Intensive chemotherapy for children and young adults with metastatic primitive neuroectodermal tumors of the soft tissue

Bone Marrow Transplant. 2002 Sep;30(5):297-302. doi: 10.1038/sj.bmt.1703617.

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.

Publication types

  • Clinical Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Age Factors
  • Antineoplastic Combined Chemotherapy Protocols / administration & dosage*
  • Bone Marrow Transplantation
  • Child
  • Child, Preschool
  • Combined Modality Therapy
  • Female
  • Humans
  • Male
  • Melphalan / administration & dosage*
  • Neuroectodermal Tumors / mortality
  • Neuroectodermal Tumors / pathology
  • Neuroectodermal Tumors / therapy*
  • Peripheral Blood Stem Cell Transplantation
  • Remission Induction / methods
  • Sarcoma / mortality
  • Sarcoma / pathology
  • Sarcoma / therapy*
  • Survival Analysis
  • Treatment Outcome

Substances

  • Melphalan