Early phase study enrollment in Canadian children with cancer near end of life: A retrospective cohort study from Cancer in Young People in Canada

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BACKGROUND: Little is known about children with cancer who participate in early phase (phase 1/2) oncology trials toward the end of life (EOL). The authors sought to describe the proportion of Canadian children with cancer who participate in trials in their last 90 days, explore predictors of enrollment, describe these trials and key EOL outcomes. METHODS: This retrospective cohort study used data from the Cancer in Young People in Canada (CYP-C) database, a national population-based registry. The study included Canadian children and adolescents with cancer 0-20 years old at death, who died from 2001 to 2021. We performed logistic regression to explore the relationships between diagnosis-related factors, treatment-related factors, demographic factors, and study enrollment. RESULTS: Of 3125 children and adolescents who died, 140 (4.5%) met study criteria. Patients with leukemia or lymphoma (odds ratio [OR], 0.54; 95% confidence interval [CI], 0.33-0.87) or a solid tumor (OR, 0.48; 95% CI, 0.29-0.79) enrolled less frequently than those with central nervous system tumors. Previous trial enrollment (OR, 3.02; 95% CI, 2.43-3.77), and initial treatment in a major early phase study center (OR, 1.65; 95% CI, 1.14-2.4) were associated with enrollment. Patients in the lowest census-based income quintiles (quintiles 1-2) enrolled less frequently than those in the highest quintiles (3-5) (OR, 0.56; 95% CI, 0.37-0.84). CONCLUSIONS: A small proportion of children and adolescents participate in early phase trials toward EOL. Diagnosis, treatment-related, and socioeconomic factors are associated with non-enrollment. Clinicians, researchers, and policy developers should consider and address the possible impact of socioeconomic and other factors on access to studies, to ensure equitable access for diverse populations.

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