title: The off-label use of targeted therapies in sarcomas: the OUTC'S program.
abstract: BACKGROUND
  Few targeted therapies (TTs) are registered for sarcoma treatment despite numerous phase II studies and yet there are potential treatment options for patients after standard treatment escape. The French Sarcoma Group - Bone Tumor Study Group (GSF-GETO) created a national registry to evaluate the outcome of patients treated with off-label TTs.
  
  
  METHODS
  Every consecutive sarcoma-patient receiving an off-label TT outside a clinical trial was included. The objective was to describe this patient efficacy and safety data in routine practice.
  
  
  RESULTS
  From October 2008 to October 2011, 249 patients in 24 centers received 278 treatment lines with TTs. Twenty-five histological subtypes were included: most frequent were leiomyosarcoma (n=48, receiving sorafenib in 63%, and sunitinib in 27%), GIST (n=39, receiving sorafenib in 79%), and angiosarcoma (n=18, receiving sorafenib in 78%). The overall response rate to TTs was 15% (95% CI [10,6-20,2]), the disease control rate at 2 months was 59%. The median progression-free survival was 4,1 months (IC 95% [3,2-4,8]). Three complete responses were observed. No toxic death occurred, grade 3 and 4 toxicities were reported in 74 (27%) and 14 patients (5%) respectively.
  
  
  CONCLUSIONS
  Off-label TTs can be used for sarcoma patients in routine practice with an acceptable toxicity profile and efficacy similar to that reported in non-randomized clinical trials.
authors.forename: Lauriane
authors.initials: L
authors.lastname: Eberst
authors.forename: Claire
authors.initials: C
authors.lastname: Cropet
authors.forename: Axel
authors.initials: A
authors.lastname: Le Cesne
authors.forename: Patricia
authors.initials: P
authors.lastname: Pautier
authors.forename: Nicolas
authors.initials: N
authors.lastname: Penel
authors.forename: Antoine
authors.initials: A
authors.lastname: Adenis
authors.forename: Christine
authors.initials: C
authors.lastname: Chevreau
authors.forename: Jacques-Olivier
authors.initials: JO
authors.lastname: Bay
authors.forename: Olivier
authors.initials: O
authors.lastname: Collard
authors.forename: Didier
authors.initials: D
authors.lastname: Cupissol
authors.forename: Florence
authors.initials: F
authors.lastname: Duffaud
authors.forename: Jean-Claude
authors.initials: JC
authors.lastname: Gentet
authors.forename: Sophie
authors.initials: S
authors.lastname: Piperno-Neumann
authors.forename: Perrine
authors.initials: P
authors.lastname: Marec-Berard
authors.forename: Emmanuelle
authors.initials: E
authors.lastname: Bompas
authors.forename: Antoine
authors.initials: A
authors.lastname: Thyss
authors.forename: Loic
authors.initials: L
authors.lastname: Chaigneau
authors.forename: Philippe
authors.initials: P
authors.lastname: Cassier
authors.forename: François
authors.initials: F
authors.lastname: Bertucci
authors.forename: Jean-Yves
authors.initials: JY
authors.lastname: Blay
authors.affiliation: Centre Léon Bérard, 28 rue Laennec 69 373, LYON CEDEX 08, France. isabelle.ray-coquard@lyon.unicancer.fr.
authors.forename: Isabelle
authors.initials: I
authors.lastname: Ray-Coquard
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000970:Antineoplastic Agents / Q000009:adverse effects / Q000627:therapeutic use*; D003520:Cyclophosphamide / Q000009:adverse effects / Q000627:therapeutic use; D018572:Disease-Free Survival; D016503:Drug Delivery Systems; D005260:Female; D006801:Humans; D007211:Indoles / Q000009:adverse effects / Q000627:therapeutic use; D008297:Male; D008875:Middle Aged; D009536:Niacinamide / Q000009:adverse effects / Q000031:analogs & derivatives / Q000627:therapeutic use; D056687:Off-Label Use; D010671:Phenylurea Compounds / Q000009:adverse effects / Q000627:therapeutic use; D011758:Pyrroles / Q000009:adverse effects / Q000627:therapeutic use; D012042:Registries; D012509:Sarcoma / Q000188:drug therapy* / Q000473:pathology; D020123:Sirolimus / Q000009:adverse effects / Q000627:therapeutic use; D000077157:Sorafenib; D000077210:Sunitinib; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D000970:Antineoplastic Agents; D007211:Indoles; D010671:Phenylurea Compounds; D011758:Pyrroles; D009536:Niacinamide; D003520:Cyclophosphamide; D000077157:Sorafenib; D000077210:Sunitinib; D020123:Sirolimus