Real-world evidence of the efficacy and tolerability of trabectedin in patients with advanced soft-tissue sarcoma

ABSTRACT

Introduction

The efficacy and tolerability of trabectedin in patients with soft tissue sarcoma (STS) have been confirmed by various clinical studies involving lipo- and leiomyosarcomas as well as many other subtypes including translocation-related sarcomas. These data have been obtained from randomized phase II and III clinical trials. Studies in real-world clinical practice are necessary to bridge the efficacy-effectiveness gap and complete the body of evidence. Furthermore, reinforcing clinical experience with data from routine clinical practice allows drug management to be optimized and clinical benefits to be maximized.

Areas covered

The present review provides the most significant data on the efficacy of trabectedin in real-world studies, and the interpretation of real-world experience with trabectedin, in patients with advanced STS.

Expert opinion

Trabectedin has demonstrated durable disease control and an adequate safety profile, indicating it to be a suitable long-term treatment drug associated with a good quality of life. Personalized strategies and individualized objectives are the way forward in the management of STS.

KEYWORDS:

• Trabectedin
• real-world
• soft-tissue sarcoma
• efficacy
• safety

Article highlights

• Efficacy and safety results in real-world studies are consistent with results obtained in clinical trials.

• The ongoing experience with trabectedin is associated with better efficacy outcomes.

• An early use of trabectedin correlates with an increased proportion of patients who are able to achieve long-term treatment.

• Trabectedin has shown activity in multiple STS subtypes.

• Trabectedin can be administered safely for prolonged periods and is well tolerated in elderly patients.

Declaration of interest

Luis Miguel De Sande declares having received support with documentation and funding from PharmaMar. Javier Martin-Broto reports research grants from PharmaMar, Eisai, Immix BioPharma and Novartis outside the submitted work; honoraria for advisory board participation and expert testimony from PharmaMar, honoraria for advisory board participation from Eli Lilly and Company, Bayer and Eisai; and research funding for clinical studies (institutional) from PharmaMar, Eli Lilly and Company, Bayer, Eisai, Lixte, Karyopharm, Deciphera, GSK, Novartis, AROG Pharmaceuticals, Blueprint, Nektar, Forma, Amgen and Daiichi-Sankyo. Jean Yves Blay declares support and honoraria from PharmaMar, Novartis, Roche, MSD, AZ, BMS, Deciphera, GSK. Bernd Kasper reports honoraria and research funding from PharmaMar. Axel Le Cesne reports honoraria from Lilly, Bayer, PharmaMar, Deciphera, Blueprint, Eisai. 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.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was financially supported by PharmaMar.