A phase II prospective study of sequential myeloablative chemotherapy with hematopoietic stem cell rescue for the treatment of selected high risk and recurrent central nervous system tumors

Amy Rosenfeld, Morris Kletzel, Reggie Duerst, David Jacobsohn, Paul Haut, Joanna Weinstein, Alfred Rademaker, Colleen Schaefer, Lauren Evans, Molly Fouts, Stewart Goldman

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16 Scopus citations


High risk/recurrent CNS tumors have a poor prognosis. We studied tandem high dose chemotherapy (HDC) with hematopoietic progenitor stem cell rescues (HPCR) as potentially curative therapy. Twenty-four patients (mean age 6.8 years) were enrolled, 19 underwent HDC/HPCR. Diagnoses were medulloblastoma (n = 9), germ cell tumor (n = 4), high grade astrocytoma (n = 2), supratentorial PNET (n = 1), pineoblastoma (n = 2), or papillary meningioma (n = 1). Cytoreduction regimen #1 consisted of carboplatin (500 mg/m2) 9 3 days, etoposide (250 mg/m2) 9 3 days, and thiotepa (300 mg/m2) 9 3 days. Patients without progression or excessive toxicity (n = 11), received regimen &has2 with melphalan (60 mg/m2) 9 3 days and cyclophosphamide (1,500 mg/m2) 9 4 days. Projected overall/event-free survival for the 19 patients was 51/37% and 34/28% at 1 and 5 years, respectively. Toxicity was significant with six treatment related deaths including four with veno-occlusive disease. This regimen of sequential HDC/HPCR in high risk/recurrent CNS tumor patients is not feasible due to toxicity.

Original languageEnglish (US)
Pages (from-to)247-255
Number of pages9
JournalJournal of Neuro-Oncology
Issue number2
StatePublished - Apr 1 2010



  • Chemotherapy
  • Hematopoietic stem cell rescue
  • Myeloablative
  • Pediatric high risk and brain tumors
  • Pediatric recurrent brain tumors

ASJC Scopus subject areas

  • Clinical Neurology
  • Cancer Research
  • Oncology
  • Neurology

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