IMPORTANCE: Whether population-based racial and ethnic survival disparities for children with high-risk neuroblastoma persist in the clinical trial setting is unknown. OBJECTIVE: To investigate racial and ethnic survival disparities among children with high-risk neuroblastoma treated on frontline clinical trials. DESIGN, SETTING, AND PARTICIPANTS: This retrospective cohort study used data from Children's Oncology Group (COG) high-risk neuroblastoma trials from January 1, 2007, to December 31, 2016, with a data freeze on June 30, 2021. Children with high-risk neuroblastoma were analyzed in 2 cohorts: induction/consolidation trial participants and post-consolidation trial participants. Statistical analyses were performed from September 2, 2021, to December 30, 2024. EXPOSURES: Race and ethnicity were the primary exposures, categorized as Hispanic, non-Hispanic Black, non-Hispanic other (American Indian or Alaska Native, Asian, and Native Hawaiian or Other Pacific Islander), or non-Hispanic White. MAIN OUTCOMES AND MEASURES: Primary outcomes included overall survival (OS) and event-free survival (EFS) from time of trial enrollment, estimated by Kaplan-Meier methods. Associations with race and ethnicity were evaluated by log-rank tests and Cox proportional hazards regression models. Secondary outcomes included induction delays, early trial withdrawal, relapse as first event, death as first event, postrelapse OS, and early phase trial enrollment. RESULTS: The induction/consolidation cohort (median follow-up, 8.3 years [IQR, 6.1-9.8 years]) included 696 patients (404 males [58.1%]
79 Hispanic patients [11.4%], 109 non-Hispanic Black patients [15.7%], 27 patients of non-Hispanic other race [3.9%], and 481 non-Hispanic White patients [69.1%]). The post-consolidation cohort (median follow-up, 7.5 years [IQR, 5.8-9.4 years]) included 935 patients (567 males [60.6%]
87 Hispanic patients [9.3%], 145 non-Hispanic Black patients [15.5%], 41 patients of non-Hispanic other race [4.4%], and 662 non-Hispanic White patients [70.8%]). In multivariable Cox proportional hazards regression models, Hispanic children experienced significantly inferior OS (hazard ratio [HR], 1.78
95% CI, 1.25-2.53
P = .01) on induction/consolidation studies compared with non-Hispanic White children
EFS did not differ. Non-Hispanic Black (HR, 1.54
95% CI, 1.13-2.11) and Hispanic children (HR, 1.63
95% CI, 1.09-2.43) experienced inferior OS on post-consolidation studies compared with non-Hispanic White children (P = .009)
Hispanic children in post-consolidation studies experienced inferior EFS (HR, 1.68
95% CI, 1.14-2.47
P = .02). Death as first event and postrelapse OS also differed by race and ethnicity. CONCLUSIONS AND RELEVANCE: This study suggests that Black and Hispanic children with high-risk neuroblastoma experienced inferior OS despite uniform planned treatment on frontline COG clinical trials. Investigated mechanisms did not completely explain survival disparities. Future evaluation of disparate treatment-related toxicities and postrelapse care as explanatory mechanisms are key next steps to promote equity.