title: Long-term outcome and effect of maintenance therapy in patients with advanced sarcoma treated with trabectedin: an analysis of 181 patients of the French ATU compassionate use program.
abstract: BACKGROUND
  The long term outcome of advanced sarcoma patients treated with trabectedin outside of clinical trials and the utility of maintenance treatment has not been reported.
  
  
  METHODS
  Between 2003 and 2008, patients with advanced sarcoma failing doxorubicin could be treated within a compassionate use program (ATU, Temporary Use Authorization) of trabectedin in France using the standard 3-weekly regimen. Data from 181 patients (55%) were collected from 11 centres and analyzed.
  
  
  RESULTS
  Trabectedin was given in first, second, third or fourth line in metastatic phase in 6%, 37%, 33% and 23% of patients respectively. With a median follow-up of 6 years, median PFS and OS were 3.6 months and 16.1 months respectively. The median number of cycles was 3 (range 1-19). Best response were partial response (PR, n = 18, 10%), stable disease (SD, n = 69, 39%) and progressive disease (PD, n = 83, 46%), non evaluable (NE, n = 9, 5%). Thirty patients (17%) had to be hospitalized for treatment- related side effects. Independent prognostic factors in multivariate analysis (Cox model) were myxoid LPS and line of trabectedin for PFS, and myxoid LPS and retroperitoneal sarcomas for OS. Patients in PR or SD after 6 cycles continuing treatment had a better PFS (median 5.3 vs 10.5 months, p = 0.001) and OS (median 13.9 vs 33.4 months, p = 0.009) as compared to patients who stopped after 6 cycles.
  
  
  CONCLUSIONS
  In this compassionate use program, trabectedin yielded similar or better PFS and OS than in clinical trials. Maintenance treatment beyond 6 cycles was associated with an improved survival.
authors: Centre Leon Berard, Lyon, France. jean-yves.blay@lyon.unicancer.fr
authors: Jean-Yves
authors: JY
authors: Blay
authors: Antoine
authors: A
authors: Italiano
authors: Isabelle
authors: I
authors: Ray-Coquard
authors: Axel
authors: A
authors: Le Cesne
authors: Florence
authors: F
authors: Duffaud
authors: Maria
authors: M
authors: Rios
authors: Olivier
authors: O
authors: Collard
authors: François
authors: F
authors: Bertucci
authors: Emmanuelle
authors: E
authors: Bompas
authors: Nicolas
authors: N
authors: Isambert
authors: Loic
authors: L
authors: Chaigneau
authors: Philippe
authors: P
authors: Cassier
authors: Binh
authors: B
authors: Bui
authors: Gauthier
authors: G
authors: Decanter
authors: Olfa
authors: O
authors: Derbel
authors: Jean-Michel
authors: JM
authors: Coindre
authors: Patrick
authors: P
authors: Zintl
authors: Nadia
authors: N
authors: Badri
authors: Nicolas
authors: N
authors: Penel
mesh_terms: D000328:Adult; D000368:Aged; D018906:Antineoplastic Agents, Alkylating / Q000627:therapeutic use*; D057176:Compassionate Use Trials; D004149:Dioxoles / Q000627:therapeutic use*; D005260:Female; D005602:France; D006801:Humans; D060046:Maintenance Chemotherapy; D008297:Male; D008875:Middle Aged; D009367:Neoplasm Staging; D012189:Retrospective Studies; D012509:Sarcoma / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology*; D044005:Tetrahydroisoquinolines / Q000627:therapeutic use*; D000077606:Trabectedin; D016896:Treatment Outcome
chemical_list: D018906:Antineoplastic Agents, Alkylating; D004149:Dioxoles; D044005:Tetrahydroisoquinolines; D000077606:Trabectedin
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