CNS atypical teratoid/rhabdoid tumor registry: An update

Abstract

12507 Background: Atypical teratoid/rhabdoid tumor (AT/RT) is a highly malignant tumor of the central nervous system (CNS) in childhood, comprising up to 20% of malignant CNS tumors in patients < 3 years old. Although aggressive approaches including surgery, chemotherapy, intrathecal chemotherapy, high dose chemotherapy with hematopoietic stem cell transplant (HD-HSCT) and/or radiation have resulted in prolonged survival, overall survival remains poor. Methods: We reviewed pediatric patients with AT/RT on an IRB- approved registry formed in 1995 with new insight based on updated outcomes and additional patient enrollment. Upon patient entry, the managing physician submitted pertinent medical records for data abstraction. Physicians were contacted in accordance with privacy regulations for updated records. The Kaplan-Meier method was used to estimate survival time (from diagnosis to death or most recent status update) and groups were compared using the log-rank test at a significance level of p<0.05 with SAS® software version 9.1. Results: Analysis was conducted on 73 patients with a 4:3 male to female ratio. Age at diagnosis ranged from 5 days to 234 months, median = 19 months. Twenty-five patients have no evidence of disease, median 59 months from diagnosis (range 10–140). Forty-three patients (59%) developed recurrent or progressive disease; 6 of these patients are alive at a median of 28 months from recurrence (range 4–38). Forty-four patients (60%) are dead of disease; median time to death was 7 months from diagnosis (range 0–62). Factors correlating with survival are: primary tumor site not in spinal cord (p=0.029), lack of metastatic disease (p=0.024), gross total resection (p<0.001), and upfront radiation particularly in children < 3 years old (p<0.001). HD-HSCT appears to result in improved survival in children < 3 years of age who do not receive radiation, though the small sample size does not yield a statistically significant survival advantage. Conclusions: In treatment of AT/RT, extent of surgical resection and upfront radiation are beneficial for overall survival, particularly in young children. Early information regarding upfront HD-HSCT in lieu of radiation for children < 3 years of age with AT/RT suggests it may be effective. Prospective studies are warranted to further investigate this treatment. No significant financial relationships to disclose.