Purpose: Therapy for central nervous system (CNS) atypical teratoid rhabdoid tumor (ATRT) is controversial. We describe 4 children treated with sarcoma-like therapy and review the literature to evaluate outcome in relation to treatment modalities.
Procedure: Reports from 1995 to 2007, describing clinical features of children (< or =18 years) were reviewed for details of demography, therapy, and outcome. Kaplan-Meier survival analyses were used to study the impact of clinical features, demography, and therapy on overall survival (OS).
Results: The median OS for patients treated with multiagent chemotherapy (n=79) was 17.3 months (range, 1.5-93 mo); unrelated to age at diagnosis, sex, tumor site, and extent of resection. Overall mortality was 67%. Disease progression (craniospinal spread in 58%) was the major cause of death. Patients (n=30) treated with intrathecal (IT) chemotherapy had significantly higher 2-year OS [64% (95% confidence interval, 46.5-82.0) vs. 17.3% (95% confidence interval, 5.4-29.3); P<0.0001] and lower prevalence of distant CNS metastasis compared with those without IT therapy (n=49) (20% vs. 59.2%; P=0.001).
Conclusions: Despite dismal OS, multimodal therapy can induce remission even in metastatic CNS ATRT with partial resection. IT chemotherapy results in higher OS and, because of an overall high rate of distant relapse, should be considered in future trials.