Carboplatin-based primary chemotherapy for infants and young children with CNS tumors

Maryam Fouladi, Sri Gururangan, Albert Moghrabi, Peter Phillips, Lindsey Gronewold, Dana Wallace, Robert A. Sanford, Amar Gajjar, Larry E. Kun, Richard Heideman

Research output: Contribution to journalArticlepeer-review

23 Scopus citations


BACKGROUND: A carboplatin-based chemotherapy regimen was used as primary postoperative therapy in infants with central nervous system (CNS) tumors to limit renal and ototoxicity and to target systemic exposure. METHODS: Fifty-three patients aged <age 3 years with embryonal CNS tumor medulloblastoma (n = 20), ependymoma (EP, n = 21), choroid plexus carcinoma (CPCA, n = 5), and primitive embryonal neoplasms including atypical teratoid rhabdoid tumors (n = 7) were treated with cyclophosphamide, etoposide, and carboplatin. Radiation therapy was used only for residual disease at the end of chemotherapy or disease progression. RESULTS: The response rate after 2 cycles of chemotherapy was 34% (complete response, 13.8%; partial response, 20.7%). Myelosuppression was the dominant toxicity; 2 patients had toxic deaths related to thrombocytopenia with trauma. The 5-year overall survival (OS) was 49% ± 7%, and the progression-free survival (PFS) was 31% ± 7%, with a median follow-up of 11.4 years (range, 5.2-15.0 years). For medulloblastoma, the 5-year PFS was 26% ± 9%; for EP it was 33% ± 10%; for CPCA it was 80% ± 18%; and for primitive neuroectodermal and atypical teratoid rhabdoid tumors it was 0%. Localized EP patients with gross total resection who did not undergo radiotherapy had a 5-year PFS of 57% ± 17% and OS of 71% ± 16%. Two patients developed late second malignancies; 1 was associated with germline p53 mutation. CONCLUSIONS: The results confirm that carboplatin has similar activity to cisplatin in otherwise similar regimens. Five-year survival data are comparable to those reported in other recent studies, including high-dose chemotherapy studies. Of note is the marked activity in CPCA and gross totally resected EP.

Original languageEnglish (US)
Pages (from-to)3243-3253
Number of pages11
Issue number14
StatePublished - 2009


  • Brain tumor
  • Carboplatin
  • Chemotherapy
  • Infants

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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