title: 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.
authors: Department of Pediatrics, University of California San Francisco School of Medicine, 505 Parnassus Avenue, M649, Box 0106, San Francisco, CA 94143, USA. Theodore.Nicolaides@ucsf.edu
authors: Theodore
authors: T
authors: Nicolaides
authors: Tarik
authors: T
authors: Tihan
authors: Biljana
authors: B
authors: Horn
authors: Jaclyn
authors: J
authors: Biegel
authors: Michael
authors: M
authors: Prados
authors: Anuradha
authors: A
authors: Banerjee
mesh_terms: D016543:Central Nervous System Neoplasms / Q000188:drug therapy* / Q000601:surgery*; D017024:Chemotherapy, Adjuvant / Q000379:methods*; D002675:Child, Preschool; D003131:Combined Modality Therapy / Q000379:methods; D018572:Disease-Free Survival; D004305:Dose-Response Relationship, Drug; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007223:Infant; D008297:Male; D012189:Retrospective Studies; D018335:Rhabdoid Tumor / Q000188:drug therapy* / Q000601:surgery*; D033581:Stem Cell Transplantation / Q000379:methods*; D017741:Survivors; D014182:Transplantation, Autologous / Q000379:methods