Intensive induction chemotherapy followed by high dose chemotherapy with autologous hematopoietic progenitor cell rescue in young children newly diagnosed with central nervous system atypical teratoid rhabdoid tumors

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Abstract
Background Central nervous system (CNS) atypical teratoid rhabdoid tumors (AT/RT) are rare tumors of childhood with a dismal prognosis. Historically, surgery and standard dose chemotherapy have resulted in a median survival of 8.5 months from diagnosis. Methods Thirteen children newly diagnosed with CNS AT/RT were treated with either the “Head Start I” (HS I) or “Head Start II” (HS II) regimens. Therapy included resection followed by five cycles of cisplatin, vincristine, cyclophosphamide, and etoposide. High dose methotrexate was added to each of the five induction courses in HS II. Consolidation for both regimens included carboplatin, thiotepa, and etoposide with autologous hematopoietic progenitor cell rescue (AHPCR). Results Six children, median age of 36 months, were treated on HS I between 1992 and 1997. Seven children, median age of 28 months, were treated on HS II between 1997 and 2002. One patient received craniospinal irradiation following AHPCR but prior to recurrence. There are presently three event-free survivors 42+, 54+, and 67+ months following diagnosis; none received RT. All three survivors were enrolled on HS II. Eight patients died of disease (six on HS I); one patient died from infection; one patient died from secondary malignancy following treatment for recurrent AT/RT. Conclusion Three of seven children with CNS AT/RT treated on HS II have experienced long term remissions. Long term survival can be achieved in a subset of young children with CNS AT/RT following resection with the use of multi-drug chemotherapy including high dose methotrexate and myeloablative chemotherapy without radiation therapy (RT). Pediatr Blood Cancer 2008;51:235–240.