title: A phase II prospective study of sequential myeloablative chemotherapy with hematopoietic stem cell rescue for the treatment of selected high risk and recurrent central nervous system tumors.
abstract: High risk/recurrent CNS tumors have a poor prognosis. We studied tandem high dose chemotherapy (HDC) with hematopoietic progenitor stem cell rescues (HPCR) as potentially curative therapy. Twenty-four patients (mean age 6.8 years) were enrolled, 19 underwent HDC/HPCR. Diagnoses were medulloblastoma (n = 9), germ cell tumor (n = 4), high grade astrocytoma (n = 2), supratentorial PNET (n = 1), pineoblastoma (n = 2), or papillary meningioma (n = 1). Cytoreduction regimen #1 consisted of carboplatin (500 mg/m(2)) x 3 days, etoposide (250 mg/m(2)) x 3 days, and thiotepa (300 mg/m(2)) x 3 days. Patients without progression or excessive toxicity (n = 11), received regimen #2 with melphalan (60 mg/m(2)) x 3 days and cyclophosphamide (1,500 mg/m(2)) x 4 days. Projected overall/event-free survival for the 19 patients was 51/37% and 34/28% at 1 and 5 years, respectively. Toxicity was significant with six treatment related deaths including four with veno-occlusive disease. This regimen of sequential HDC/HPCR in high risk/recurrent CNS tumor patients is not feasible due to toxicity.
authors.affiliation: Center for Cancer and Blood Disorders, Phoenix Children's Hospital, 1919 East Thomas Road, Phoenix, AZ, 85016, USA. arosenfeld@phoenixchildrens.com
authors.forename: Amy
authors.initials: A
authors.lastname: Rosenfeld
authors.forename: Morris
authors.initials: M
authors.lastname: Kletzel
authors.forename: Reggie
authors.initials: R
authors.lastname: Duerst
authors.forename: David
authors.initials: D
authors.lastname: Jacobsohn
authors.forename: Paul
authors.initials: P
authors.lastname: Haut
authors.forename: Joanna
authors.initials: J
authors.lastname: Weinstein
authors.forename: Alfred
authors.initials: A
authors.lastname: Rademaker
authors.forename: Colleen
authors.initials: C
authors.lastname: Schaefer
authors.forename: Lauren
authors.initials: L
authors.lastname: Evans
authors.forename: Molly
authors.initials: M
authors.lastname: Fouts
authors.forename: Stewart
authors.initials: S
authors.lastname: Goldman
mesh_terms: D000293:Adolescent; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D016190:Carboplatin / Q000008:administration & dosage / Q000009:adverse effects; D016543:Central Nervous System Neoplasms / Q000401:mortality / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D003520:Cyclophosphamide / Q000008:administration & dosage; D005047:Etoposide / Q000008:administration & dosage / Q000009:adverse effects; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D007223:Infant; D053208:Kaplan-Meier Estimate; D008297:Male; D008558:Melphalan / Q000008:administration & dosage / Q000009:adverse effects; D013852:Thiotepa / Q000008:administration & dosage / Q000009:adverse effects; D019172:Transplantation Conditioning
chemical_list: D005047:Etoposide; D003520:Cyclophosphamide; D013852:Thiotepa; D016190:Carboplatin; D008558:Melphalan