Outcome for young children newly diagnosed with ependymoma, treated with intensive induction chemotherapy followed by myeloablative chemotherapy and autologous stem cell rescue

Stergios Zacharoulis, Adam Levy, Susan N. Chi, Sharon Gardner, Marc Rosenblum, Douglas C. Miller, Ira Dunkel, Blanca Diez, Richard Sposto, Lingyun Ji, Shahab Asgharzadeh, Juliette Hukin, Jean Belasco, Ronald Dubowy, Stewart Kellie, Amanda Termuhlen, Jonathan Finlay

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75 Scopus citations


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. Patients and 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.

Original languageEnglish (US)
Pages (from-to)34-40
Number of pages7
JournalPediatric Blood and Cancer
Issue number1
Publication statusPublished - Jul 1 2007



  • CNS tumors
  • Chemotherapy
  • Ependymoma
  • Transplantation

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Oncology

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