title: Intensive chemotherapy for children and young adults with metastatic primitive neuroectodermal tumors of the soft tissue.
abstract: The MMT4 study was designed to explore an intensive chemotherapy regimen (MMT4-89) and the role of high-dose melphalan (MMT4-91) in children with metastatic soft tissue sarcoma, including extraosseous peripheral neuroectodermal tumor (PNET). Thirty-one patients with PNET were treated between 1989 and 1995 (11 according to MMT4-89 and 20 according to MMT4-91). Chemotherapy consisted of four CEVAIE cycles, each including three 3-week courses: CEV (carboplatin 500 mg/m(2), epirubicin 150 mg/m(2), vincristine 1.5 mg/m(2)), IVA ifosfamide 9 g/m(2), actinomycin 1.5 mg/m(2), vincristine 1.5 mg/m(2)), IVE (ifosfamide 9 g/m(2), etoposide 600 mg/m(2), vincristine 1.5 mg/m(2)). In MMT4-91 the fourth CEVAIE was replaced with melphalan 200 mg/m(2) with stem cell rescue. The CEV combination was evaluated as a window study. Surgery followed the second cycle. Radiotherapy was administered to post-surgical residual disease. The response rate was 55% after CEV, rising to 80% after the first CEVAIE. Twenty-five patients achieved complete remission (CR). Overall, the 5-year EFS was 22.6%: 36.4% and 15% for patients treated according to MMT4-89 and MMT4-91, respectively (P = 0.3). Local control was achieved in 77% of irradiated patients vs 45% of non-irradiated. Age >10 years was associated with significantly poorer outcome (P = 0.04). In conclusion, despite the high CR rate, intensive chemotherapy with or without high-dose melphalan appeared to have little impact on the survival of patients with metastatic extraosseus PNET.
authors.affiliation: Hematology/Oncology Division, Department of Pediatrics, Padova, Italy.
authors.forename: G
authors.initials: G
authors.lastname: Bisogno
authors.forename: M
authors.initials: M
authors.lastname: Carli
authors.forename: M
authors.initials: M
authors.lastname: Stevens
authors.forename: O
authors.initials: O
authors.lastname: Oberlin
authors.forename: J
authors.initials: J
authors.lastname: Treuner
authors.forename: G
authors.initials: G
authors.lastname: Scarzello
authors.forename: R
authors.initials: R
authors.lastname: Colombatti
authors.forename: L
authors.initials: L
authors.lastname: De Zen
authors.forename: C R
authors.initials: CR
authors.lastname: Pinkerton
mesh_terms: D000293:Adolescent; D000367:Age Factors; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000008:administration & dosage*; D016026:Bone Marrow Transplantation; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D005260:Female; D006801:Humans; D008297:Male; D008558:Melphalan / Q000008:administration & dosage*; D017599:Neuroectodermal Tumors / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D036102:Peripheral Blood Stem Cell Transplantation; D012074:Remission Induction / Q000379:methods; D012509:Sarcoma / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D016019:Survival Analysis; D016896:Treatment Outcome
chemical_list: D008558:Melphalan