Clinical characteristics and prognostic factors in pediatric atypical teratoid/rhabdoid tumor: a single-center retrospective study

Abstract

Abstract

Atypical teratoid/rhabdoid tumor (AT/RT) is a highly aggressive embryonal central nervous system tumor that predominantly affects young children. Despite progress in multimodal therapy, optimal treatment strategies remain controversial, especially for patients with metastatic disease. We retrospectively analyzed clinical data from 22 pediatric patients with AT/RT treated at our center between November 2017 and April 2025. We evaluated demographic characteristics, extent of surgical resection, chemotherapy regimens, radiotherapy strategies, and survival outcomes. Overall survival (OS) and progression-free survival (PFS) were estimated by the Kaplan-Meier method. Prognostic factors were assessed using Cox proportional hazards regression models. The median age at diagnosis was 33 months (range, 7–110). Twelve patients (54.5%) presented with metastatic disease, and gross total resection(GTR) was achieved in 12 patients (54.5%). Eleven patients (50.0%) completed the initially planned chemotherapy regimen. At a reverse Kaplan-Meier median follow-up of 38 months (95% CI, 19–46 months), the 2-year OS and PFS rates were 64.3% (95% CI, 42.7%–85.9%) and 27.3% (95% CI, 9.7%–44.9%), respectively. In univariate analysis, radiotherapy and gross total resection were significantly associated with improved progression-free survival (PFS). In exploratory multivariable Cox regression analysis, metastatic disease and elevated lactate dehydrogenase(LDH) levels remained associated with inferior OS, whereas no variables retained independent significance for PFS. Metastatic presentation and elevated LDH levels were associated with poorer overall survival in this exploratory single-center analysis. These findings require validation in larger multicenter cohorts.