEO Research Institute.