Purpose: To describe clinical features and therapeutic approaches and to identify prognostic factors in children with ATRT of the CNS.
Method: Observational study including all patients aged less than 18 years, diagnosed with CNS ATRT in France between January 1998 and July 2008, identified from hospital files and French Pediatric Cancer registry. Pathology review included histological and immunohistochemical analysis, including INI-1 staining. Impact of clinical characteristics (age, sex, site of primary tumor and metastatic status) on the overall survival (OS) was assessed using Cox models.
Results: seventy out of the 71 patients identified with ATRT over this 10-year period were included in the study (1 patient excluded due to incomplete clinical data). Median age was 2.8 years (range, 15 days-12.8 years). Primary tumor site was supratentorial (ST) in 34, posterior fossa (PF) in 30, mixed (STþPF) in 2 and medullar in 4 patients. The disease was disseminated at diagnosis in 22 patients. Five patients had non-CNS disease associated with CNS disease. Surgical resection was complete in 41 patients. Adjuvant therapy included chemotherapy in 55 cases and radiotherapy in 20 patients. Chemotherapy regimens were not standardized more than the study period: ATRT04, PNET High Risk and BB SFOP protocols were most frequently used. Median follow-up was 52 months (range, 13 months-10 years). Disease progression or relapse occurred in 51 children. Median time to progression/relapse was 4.4 months. Median survival time was 9.9 months. One-year progression-free survival and OS were 21% and 42%, respectively. Metastatic status at diagnosis was the only prognostic factor (Hazard ratio for death: 2.1, 95%CI: 1.2-3.8, p¼0.01).
Conclusion: Children with ATRT of the CNS have a dismal prognosis. Innovative therapeutic are urgently needed.