Efficacy of trabectedin in malignant solitary fibrous tumors: a retrospective analysis from the French Sarcoma Group

J Khalifa, M Ouali, L Chaltiel, S Le Guellec, A Le Cesne, J-Y Blay, P Cousin, L Chaigneau, E Bompas, S Piperno-Neumann, B Bui-Nguyen, M Rios, J-P Delord, N Penel, C Chevreau, J Khalifa, M Ouali, L Chaltiel, S Le Guellec, A Le Cesne, J-Y Blay, P Cousin, L Chaigneau, E Bompas, S Piperno-Neumann, B Bui-Nguyen, M Rios, J-P Delord, N Penel, C Chevreau

Abstract

Background: Advanced malignant solitary fibrous tumors (SFTs) are rare soft-tissue sarcomas with a poor prognosis. Several treatment options have been reported, but with uncertain rates of efficacy. Our aim is to describe the activity of trabectedin in a retrospective, multi-center French series of patients with SFTs.

Methods: Patients were mainly identified through the French RetrospectYon database and were treated between January 2008 and May 2013. Trabectedin was administered at an initial dose of 1.5 mg/m(2), q3 weeks. The best tumor response was assessed according to the Response Evaluation Criteria In Solid Tumors 1.1. The Kaplan-Meier method was used to estimate median progression-free survival (PFS) and overall survival (OS). The growth-modulation index (GMI) was defined as the ratio between the time to progression with trabectedin (TTPn) and the TTP with the immediately prior line of treatment (TTPn-1).

Results: Eleven patients treated with trabectedin for advanced SFT were identified. Trabectedin had been used as second-line treatment in 8 patients (72.7 %) and as at least third-line therapy in a further 3 (27.3 %). The best RECIST response was a partial response (PR) in one patient (9.1 %) and stable disease (SD) in eight patients (72.7 %). Disease-control rate (DCR = PR + SD) was 81.8 %. After a median follow-up of 29.2 months, the median PFS was 11.6 months (95 % CI = 2.0; 15.2 months) and the median OS was 22.3 months (95 % CI = 9.1 months; not reached). The median GMI was 1.49 (range: 0.11-4.12).

Conclusion: Trabectedin is a very promising treatment for advanced SFTs. Further investigations are needed.

Figures

Fig. 1
Fig. 1
Kaplan-Meier curves for progression-free and overall survival from the start of trabectedin therapy
Fig. 2
Fig. 2
Comparison of time to progression with trabectedin (TTP2) versus time to progression with prior therapy (TTP1) for the 11 patients. The growth modulation index (GMI) is the ratio between TTP2 and TTP1. Two patients did not progress under trabectedin (patient 1 and patient 5), so that only a minimum predictive GMI could be estimated. All in all, seven patients had a GMI (effective or predictive) > 1.33 (63.6 %, 95 % CI = [30.8–89.1 %])

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