A groundbreaking study from UT Southwestern Medical Center has revealed that Black and Hispanic children with high-risk neuroblastoma experience significantly worse survival outcomes than their white counterparts, even when receiving standardized treatment through clinical trials. The research, published in JAMA Network Open, is the first comprehensive evaluation of survival disparities by race and ethnicity in a national cohort of children with high-risk neuroblastoma enrolled in clinical trials.
"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," said lead author Dr. Puja Umaretiya, Assistant Professor of Pediatrics in the Division of Hematology and Oncology at UT Southwestern Medical Center.
Study Reveals Persistent Survival Gaps
The research team analyzed data from two cohorts of children with high-risk neuroblastoma treated through the Children's Oncology Group (COG) between 2007 and 2016. The first cohort included 696 children on chemotherapy induction/consolidation studies, while the second comprised 935 children on post-consolidation trials.
Among patients in induction/consolidation trials, Hispanic children had a 5-year overall survival rate of 47.2% compared to 61.3% for white children. After adjusting for tumor characteristics including MYCN amplification, tumor histology, and disease stage, Hispanic children still had a 78% higher hazard of death (HR, 1.78; 95% CI, 1.25-2.53; P = .01).
In post-consolidation trials, the disparities were even more pronounced. Five-year overall survival rates were 67.7% for Black patients, 62.2% for Hispanic patients, and 75.7% for white patients. Multivariate analysis revealed significantly shorter overall survival for both Black (HR, 1.54; 95% CI, 1.13-2.11) and Hispanic populations (HR, 1.63; 95% CI, 1.09-2.43) compared to white patients.
Social Determinants Likely Play Critical Role
Importantly, the researchers found no significant racial or ethnic differences in care delivery metrics, including induction therapy duration, early trial withdrawal, or patterns of relapse and progression. This suggests that the disparities cannot be explained by differences in the medical care provided within the clinical trial setting.
"These data identify the need for studies focused in two areas," Dr. Umaretiya explained. "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. Collecting more specific data, such as social determinants of health, may provide insight into the underlying causes."
The study noted significant socioeconomic disparities among the patient populations. In the induction/consolidation cohort, 58.2% of Hispanic patients and 50.5% of Black patients lived in areas with high household poverty, compared to just 25.2% of white patients.
Previous research from Dr. Umaretiya's team found that nearly three-quarters of Black and Hispanic families of children with cancer face at least one unmet health-related social need — such as food, housing, or transportation insecurity — which may affect outcomes despite access to standardized clinical trial protocols.
Neuroblastoma: A Challenging Childhood Cancer
Neuroblastoma is the most common extracranial solid tumor in children, developing from immature nerve cells. High-risk neuroblastoma is particularly aggressive and requires intensive multimodal therapy, including chemotherapy, surgery, radiation, stem cell transplantation, and immunotherapy.
The standard treatment approach for high-risk neuroblastoma follows a sequence of induction chemotherapy, surgery, consolidation with high-dose chemotherapy and stem cell rescue, and post-consolidation therapy with immunotherapy and differentiation agents. Despite this intensive approach, survival rates remain challenging, with approximately 40-50% of high-risk patients experiencing long-term survival.
Implications for Future Research and Interventions
Dr. Umaretiya emphasized that alongside better data collection, "we need to embed supportive care interventions for groups at risk for worse outcomes despite standardized therapies."
Dr. Sandi L. Pruitt, Professor in the Peter O'Donnell Jr. School of Public Health and Associate Director of Community Outreach and Engagement in the Simmons Cancer Center, who contributed to the study, noted that addressing these disparities will require a multifaceted approach that extends beyond the clinical setting.
The findings highlight that equal access to cutting-edge clinical trials does not automatically translate to equal outcomes. Future research must focus on understanding and addressing the underlying social and economic factors that contribute to these persistent disparities in childhood cancer survival.
The study was funded by the American Society of Clinical Oncology Young Investigator Award, Children's Oncology Group grants, St. Baldrick's Foundation, and the National Cancer Institute Cancer Center Support Grant.
