Phase II treatment of medulloblastoma and pineoblastoma with melphalan: Clinical therapy based on experimental models of human medulloblastoma

Academic Article


  • We conducted a phase II study of intravenous (IV) melphalan in the treatment of children with recurrent medulloblastoma and in the initial treatment of children with poor-prognosis medulloblastoma and pineoblastoma. There was one complete response (CR) and two partial responses (PRs) among the 12 children with recurrent medulloblastoma. There were three PRs in the four patients initially treated with melphalan for poor-prognosis medulloblastoma or pine oblastoma. Toxicity was limited to severe myelosuppression with marked neutropenia and thrombocytopenia. These results support our laboratory studies demonstrating melphalan activity in human medulloblastoma, suggest that similar activity may be demonstrated against pineoblastoma, and support further trials with this agent (administered prior to radiotherapy) in the treatment of patients with newly diagnosed poor-prognosis medulloblastoma.
  • Authors

    Digital Object Identifier (doi)

    Author List

  • Friedman HS; Schold SC; Mahaley MS; Colvin OM; Oakes WJ; Vick NA; Burger PC; Bigner SH; Borowitz M; Halperin EC
  • Start Page

  • 904
  • End Page

  • 911
  • Volume

  • 7
  • Issue

  • 7