title: Phase II treatment of medulloblastoma and pineoblastoma with melphalan: clinical therapy based on experimental models of human medulloblastoma.
abstract: 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 pineoblastoma. 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.affiliation: Department of Pediatrics, Duke University Medical Center, Durham, NC.
authors.forename: H S
authors.initials: HS
authors.lastname: Friedman
authors.forename: S C
authors.initials: SC
authors.lastname: Schold
authors.forename: M S
authors.initials: MS
authors.lastname: Mahaley
authors.forename: O M
authors.initials: OM
authors.lastname: Colvin
authors.forename: W J
authors.initials: WJ
authors.lastname: Oakes
authors.forename: N A
authors.initials: NA
authors.lastname: Vick
authors.forename: P C
authors.initials: PC
authors.lastname: Burger
authors.forename: S H
authors.initials: SH
authors.lastname: Bigner
authors.forename: M
authors.initials: M
authors.lastname: Borowitz
authors.forename: E C
authors.initials: EC
authors.lastname: Halperin
mesh_terms: D000293:Adolescent; D000328:Adult; D001932:Brain Neoplasms / Q000188:drug therapy*; D002528:Cerebellar Neoplasms / Q000188:drug therapy*; D002675:Child, Preschool; D004341:Drug Evaluation; D005260:Female; D006801:Humans; D007223:Infant; D008527:Medulloblastoma / Q000188:drug therapy* / Q000556:secondary; D008558:Melphalan / Q000008:administration & dosage* / Q000009:adverse effects; D009364:Neoplasm Recurrence, Local / Q000188:drug therapy; D010871:Pinealoma / Q000188:drug therapy*; D011379:Prognosis
chemical_list: D008558:Melphalan