Atypical Teratoid/Rhabdoid Tumor in Taiwan: A Nationwide, Population-Based Study

SIMPLE SUMMARY: Atypical teratoid/rhabdoid tumor (AT/RT) is a rare, highly malignant CNS neoplasm with poor prognosis. A retrospective population-based analysis of patients with the diagnosis of AT/RT, registered between 1999 and 2014 in Taiwan, showed that: (1) AT/RT had a higher prevalence in males, in children < 36 months of age, and at infratentorial sites; (2) older age (≥12 months), presence of the tumor in the supratentorial region, use of radiotherapy, chemotherapy, or both were associated with better prognosis compared to surgery or no treatment. These data represent a historical experience with AT/RT in Taiwan and may inform risk stratification and clinical trial design. ABSTRACT: Background: Atypical teratoid/rhabdoid tumor (AT/RT) is a rare, highly aggressive embryonal brain tumor most commonly presenting in young children. Methods: We performed a nationwide, population-based study of AT/RT (ICD-O-3 code: 9508/3) in Taiwan using the Taiwan Cancer Registry Database and the National Death Certificate Database. Results: A total of 47 cases (male/female = 29:18; median age at diagnosis, 23.3 months (IQR: 12.5–87.9)) were diagnosed with AT/RT between 1999 and 2014. AT/RT had higher prevalence in males (61.70%), in children < 36 months (55.32%), and at infratentorial or spinal locations (46.81%). Survival analyses demonstrated that patients ≥ 3 years of age (n = 21 (45%)) had a 5y-OS of 41% (p < 0.0001), treatment with radiotherapy only (n = 5 (11%)) led to a 5y-OS of 60%, treatment with chemotherapy with or without radiotherapy (n = 27 (62%)) was associated with a 5y-OS of 45% (p < 0.0001), and patients with a supratentorial tumor (n = 11 (23%)) had a 5y-OS of 51.95%. Predictors of better survival on univariate Cox proportional hazard modeling and confirmed with multivariate analysis included older age (≥1 year), supratentorial sites, and the administration of radiotherapy, chemotherapy, or both. Gender had no effect on survival. Conclusion: Older age, supratentorial site, and treatment with radiotherapy, chemotherapy, or both significantly improves the survival of patients with AT/RT.