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Central Nervous System Atypical Teratoid/Rhabdoid Tumor: Results of Therapy in Children Enrolled in a Registry

From the Department of Pediatric Hematology/Oncology, The Children’s Hospital, The Cleveland Clinic, Cleveland, OH; Department of Pathology/NeuroPathology, Johns Hopkins Hospital, Baltimore, MD; Department of Hematology/Oncology, Hassenfeld Children’s Center, New York University Medical Center, New York, NY; Division of Neurology Research, Department of Pathology, and Division of Genetics Children’s Hospital of Philadelphia, Philadelphia, PA; Division of Anatomic Pathology, Mayo Clinic, Rochester, MN; Department of Hematology/Oncology, Christiana Care Health Services/A.I. duPont Institute, Wilmington, DE

Address reprint requests to Joanne M. Hilden, MD, Department of Pediatric Hematology/Oncology, The Children’s Hospital, The Cleveland Clinic, 9500 Euclid Ave, Desk S20, Cleveland, OH 44195; e-mail: hildenj{at}ccf.org

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.

Supported by National Institutes of Health (CA46274) and the Children’s Oncology Group Brain Tumor Resource Laboratory (J.A.B).

Authors’ disclosures of potential conflicts of interest are found at the end of this article.

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