Atypical teratoid or rhabdoid tumors: improved outcome with high-dose chemotherapy.
OBJECTIVE
To retrospectively review an institutional experience in managing atypical teratoid/rhabdoid tumors (AT/RT) of the Central Nervous System with high-dose chemotherapy in infants and children less than 4 years old.


METHODS
Eight AT/RT patients were identified during the study period 2003 to 2008. Tumor location was supratentorial in 3 cases, infratentorial in 3 cases, and multifocal in 2 patients. Five patients presented with leptomeningeal dissemination. Two of these patients did not receive any active therapy. After surgery, the 6 remaining patients received induction therapy followed by sequential high-dose chemotherapy with autologous stem cell rescue. Two patients receive focal irradiation.


RESULTS
At a median follow-up of 52 months, 4 patients are alive without evidence of tumor. Three of these patients, including 2 with metastatic disease were not irradiated. However, all surviving patients exhibit neuro-cognitive impairment, either at baseline assessment or upon follow-up.


CONCLUSIONS
This experience confirms that a subset of young AT/RT patients may achieve long-term survival with intensive and high-dose chemotherapy. The role of radiotherapy is still unclear. However, evidence of neuro-cognitive impairment even in the absence of radiotherapy in this young population suggests that systematic introduction of radiotherapy in current protocols should be carefully assessed.
Finkelstein-Shechter
Tal
T
Bone Marrow Transplant Program, Hospital for Sick Children, Toronto, Canada.
Gassas
Adam
A
Mabbott
Donald
D
Huang
Annie
A
Bartels
Ute
U
Tabori
Uri
U
Janzen
Laura
L
Laura
Janzen
J
Hawkins
Cynthia
C
Taylor
Michael
M
Bouffet
Eric
E
D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D001932:Brain Neoplasms / Q000188:drug therapy* / Q000473:pathology / Q000532:radiotherapy; D002675:Child, Preschool; D002945:Cisplatin / Q000008:administration & dosage; D003131:Combined Modality Therapy; D016371:Cranial Irradiation; D003520:Cyclophosphamide / Q000008:administration & dosage; D005047:Etoposide / Q000008:administration & dosage; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D009362:Neoplasm Metastasis; D011879:Radiotherapy Dosage; D012189:Retrospective Studies; D018335:Rhabdoid Tumor / Q000188:drug therapy* / Q000473:pathology / Q000532:radiotherapy; D015996:Survival Rate; D013724:Teratoma / Q000188:drug therapy* / Q000473:pathology / Q000532:radiotherapy; D016896:Treatment Outcome; D014750:Vincristine / Q000008:administration & dosage
D014750:Vincristine; D005047:Etoposide; D003520:Cyclophosphamide; D002945:Cisplatin