Open Access Research article

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

Jean-Yves Blay1*, Antoine Italiano2, Isabelle Ray-Coquard1, Axel Le Cesne3, Florence Duffaud4, Maria Rios5, Olivier Collard6, François Bertucci7, Emmanuelle Bompas8, Nicolas Isambert9, Loic Chaigneau10, Philippe Cassier1, Binh Bui2, Gauthier Decanter11, Olfa Derbel1, Jean-Michel Coindre2, Patrick Zintl11, Nadia Badri11 and Nicolas Penel12

Author affiliations

1 Centre Leon Berard, Lyon, France

2 Institut Bergonie, Bordeaux, France

3 Institut Gustave Roussy, Villejuif, France

4 Hopital de La Timone, Marseille, France

5 Centre Alexis Vautrin, Nancy, France

6 Institut de Cancérologie de la Loire, Saint Etienne, France

7 Institut Paoli-Calmettes Calmette, Marseille, France

8 Centre René Gauducheau, Nantes, France

9 Centre GF Leclerc, Dijon, France

10 CHU Besançon, Besançon, France

11 Pharmamar, Madrid, Spain

12 Centre Oscar Lambret, Lille, France

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Citation and License

BMC Cancer 2013, 13:64  doi:10.1186/1471-2407-13-64

Published: 6 February 2013



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.


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.


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.


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.