Outcome for young children newly diagnosed with ependymoma, treated with intensive induction chemotherapy followed by myeloablative chemotherapy and autologous stem cell rescue.
BACKGROUND
The purpose of this study is to investigate the efficacy of an intensive chemotherapy induction regimen followed by myeloablative chemotherapy and autologous hematopoietic stem cell rescue (AHSCR) in children with newly diagnosed ependymoma.


METHODS
Twenty-nine children less than 10 years of age at diagnosis of ependymoma were enrolled on the "Head Start" studies. Twenty-four patients with localized disease received an induction regimen including five cycles of chemotherapy (cisplatin, vincristine, etoposide cyclophosphamide, and high dose methotrexate for patients with metastatic disease). Following induction, individuals without evidence of disease proceeded to marrow-ablative chemotherapy (thiotepa, carboplatin, and etoposide) with AHSCR.


RESULTS
The estimated 5-year event free survival (EFS) and overall survival (OS) from diagnosis were 12% (+/-6%) and 38% (+/-10%), respectively. The toxic mortality amongst this group of 29 patients was 10.3%. Younger age (less than 18 months at diagnosis) was the only statistically significant prognostic factor. The estimated 5-year OS rate for the five patients with metastatic disease at presentation was 80% (+/-18%). Overall, radiation-free survival at 5 years from diagnosis was 8% (+/-5%).


CONCLUSIONS
The use of an intensive induction chemotherapy regimen including myeloablative chemotherapy followed by AHSCR in newly diagnosed young children with ependymoma is not superior to other previously reported chemotherapeutic strategies.
Zacharoulis
Stergios
S
Department of Pediatric Hematology/Oncology, Children's Hospital Los Angdes, Neural Tumors Program, Los Angles, CA 900 27, USA. szacharoulis@chla.usc.edu
Levy
Adam
A
Chi
Susan N
SN
Gardner
Sharon
S
Rosenblum
Marc
M
Miller
Douglas C
DC
Dunkel
Ira
I
Diez
Blanca
B
Sposto
Richard
R
Ji
Lingyun
L
Asgharzadeh
Shahab
S
Hukin
Juliette
J
Belasco
Jean
J
Dubowy
Ronald
R
Kellie
Stewart
S
Termuhlen
Amanda
A
Finlay
Jonathan
J
D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D001932:Brain Neoplasms / Q000175:diagnosis / Q000628:therapy*; D016190:Carboplatin / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D002945:Cisplatin / Q000008:administration & dosage; D003131:Combined Modality Therapy; D003520:Cyclophosphamide / Q000008:administration & dosage; D018572:Disease-Free Survival; D004806:Ependymoma / Q000175:diagnosis / Q000628:therapy*; D005047:Etoposide / Q000008:administration & dosage; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D008297:Male; D008727:Methotrexate / Q000008:administration & dosage; D009364:Neoplasm Recurrence, Local / Q000628:therapy; D013852:Thiotepa / Q000008:administration & dosage; D014182:Transplantation, Autologous; D016896:Treatment Outcome; D014750:Vincristine / Q000008:administration & dosage
D014750:Vincristine; D005047:Etoposide; D003520:Cyclophosphamide; D013852:Thiotepa; D016190:Carboplatin; D002945:Cisplatin; D008727:Methotrexate