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Review
. 2022 Mar;23(3):439-449.
doi: 10.1007/s11864-021-00928-y. Epub 2022 Mar 11.

What Clinical Trials Are Needed for Treatment of Leiomyosarcoma?

Bernd Kasper  1 , Lorenzo D'Ambrosio  2 , Elizabeth J Davis  3 , Matthew Ingham  4 , Javier Martin Broto  5 , Jonathan C Trent  6 , Winan J van Houdt  7 , Brian A Van Tine  8
Affiliations
Review

What Clinical Trials Are Needed for Treatment of Leiomyosarcoma?

Bernd Kasper et al. Curr Treat Options Oncol. 2022 Mar.

Abstract

Leiomyosarcoma is one of the most common subtypes of soft tissue sarcomas accounting for approximately 20% of sarcomas. As leiomyosarcoma patients frequently develop metastatic disease, effective systemic therapies are needed to improve clinical outcomes. The overall activity of the currently available conventional systemic therapies and the prognosis of patients with advanced and/or metastatic disease are poor. As such, the treatment of this patient population remains challenging. As a result, there is a clear unmet medical need, and designing and performing meaningful clinical studies are of utmost importance to improve the prognosis of this patient group. Therefore, the aim of this review is to briefly summarize state-of-the-art treatments for leiomyosarcoma patients and to describe trial characteristics needed for informative clinical studies.

Keywords: Clinical trials; Leiomyosarcoma; NLMSF; Research; Treatment.

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Conflict of interest statement

BK declares personal fees from Ayala, Bayer, Blueprint, Boehringer Ingelheim, GSK, PharmaMar, Roche, and SpringWorks. EJD receives research funding from Bristol-Myers Squibb, Incyte, Five Prime, Karyopharm, Top Alliance BioSciences, Actuate, and Genentech and personal fees from Karyopharm and Deciphera. JCT serves as a consultant for Deciphera, Blueprint, Cogent Biosciences, Epizyme, C4 Therapeutics, and Daiichi-Sankyo. BAVT declares grants from Merck; grants and personal fees from Pfizer; grants from TRACON Pharmaceuticals; grants, personal fees, and others from GlaxoSmithKline; personal fees from Polaris Inc.; personal fees from Lilly; personal fees from Caris Life Sciences; personal fees from Novartis; personal fees from CytRX; personal fees from Plexxikon; personal fees from Epizyme; personal fees from Daiichi Sankyo; personal fees from Adaptimmune; personal fees from Immune Design; personal fees fromBayer; personal fees fromCytokinetics; and personal fees fromDeciphera; and has a patent issued for the use of ME1 as a biomarker and ACXT3102.

Figures

Fig. 1
Fig. 1
Trial characteristics for designing clinical studies in LMS.
See this image and copyright information in PMC

References

References and Recommended Reading

Papers of particular interest, published recently, have been highlighted as: • Of importance •• Of major importance
    1. Martin-Liberal J. Leiomyosarcoma: principles of management. Intractable Rare Dis Res. 2013;2:127–129. - PMC - PubMed
    1. Fletcher B, Hogendoorn PC, Mertens F. WHO Classification of Tumours of Soft Tissue and Bone. World Health Organisation 2013;467.
    1. Reichardt P. Soft tissue sarcomas, a look into the future: different treatments for different subtypes. Future Oncol. 2014;10(Suppl 8):s19–s27. - PubMed
    1. Casali PG, Abecassis N, Aro HT, Bauer S, Biagini R, et al. Soft tissue and visceral sarcomas: ESMO-EURACAN Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2018;29(Suppl 4):iv51–iv67. - PubMed
    1. Kasper B, Achee A, Schuster K, Wilson R, van Oortmerssen G, Gladdy RA, et al. Unmet medical needs and future perspectives for leiomyosarcoma patients-a position paper from the National LeioMyoSarcoma Foundation (NLMSF) and Sarcoma Patients EuroNet (SPAEN) Cancers (Basel) 2021;13:886. - PMC - PubMed

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