TY - JOUR
T1 - Central nervous system atypical teratoid/rhabdoid tumor
T2 - Results of therapy in children enrolled in a registry
AU - Hilden, Joanne M.
AU - Meerbaum, Sharon
AU - Burger, Peter
AU - Finlay, Jonathan
AU - Janss, Anna
AU - Scheithauer, Bernd W.
AU - Walter, Andrew W.
AU - Rorke, Lucy B.
AU - Biegel, Jaclyn A.
PY - 2004
Y1 - 2004
N2 - Purpose: Atypical teratoid/rhabdoid tumor (AT/RT) of the CNS is an extremely rare and aggressive tumor of early childhood. The poor outcome with conventional infant brain tumor therapy has resulted in a lack of clear treatment guidelines. A registry has been established to create an outcomes database and to facilitate biology studies for this tumor. Materials and Methods: A standardized data sheet was provided to treating physicians listing the reports that were to be sent to the registry for abstraction. Follow-up information was sought twice yearly. Results: Information was complete for 42 patients. Median age at diagnosis was 24 months. Nine patients (21%) had disseminated disease at diagnosis. Sixteen tumors were infratentorial; 26 were supratentorial. Twenty patients (48%) received a primary complete resection. Primary therapy included chemotherapy in all patients, radiotherapy in 13 patients (31%), stem-cell rescue in 13 patients (31%), and intrathecal chemotherapy in 16 patients (38%). Recurrent or progressive disease was reported in nine and 19 patients, respectively. Twenty-seven patients (64%) are dead of disease (3 to 62 months from diagnosis) and one patient died of toxicity. Fourteen patients (33%) show no evidence of disease (9.5 to 96 months from diagnosis). The median survival is 16.75 months and the median event-free survival is 10 months. Conclusion: Aggressive therapy has prolonged the natural history in a subset of children. Prospective multi-institutional and national clinical trials designed specifically for AT/RT are needed. Enrollment onto the AT/RT registry should be continued.
AB - Purpose: Atypical teratoid/rhabdoid tumor (AT/RT) of the CNS is an extremely rare and aggressive tumor of early childhood. The poor outcome with conventional infant brain tumor therapy has resulted in a lack of clear treatment guidelines. A registry has been established to create an outcomes database and to facilitate biology studies for this tumor. Materials and Methods: A standardized data sheet was provided to treating physicians listing the reports that were to be sent to the registry for abstraction. Follow-up information was sought twice yearly. Results: Information was complete for 42 patients. Median age at diagnosis was 24 months. Nine patients (21%) had disseminated disease at diagnosis. Sixteen tumors were infratentorial; 26 were supratentorial. Twenty patients (48%) received a primary complete resection. Primary therapy included chemotherapy in all patients, radiotherapy in 13 patients (31%), stem-cell rescue in 13 patients (31%), and intrathecal chemotherapy in 16 patients (38%). Recurrent or progressive disease was reported in nine and 19 patients, respectively. Twenty-seven patients (64%) are dead of disease (3 to 62 months from diagnosis) and one patient died of toxicity. Fourteen patients (33%) show no evidence of disease (9.5 to 96 months from diagnosis). The median survival is 16.75 months and the median event-free survival is 10 months. Conclusion: Aggressive therapy has prolonged the natural history in a subset of children. Prospective multi-institutional and national clinical trials designed specifically for AT/RT are needed. Enrollment onto the AT/RT registry should be continued.
UR - http://www.scopus.com/inward/record.url?scp=4344710791&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=4344710791&partnerID=8YFLogxK
U2 - 10.1200/JCO.2004.07.073
DO - 10.1200/JCO.2004.07.073
M3 - Article
C2 - 15254056
AN - SCOPUS:4344710791
SN - 0732-183X
VL - 22
SP - 2877
EP - 2884
JO - Journal of Clinical Oncology
JF - Journal of Clinical Oncology
IS - 14
ER -