Data Show Racial/Ethnic Disparities in Pediatric Brain Cancer Survival

“The findings show that access to clinical trials alone is insufficient to overcome the inferior survival outcomes experienced by Black and Hispanic children with cancer,” according to lead study author Puja Umaretiya, MD, MS.

Despite uniformly planned treatment across frontline Children’s Oncology Group (COG) clinical trials, pediatric patients who were Black and Hispanic with high-risk neuroblastoma experienced worse overall survival (OS) outcomes, according to findings from a retrospective cohort study published in JAMA Network Open.¹

Among patients who were included in induction/consolidation trials, the 5-year OS rate was 47.2% and 61.3% for patients who were Hispanic and White, respectively (HR, 1.55; 95% CI, 1.11-2.15; P = .047). Per multivariate analysis adjusting for factors including MYCN amplification, tumor histology, and International Neuroblastoma Staging System (INSS) stage, investigators noted worse OS outcomes for Hispanic populations compared with those who were White (HR, 1.78; 95% CI, 1.25-2.53; P = .01).

Regarding patients who participated in post-consolidation setting trials, univariate analysis revealed significantly worse event-free survival (EFS) outcomes (P = .01) for patients who were non-Hispanic Black (HR, 1.37; 95% CI, 1.05-1.79) and Hispanic (HR, 1.56; 95% CI, 1.14-2.15) compared with those who were White. When adjusting for tumor histology, INSS stage, and end-of-induction (EOI) disease response, multivariate analysis showed significantly worse EFS outcomes for patients who were Hispanic than those who were White (HR, 1.68; 95% CI, 1.14-2.47; P = .02).

Five-year OS rates were 67.7% for patients who were Black, 62.2% for patients who were Hispanic, and 75.7% for patients who were White. Compared with White populations, data showed significantly worse OS outcomes per univariate analysis (P <.001) for patients who were Black (HR, 1.65; 95% CI, 1.23-2.22) and Hispanic (HR, 1.73; 95% CI, 1.21-2.47). Additionally, multivariate analysis adjusting for stage and EOI response revealed significantly shorter OS (P = .009) for Black (HR, 1.54; 95% CI, 1.13-2.11) and Hispanic populations (HR, 1.63; 95% CI, 1.09-2.43) compared with patients who were White.

“The findings show that access to clinical trials alone is insufficient to overcome the inferior survival outcomes experienced by Black and Hispanic children with cancer,” lead study author Puja Umaretiya, MD, MS, assistant professor of Pediatrics in the Division of Hematology and Oncology and a member of the Harold C. Simmons Comprehensive Cancer Center at UT Southwestern Medical Center, stated in a press release on these findings.² “While further work is needed to understand disparate survival outcomes, any efforts to improve outcomes for children with cancer will likely need to focus on health-related social needs, which are highly prevalent and may contribute to worse outcomes in Black and Hispanic children.”

Investigators of this retrospective study collected data from COG high-risk neuroblastoma trials conducted between January 1, 2007, and December 31, 2016; a data freeze occurred on June 30, 2021. The study included 2 patient cohorts: those who participated in an induction or consolidation setting trial, and those who participated in a post-consolidation setting trial.

The primary exposures of interest were race and ethnicity, with investigators categorizing patients as Hispanic; non-Hispanic Black; non-Hispanic other, which included those who were American Indian or Alaska Native, Asian, and Native Hawaiian or other Pacific Islander; and non-Hispanic White. The study’s primary end points were EFS and OS. Secondary end points were care delivery points believed to have mechanically impacted disparate survival outcomes, which included factors like induction delay, early trial withdrawal, relapse or progression as first event, and early phase trial enrollment among those experiencing relapse or progression.

The induction/consolidation cohort included 696 patients who were mostly White (n = 481) followed by Black (n = 109), Hispanic (n = 79), and other races (n = 27). Disproportionate household poverty exposure (P <.001) was noted in patients who were Hispanic (58.2%) and Black (50.5%) compared with those who were White (25.2%).

Investigators included 935 patients in the post-consolidation cohort, with most being White (n = 662) followed by Black (n = 145), Hispanic (n = 87), and other races (n = 41). Correlations between race and ethnicity and poverty and rurality measures in the post-consolidation cohort mirrored those in the induction/consolidation cohort.

No significant differences in induction therapy duration or early trial withdrawal by race and ethnicity occurred. Additionally, investigators observed no significant differences by race and ethnicity for relapse and progression across the induction/consolidation and post-consolidation trials.

The 5-year cumulative incidence of death in induction/consolidation trials was 5.9%, 4.6%, 9.5%, and 16.2% for patients who were White, Black, Hispanic, and other races, respectively, which revealed significant differences by race and ethnicity (P = .049). Across the post-consolidation trials, the 5-year cumulative incidence of death was 0.8%, 5.9%, 2.5%, and 2.4% in each racial and ethnic population (P = .001).

OS from time of first relapse showed significant differences across racial and ethnic populations (P = .04). No significant differences by race and ethnicity were noted for the characteristics of patients who relapsed or for enrollment on early phase trials after relapse.

“These data identify the need for studies focused in 2 areas. First, we need to be thoughtful about the data we collect on clinical trials to understand why marginalized groups do not experience the same outcomes,” Umaretiya stated.² “Collecting more specific data, such as social determinants of health, may provide insight into the underlying causes. Simultaneously, we need to embed supportive care interventions for groups at risk for worse outcomes despite standardized therapies.”

References

1. Umaretiya PJ, Naranjo A, Zhang FF, et al. Racial and ethnic survival disparities among children with high-risk neuroblastoma: a Children’s Oncology Group report. JAMA Network Open. 2025;8(2):e2458531. doi:10.1001/jamanetworkopen.2024.58531
2. Differences in survival persist despite access to cancer clinical trials. News release. UT Southwestern Medical Center. April 29, 2025. Accessed May 1, 2025. https://tinyurl.com/2jyj9932