Quality of Life (QoL) of Children and Adolescents Participating in a Precision Medicine Trial for High-Risk Childhood Cancer

SIMPLE SUMMARY: We assessed quality of life in patients participating in Australia’s national clinical trial of precision medicine for children with high-risk cancer. Quality of life measures aim to capture an individuals’ perceptions of physical, psychological, and social aspects of health. Knowledge of patient quality of life can help clinicians and parents make decisions in a setting where survival time must be weighed against patients’ experiences of illness and treatment. We found that most patients experienced compromised quality of life at the time of trial enrolment, typically in multiple domains. In patients whom we were able to follow-up after receipt of trial sequencing results, this did not change. We found an association between the outcomes of trial testing and patient quality of life which warrants unpacking in future research. Integrating collection of patient quality of life data into clinical processes would provide a more complete picture. ABSTRACT: Precision medicine is changing the treatment of childhood cancer globally, however little is known about quality of life (QoL) in children and adolescents participating in precision medicine trials. We examined QoL among patients enrolled in PRISM, the Zero Childhood Cancer Program’s precision medicine trial for high-risk childhood cancer. We assessed patient QoL via self-report (aged 12–17 years) and parent-proxy (aged 4–17 years) completion of the EQ-5D-Y. We analysed data using descriptive statistics and regression models. Patients (n = 23) and parents (n = 136) provided data after trial enrolment and following receipt of trial results and treatment recommendations (n = 8 patients, n = 84 parents). At enrolment, most patients were experiencing at least some difficulty across more than one QoL domain (81% patient self-report, 83% parent report). We did not find strong evidence of a change in QoL between timepoints, or of demographic or disease factors that predicted parent-reported patient QoL (EQ-VAS) at enrolment. There was strong evidence that receiving a treatment recommendation but not a change in cancer therapy was associated with poorer parent-reported patient QoL (EQ-VAS; Mdiff = −22.5, 95% CI: −36.5 to −8.5, p = 0.006). Future research needs to better understand the relationship between treatment decisions and QoL and would benefit from integrating assessment of QoL into routine clinical care.