Differentiation of Outcomes by Treatment Regimen and Histology in CNS Primary Embryonal Tumors

Abstract Background: CNS embryonal tumors are malignant neoplasms of undifferentiated embryonic cells that typically occur in the pediatric population. They are further divided into many subgroups by distinct histological and genetic profiles. We present the largest-to-date study to identify differential survival outcomes within each subgroup by treatment regimen. Methods: The Surveillance, Epidemiology, and End Result database was queried from 1973–2015 for embryonal tumors of primary CNS origin (n=3,900). The effects of patient demographics, tumor characteristics, and treatment regimen were analyzed using a multivariate Cox proportional hazard model in CNS embryonal tumor subtypes divided into medulloblastoma, atypical teratoid/rhabdoid tumor, and primitive neuroectodermal tumor. Results: No significant patient demographic factors were found to be associated with increased mortality. In all three CNS embryonal tumor subtypes, the majority of monotherapy and combinatorial treatment paradigms exhibited a higher hazard ratio compared to gross total resection with adjuvant chemoradiotherapy (HR 1.72-22.94, p Conclusions: CNS embryonal tumors are highly malignant in all populations and the best survival is seen with aggressive combination therapies. Radiation therapy may have a role in prolonging survival in medulloblastoma patients ≤3 years of age.