title: Treatment of patients with pineoblastoma with high dose cyclophosphamide.
abstract: The outcome for patients with pineoblastoma has historically been very poor, with most patients dying of disseminated disease despite irradiation. Furthermore, the low incidence of this tumor has hindered progress toward defining better treatment strategies. Here we report the activity and toxicity of cyclophosphamide administered as a single agent at a dose schedule of 2 g/m2/day for 2 successive days at monthly intervals for a maximum of four courses. Eight patients were evaluated, six newly diagnosed and two recurrent. Amongst the six newly diagnosed patients, there were three patients demonstrating partial responses, and three had stable disease throughout the cyclophosphamide treatment period. All six patients are alive and disease free after further therapy. One patient with recurrent disease demonstrated tumor progression on cyclophosphamide, and the other had stable disease throughout the cyclophosphamide treatment period. Both patients subsequently died of progressive disease. The major toxicity of high dose cyclophosphamide was hematopoietic, with one patient requiring a dose reduction after three courses due to prolonged thrombocytopenia. One patient was also withdrawn from treatment with cyclophosphamide due to impaired pulmonary function. This study demonstrates the activity of high dose cyclophosphamide in the treatment of pineoblastoma and may serve as basis for the design of future studies of this tumor.
authors.affiliation: Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.
authors.forename: D M
authors.initials: DM
authors.lastname: Ashley
authors.forename: D
authors.initials: D
authors.lastname: Longee
authors.forename: R
authors.initials: R
authors.lastname: Tien
authors.forename: H
authors.initials: H
authors.lastname: Fuchs
authors.forename: M L
authors.initials: ML
authors.lastname: Graham
authors.forename: J
authors.initials: J
authors.lastname: Kurtzberg
authors.forename: J
authors.initials: J
authors.lastname: Casey
authors.forename: J
authors.initials: J
authors.lastname: Olson
authors.forename: L
authors.initials: L
authors.lastname: Meier
authors.forename: L
authors.initials: L
authors.lastname: Ferrell
authors.forename: T
authors.initials: T
authors.lastname: Kerby
authors.forename: M
authors.initials: M
authors.lastname: Duncan-Brown
authors.forename: E
authors.initials: E
authors.lastname: Stewart
authors.forename: O M
authors.initials: OM
authors.lastname: Colvin
authors.forename: J M
authors.initials: JM
authors.lastname: Pipas
authors.forename: G
authors.initials: G
authors.lastname: McCowage
authors.forename: R
authors.initials: R
authors.lastname: McLendon
authors.forename: D D
authors.initials: DD
authors.lastname: Bigner
authors.forename: H S
authors.initials: HS
authors.lastname: Friedman
mesh_terms: D000293:Adolescent; D000328:Adult; D018906:Antineoplastic Agents, Alkylating / Q000627:therapeutic use*; D001932:Brain Neoplasms / Q000188:drug therapy*; D002675:Child, Preschool; D003131:Combined Modality Therapy; D016371:Cranial Irradiation; D003520:Cyclophosphamide / Q000627:therapeutic use*; D018450:Disease Progression; D018572:Disease-Free Survival; D004334:Drug Administration Schedule; D005260:Female; D006410:Hematopoiesis / Q000187:drug effects; D006801:Humans; D008168:Lung / Q000187:drug effects / Q000503:physiopathology; D008297:Male; D009364:Neoplasm Recurrence, Local / Q000188:drug therapy; D010870:Pineal Gland / Q000473:pathology*; D010871:Pinealoma / Q000188:drug therapy*; D018714:Radiotherapy, Adjuvant; D012074:Remission Induction; D012107:Research Design; D013116:Spinal Cord / Q000528:radiation effects; D015996:Survival Rate; D013921:Thrombocytopenia / Q000139:chemically induced; D016896:Treatment Outcome
chemical_list: D018906:Antineoplastic Agents, Alkylating; D003520:Cyclophosphamide