title: Carboplatin-based primary chemotherapy for infants and young children with CNS tumors.
abstract: 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.
authors.affiliation: Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229, USA. fouladi@cchmc.org
authors.forename: Maryam
authors.initials: M
authors.lastname: Fouladi
authors.forename: Sri
authors.initials: S
authors.lastname: Gururangan
authors.forename: Albert
authors.initials: A
authors.lastname: Moghrabi
authors.forename: Peter
authors.initials: P
authors.lastname: Phillips
authors.forename: Lindsey
authors.initials: L
authors.lastname: Gronewold
authors.forename: Dana
authors.initials: D
authors.lastname: Wallace
authors.forename: Robert A
authors.initials: RA
authors.lastname: Sanford
authors.forename: Amar
authors.initials: A
authors.lastname: Gajjar
authors.forename: Larry E
authors.initials: LE
authors.lastname: Kun
authors.forename: Richard
authors.initials: R
authors.lastname: Heideman
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects / Q000627:therapeutic use*; D016190:Carboplatin / Q000008:administration & dosage* / Q000009:adverse effects; D016543:Central Nervous System Neoplasms / Q000188:drug therapy*; D017024:Chemotherapy, Adjuvant; D002675:Child, Preschool; D003131:Combined Modality Therapy; D003520:Cyclophosphamide / Q000008:administration & dosage; D005047:Etoposide / Q000008:administration & dosage; D006801:Humans; D007223:Infant; D016019:Survival Analysis; D013997:Time Factors; D016896:Treatment Outcome
chemical_list: D005047:Etoposide; D003520:Cyclophosphamide; D016190:Carboplatin