title: Treatment of children with metastatic soft tissue sarcoma with oral maintenance compared to high dose chemotherapy: report of the HD CWS-96 trial.
abstract: OBJECTIVE
  We prospectively studied the efficacy of high dose therapy (HDT) versus an oral maintenance treatment (OMT) in patients with stage IV soft tissue sarcoma (STS).
  
  
  METHODS
  Both groups were pretreated with the CEVAIE combination consisting of carboplatin, etoposide, vincristine, actinomycin D, ifosfamide, and epirubicin. HDT consisted of a tandem cycle of thiotepa (600 mg/m(2)) plus cyclophosphamide (4,500 mg/m(2)) and melphalan (120 mg/m(2)) plus etoposide (1,800 mg/m(2)). This treatment was compared with OMT, consisting of four cycles trofosfamide (10 days 2 x 75 mg/m(2)/day) plus etoposide (10 days 2 x 25 mg/m(2)/day), and 4 cycles trofosfamide (10 days 2 x 75 mg/m(2)/day) plus idarubicin (10 days 4 x 5 mg/m(2)). Eligibility criteria were: diagnosis confirmed by reference pathology, primary stage IV, below 22 years of age, and having completed the study therapy.
  
  
  RESULTS
  From 96 patients 45 were treated with HDT and 51 with OMT. The main risk parameters were equally distributed in both arms. After a median follow-up of 57.4 months, 11/45 (24.4%) patients in the HDT-arm and 26/51 (57.8%) patients in OMT-arm were alive. Kaplan-Meier analysis demonstrated an overall survival for the whole group of 0.27 (OMT group: 0.52, HDT group 0.27, log rank P = 0.03). The proportional hazard analysis for patients with rhabdomyosarcoma (RMS) or "RMS-like" tumors (77.1% of all patients) demonstrated an independent benefit of OMT on outcome.
  
  
  CONCLUSIONS
  Oral maintenance therapy seems to be a promising option for patients with RMS-like stage IV tumors.
authors.affiliation: Children's Hospital of Frankfurt University Hospital, Frankfurt, Germany. thomas.klingebiel@kgu.de
authors.forename: Thomas
authors.initials: T
authors.lastname: Klingebiel
authors.forename: Joachim
authors.initials: J
authors.lastname: Boos
authors.forename: Florian
authors.initials: F
authors.lastname: Beske
authors.forename: Erika
authors.initials: E
authors.lastname: Hallmen
authors.forename: Christoph
authors.initials: C
authors.lastname: Int-Veen
authors.forename: Tobias
authors.initials: T
authors.lastname: Dantonello
authors.forename: Joern
authors.initials: J
authors.lastname: Treuner
authors.forename: Helmut
authors.initials: H
authors.lastname: Gadner
authors.forename: Ildiko
authors.initials: I
authors.lastname: Marky
authors.forename: Bernarda
authors.initials: B
authors.lastname: Kazanowska
authors.forename: Ewa
authors.initials: E
authors.lastname: Koscielniak
mesh_terms: D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D016190:Carboplatin / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000008:administration & dosage / Q000031:analogs & derivatives; D003609:Dactinomycin / Q000008:administration & dosage; D015251:Epirubicin / Q000008:administration & dosage; D005047:Etoposide / Q000008:administration & dosage; D005260:Female; D006801:Humans; D015255:Idarubicin / Q000008:administration & dosage; D007069:Ifosfamide / Q000008:administration & dosage; D007223:Infant; D053208:Kaplan-Meier Estimate; D008297:Male; D008558:Melphalan / Q000008:administration & dosage; D012509:Sarcoma / Q000188:drug therapy* / Q000401:mortality; D012983:Soft Tissue Neoplasms / Q000188:drug therapy* / Q000401:mortality; D013852:Thiotepa / Q000008:administration & dosage; D016896:Treatment Outcome; D014750:Vincristine / Q000008:administration & dosage
chemical_list: D003609:Dactinomycin; D015251:Epirubicin; D014750:Vincristine; D005047:Etoposide; D003520:Cyclophosphamide; D013852:Thiotepa; D016190:Carboplatin; C003726:trofosfamide; D008558:Melphalan; D007069:Ifosfamide; D015255:Idarubicin