High-dose chemotherapy and autologous stem cell rescue for atypical teratoid/rhabdoid tumor of the central nervous system

Abstract

Atypical Teratoid/Rhabdoid tumors (AT/RT) of the central nervous system are rare but aggressive tumors of childhood. Median survival with surgery and standard chemotherapy is less than 12 months. In an attempt to improve outcome, patients were treated with aggressive surgical resection and multi-agent chemotherapy, followed by high dose chemotherapy with autologous stem cell rescue. Nine consecutive children (median age 21 months) were diagnosed with AT/RT at the University of California San Francisco Childrens Hospital from 1997 to 2007 and treated with this aggressive approach. Diagnosis was confirmed using molecular markers. There are two long-term survivors (78 and 98 months from diagnosis). One additional patient is alive with disease. Three patients died of disease during therapy. Three patients died of disease after therapy was complete. There were no toxic deaths. Two of nine patients treated for AT/RT at our institution with high dose chemotherapy and autologous bone marrow transplant are long-term survivors, suggesting that a subset of patients can be cured with this approach.

Fig. 1

Immuno-histochemistry. a H and E of small cell component of AT/RT. b H and E of large cell component, with necrotic area (arrow). c Higher magnification of b. d Positive EMA stain. e Positive Cytokeratin stain. f BAF47/INI1 antibody stain showing loss of INI1 expression in AT/RT. Retained expression is noted in scattered inflammatory cells, constituting a positive internal control

Fig. 2

Survival curves. a Kaplan–Meier curve showing overall survival. b Kaplan–Meier curve showing survival based on extent of resection