Abstract

Abstract Background Atypical teratoid/rhabdoid tumor (ATRT) is a rare malignant brain tumor that affects children ≤3 years and is associated with significant mortality. ATRT was established as a unique entity in 2001 and is diagnosed by specific molecular and methylation features. In this analysis, we present the most up-to-date descriptive epidemiology of ATRT in children and adolescents. Methods The Central Brain Tumor Registry of the United States, a combined dataset of the CDC’s National Program of Cancer Registries (NPCR) and NCI’s Surveillance, Epidemiology, and End Results Program, was used to estimate average annual age-adjusted incidence rates (AAAIR) and 95% confidence intervals (95%CI) per 100,000 population for ATRT diagnosed between 2005-2021 in those 0-19 years at diagnosis. AAAIR were calculated overall and by age group at diagnosis, sex, race/ethnicity, and tumor site (supratentorial, infratentorial, spine and cauda equina, and other brain/CNS). Relative survival rates (RS) from 2005-2020 using the CDC’s NPCR survival dataset by age group, sex, race/ethnicity, tumor site, and treatment (surgery and radiation, surgery only, or other/unknown). Results There were 1,240 new cases of ATRT diagnosed from 2005-2021 (AAAIR=0.09/100,000, 95%CI=0.08-0.09). Incidence did not significantly vary by sex and was lowest among non-Hispanic Black individuals. AAAIR was highest in children 1 year, with decreasing incidence with increasing age. Among those 1 year, infratentorial tumors had the highest incidence (AAAIR=0.26/100,000, 95%CI=0.22-0.30). Overall, 1- and 10-year RS were 58.7% and 33.5%, respectively. RS was highest among those who were 4-19 years, non-Hispanic White, with supratentorial tumor location, and who received surgery and radiation. Conclusion ATRT is associated with some of the poorest outcomes among rare pediatric CNS tumors. Survival varies significantly by race/ethnicity, treatment pattern, and tumor location. Understanding populations most affected and differences related to ATRT subgroups is necessary for furthering etiologic and clinical research.