ABSTRACT
Introduction: There has been significant progress in the development of new anti-soft tissue sarcoma (STS) medications with an orphan drug status in the last two decades. The histological diversity of STS (more than 50 histotypes) makes it challenging to ascertain the treatment choice in a patient-centered manner. Authors aim to reveal especially the place of trabectedin in the treatment of STS in the context of the existing competitive treatments.
Areas covered: An umbrella review was performed of level one of evidence, as defined for Supportive and Palliative Care Studies for orphan drugs. Systematic reviews, meta-analyses and phase 3 clinical trials published until January 2019 were retrieved from Medline, Scopus, Cochrane databases, and Google Scholar.
Expert opinion: Trabectedin is indicated for cases with relapse or as a second-line treatment, either as a monotherapy or in combination with other anti-cancer drugs. Although not universally useful, trabectedin does not have a significant adverse effect on quality of life, presents a favorable toxicological profile and boasts achievement of disease control and improvements in progression-free survival. Further studies are urgently needed to evaluate the efficacy and safety of trabectedin as compared with the emerging competitive drugs.
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Article highlights
Soft tissue sarcomas (STS) are rare tumors treated with established market drugs, but gaps in treatment outcomes exist.
Trabectedin is used as a second-line treatment for L-sarcoma in the USA and any STS in the rest of the world.
A unique mechanism of action involving changes in the tumor microenvironment.
Standard chemotherapeutic regimens with established drugs are still preferred in a majority of cases.
The rarity of the disease limits research efforts; further rigorous research is needed to uncover potential.
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Declaration of interest
The authors have no 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. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.
Reviewer Disclosures
Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.
Notes
1. Authors did not provide the width of confidence interval.
2. The planned meta-analysis could not be done, because there were no common comparators in the studies identified.