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

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;  < .01) and histology (large cell; HR, 1.78; 95% CI,1.08-2.94;  < .05). In multivariable analysis, both remained significant predictors of survival (large cell: HR, 1.68;  < .05; rural residence: HR, 2.74;  < .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 ( < .05) or more favorable insurance status ( < .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.