Differentiation of Outcomes by Treatment Regimen and Histology in Central Nervous System Primary Embryonal Tumors

Abstract

Central nervous system (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 histologic and genetic profiles. We present the largest study to date to identify differential survival outcomes within each subgroup by treatment regimen. The SEER (Surveillance Epidemiology and End Results) database was queried from 1973 to 2015 for embryonal tumors of primary CNS origin (n= 3900). 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. No significant patient demographic factors were found to be associated with increased mortality. In all 3 CNS embryonal tumor subtypes, most monotherapy and combinatorial treatment paradigms showed a higher hazard ratio compared with gross total resection with adjuvant chemoradiotherapy (hazard ratio, 1.72-22.94; P < 0.05 for all). In a subgroup analysis of patients with medulloblastoma ≤3 years of age, patients who did not receive radiation showed lower survival probabilities at 1, 5, and 10 years (odds ratio [OR], 0.37, P < 0.0001; OR, 0.39, P < 0.0001; OR, 0.34, P < 0.0001, respectively). Kaplan-Meier analysis of medulloblastoma histologic subtypes showed that use of radiation imparted a higher survival probability in the desmoplastic/nodular medulloblastoma and medulloblastoma not otherwise specified groups (P < 0.001 for both). 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 patients with medulloblastoma ≤3 years of age.