title: Sorafenib in patients with locally advanced and metastatic chordomas: a phase II trial of the French Sarcoma Group (GSF/GETO).
abstract: BACKGROUND
  There is no consensual treatment of locally advanced or metastatic chordomas.
  
  
  METHODS
  We conducted a multicenter, open-label, uncontrolled phase II trial of sorafenib (800 mg/day). The primary end point was the 9-month progression-free rate according to RECIST 1.1. All patients had documented progressive disease at the time of study entry.
  
  
  RESULTS
  Twenty-seven patients were enrolled between May 2011 and January 2014. The median age was 64 (range, 30-86) years. There were 17 men and 10 women. Twelve patients had been previously treated with chemotherapy and molecularly targeted agents. The maximum toxicity grade per patient was grade 3 in 21 cases (77.8%) and grade 4 in 4 cases (14.8%). Sorafenib provided an intent-to-treat best objective response of 1/27 [3.7%; 95% confidence interval (CI) 0.1% to 19.0%], a 9-month progression-free rate of 73.0% (95% CI 46.1-88.0) and a 12-month overall survival rate of 86.5% (95% CI 55.8-96.5). Survival curves were similar in pretreated and not pretreated patients.
  
  
  CONCLUSIONS
  Additional clinical trials further exploring sorafenib as a treatment of locally advanced or metastatic chordomas are warranted.
authors.affiliation: Department of Medical Oncology, Centre René Gauducheau, Nantes.
authors.forename: E
authors.initials: E
authors.lastname: Bompas
authors.affiliation: Department of Medical Oncology, Institut Gustave Roussy, Villejuif.
authors.forename: A
authors.initials: A
authors.lastname: Le Cesne
authors.affiliation: Methodology and Biostatistics Unit.
authors.forename: E
authors.initials: E
authors.lastname: Tresch-Bruneel
authors.affiliation: Department of General Oncology, Centre Oscar Lambret, Lille.
authors.forename: L
authors.initials: L
authors.lastname: Lebellec
authors.affiliation: Department of Medical Oncology, Institut Curie, Paris.
authors.forename: V
authors.initials: V
authors.lastname: Laurence
authors.affiliation: Department of Medical Oncology, Institut de Cancérologie de la Loire, Lucien Neuwirth, Saint Priest en Jarez.
authors.forename: O
authors.initials: O
authors.lastname: Collard
authors.affiliation: Department of Medical Oncology, Centre Antoine Lacassagne, Nice.
authors.forename: E
authors.initials: E
authors.lastname: Saada-Bouzid
authors.affiliation: Department of Medical Oncology, Centre GF Leclerc, Dijon.
authors.forename: N
authors.initials: N
authors.lastname: Isambert
authors.affiliation: Department of Medical Oncology, Centre Léon Bérard, Lyon.
authors.forename: J Y
authors.initials: JY
authors.lastname: Blay
authors.affiliation: Department of General Oncology, Centre Oscar Lambret, Lille.
authors.forename: E Y
authors.initials: EY
authors.lastname: Amela
authors.affiliation: Department of Medical Oncology, Hopital La Timone, Marseille.
authors.forename: S
authors.initials: S
authors.lastname: Salas
authors.affiliation: Department of Medical Oncology, Institut Claudius Regaud, Toulouse.
authors.forename: C
authors.initials: C
authors.lastname: Chevreau
authors.affiliation: Department of Medical Oncology, Institut Paoli Calmette, Marseille.
authors.forename: F
authors.initials: F
authors.lastname: Bertucci
authors.affiliation: Department of Medical Oncology, Institut Bergonié, Bordeaux.
authors.forename: A
authors.initials: A
authors.lastname: Italiano
authors.affiliation: Clinical Research Unit, Centre Oscar Lambret, Lille.
authors.forename: S
authors.initials: S
authors.lastname: Clisant
authors.affiliation: Department of General Oncology, Centre Oscar Lambret, Lille Clinical Research and Methodology Platform, SIRIC OncoLille Consortium, Lille, France n-penel@o-lambret.fr.
authors.forename: N
authors.initials: N
authors.lastname: Penel
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D002817:Chordoma / Q000188:drug therapy* / Q000401:mortality / Q000556:secondary; D005260:Female; D005500:Follow-Up Studies; D005602:France; D006801:Humans; D008297:Male; D008875:Middle Aged; D009367:Neoplasm Staging; D009536:Niacinamide / Q000031:analogs & derivatives* / Q000627:therapeutic use; D010671:Phenylurea Compounds / Q000627:therapeutic use*; D011379:Prognosis; D012509:Sarcoma / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology; D000077157:Sorafenib; D015996:Survival Rate
chemical_list: D000970:Antineoplastic Agents; D010671:Phenylurea Compounds; D009536:Niacinamide; D000077157:Sorafenib
keywords: chordoma; phase II trial; sorafenib