Outcomes and patterns of care in a nationwide cohort of pediatric medulloblastoma: Factors affecting proton therapy utilization

Abstract

Purpose: We examined national outcomes and patterns of care for pediatric patients with medulloblastoma (MB) in a large observational cohort. Methods and materials: Using the National Cancer Database, we evaluated the clinical features and survival outcomes of patients diagnosed with MB. The association between intervention, covariables, and outcome was assessed in a multivariable Cox analysis and through logistic regression analysis. Survival was estimated using the Kaplan-Meier method. Results: Among the 4032 patients in the National Cancer Database with pediatric brain tumors, 1300 patients met the inclusion criteria of histologic diagnosis, receipt of chemotherapy and radiation, and age ≤18 years. The median age and follow-up were 8.4 years and 4.5 years, respectively. Five-year survival was 79.0%. In the univariate analysis, inferior outcome (overall survival) was associated with rural residence (hazard ratio [HR], 2.78; 95% confidence interval [CI],1.47-5.29; P < .01) and histology (large cell; HR, 1.78; 95% CI,1.08-2.94; P < .05). In multivariable analysis, both remained significant predictors of survival (large cell: HR, 1.68; P < .05; rural residence: HR, 2.74; P < .01). In 2013, the utilization rate of proton therapy (23% of patients) in the United States surpassed intensity modulate radiation therapy (16%), more frequently for patients with higher income (P < .05) or more favorable insurance status (P < .05). Conclusions: As one of the largest data sets on pediatric MB, the observed variations in treatment intervention and survival outcomes may represent a target for further research.