Experience of the use of trabectedin in pretreated unresectable, advanced, or metastatic soft tissue sarcoma in nine centers in Lebanon on a compassionate-use basis.

Abstract

e20533 Background: Soft tissue sarcoma (STS) comprises a large variety of rare malignant tumors. Development of distant metastasis is frequent, even in patients undergoing initial curative surgery. Trabectedin, a tetrahydroisoquinoline alkaloid isolated from the Caribbean marine tunicate Ecteinascidia turbinata, was approved in 2007 by EMEA for patients with advanced STS after failure of previous lines chemotherapy. In this study, we retrospectively analyzed 15 patients who had been treated with trabectedin between 2008 and 2010 in 9 centers in Lebanon on a compassionate use basis. Methods: Fifteen patients with advanced refractory sarcoma were treated with trabectedin on a named patient compassionate basis program. All patients had been heavily pre-treated with at least 2 previous lines of chemotherapy. Results: The patients received a median of three cycles of treatment. Five patients achieved stable disease. The estimated 3 month progression-free survival median was 48%. Four patients experienced early disease progression, and one patient died while on treatment. None of the deaths was due to treatment-related toxicity, but to disease progression. Conclusions: Trabectedin is a generally well tolerated and effective treatment for soft tissue sarcomas STS even in a heavily pre-treated patient population. Major toxicities include changes in liver function tests and nausea. Dose reductions usually allow continuation of treatment. Progression-free survival and overall survival were significantly prolonged in responders and seem not to be inferior to other clinically used second-line treatments.