title: Phase II clinical trial of neoadjuvant trabectedin in patients with advanced localized myxoid liposarcoma.
abstract: BACKGROUND
  To evaluate neoadjuvant trabectedin (1.5 mg/m(2) 24-h i.v. infusion every 3 weeks; three to six cycles) in patients with locally advanced myoxid liposarcoma (ML) previously untreated with chemotherapy or radiation.
  
  
  METHODS
  Primary efficacy end point was pathological complete response (pCR) or tumoral regression rate. Objective response according to RECIST (v.1.0) was a secondary end point.
  
  
  RESULTS
  Three of 23 assessable patients had pCR [13%; 95% confidence interval (CI), 3% to 34%]. Furthermore, very good and moderate histological responses were observed in another 2 and 10 patients, respectively. Histological decrement in the cellular and vascular tumor component and maturation of tumor cells to lipoblasts were observed in both myoxid and myoxid/round cell variants. Seven patients had partial response according to RECIST (objective response rate of 24%; 95% CI, 10% to 44%). No disease progression was reported. Neoadjuvant trabectedin was usually well tolerated, with a safety profile similar to that described in patients with soft tissue sarcoma or other tumor types.
  
  
  CONCLUSIONS
  Trabectedin 1.5 mg/m(2) given as a 24-h i.v. infusion every 3 weeks is a therapeutic option in the neoadjuvant setting of ML.
authors.affiliation: Department of Surgery, National Cancer Institute, Milano, Italy. Electronic address: alessandro.gronchi@istitutotumori.mi.it.
authors.forename: A
authors.initials: A
authors.lastname: Gronchi
authors.affiliation: Department of Medical Oncology, Institute Bergonié, Bourdaux.
authors.forename: B N
authors.initials: BN
authors.lastname: Bui
authors.affiliation: Departments of Surgery; Medical Oncology, Institute Gustave Roussy, Paris, France.
authors.forename: S
authors.initials: S
authors.lastname: Bonvalot
authors.affiliation: Department of Surgery, National Cancer Institute, Milano, Italy.
authors.forename: S
authors.initials: S
authors.lastname: Pilotti
authors.affiliation: Department of Chemotherapy, Orthopedic Institute Rizzoli, Bologna, Italy.
authors.forename: S
authors.initials: S
authors.lastname: Ferrari
authors.affiliation: Division of Surgical Oncology and Thoracic Surgery, Mannheim University Medical Center, University of Heidelberg, Germany.
authors.forename: P
authors.initials: P
authors.lastname: Hohenberger
authors.affiliation: Department of Internal Medicine, Carver College of Medicine, Division of Hematology, Oncology and Blood and Marrow Transplantation, Iowa.
authors.forename: R J
authors.initials: RJ
authors.lastname: Hohl
authors.affiliation: Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, USA.
authors.forename: G D
authors.initials: GD
authors.lastname: Demetri
authors.affiliation: Departments of Surgery; Medical Oncology, Institute Gustave Roussy, Paris, France.
authors.forename: A
authors.initials: A
authors.lastname: Le Cesne
authors.affiliation: Department of Clinical R&D, PharmaMar, Colmenar Viejo, Madrid, Spain.
authors.forename: P
authors.initials: P
authors.lastname: Lardelli
authors.affiliation: Department of Clinical R&D, PharmaMar, Colmenar Viejo, Madrid, Spain.
authors.forename: I
authors.initials: I
authors.lastname: Pérez
authors.affiliation: Department of Clinical R&D, PharmaMar, Colmenar Viejo, Madrid, Spain.
authors.forename: A
authors.initials: A
authors.lastname: Nieto
authors.affiliation: Department of Clinical R&D, PharmaMar, Colmenar Viejo, Madrid, Spain.
authors.forename: J C
authors.initials: JC
authors.lastname: Tercero
authors.affiliation: Department of Clinical R&D, PharmaMar, Colmenar Viejo, Madrid, Spain.
authors.forename: V
authors.initials: V
authors.lastname: Alfaro
authors.affiliation: Department of Surgery, National Cancer Institute, Milano, Italy.
authors.forename: E
authors.initials: E
authors.lastname: Tamborini
authors.affiliation: Department of Medical Oncology; Léon Bérard Cancer Center, Lyon, France.
authors.forename: J Y
authors.initials: JY
authors.lastname: Blay
mesh_terms: D000328:Adult; D000368:Aged; D018906:Antineoplastic Agents, Alkylating / Q000627:therapeutic use*; D004149:Dioxoles / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D018208:Liposarcoma, Myxoid / Q000188:drug therapy*; D008297:Male; D008875:Middle Aged; D020360:Neoadjuvant Therapy; D044005:Tetrahydroisoquinolines / Q000627:therapeutic use*; D000077606:Trabectedin; D055815:Young Adult
chemical_list: D018906:Antineoplastic Agents, Alkylating; D004149:Dioxoles; D044005:Tetrahydroisoquinolines; D000077606:Trabectedin