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Drug Evaluation

Efficacy of trabectedin for the treatment of liposarcoma

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Pages 1953-1962 | Received 24 May 2016, Accepted 23 Aug 2016, Published online: 15 Sep 2016
 

ABSTRACT

Introduction: Trabectedin (ET-743) is a synthetic marine derived alkylating agent, extracted originally from a Caribbean Sea sponge. It is approved for the treatment of Soft Tissue sarcomas (STS) in Europe and recently by the FDA for liposarcomas and leiomyosarcomas.

Areas covered: Trabectedin has multiple mechanisms of action, including one targeting the FUS-CHOP oncogene in Myxoid/Round cell Liposarcomas. Numerous Phase I, II and III clinical trials have been conducted with Trabectedin. It has been studied as monotherapy or in combination with other chemotherapeutic agents. The recommended dose based on clinical trials is 1.5 milligrams/m2 continuous infusion over 24 hours once every 3 weeks for STS with evidence of disease control in multiple clinical trials at this dose. The most common Grade 3/4 toxicities include neutropenia and transient noncumulative elevations of ALT and AST. Steroid pretreatment has shown efficacy in reducing liver and bone marrow toxicity. In phase III testing comparing trabectedin to dacarbazine, trabectedin was associated with a significantly improved progression free survival rate in patients with advanced lipo- and leiomyosarcomas.

Expert opinion: Trabectedin is an important new addition to the limited treatment options currently available for STS, especially for patients with liposarcoma that have progressed on standard chemotherapeutic regimens.

Declaration of interest

M von Mehren is a consultant for and member of the scientific steering committee on trabectedin trials and a consultant for Esai. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Additional information

Funding

This paper is not funded

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