Unmet Medical Needs and Future Perspectives for Leiomyosarcoma Patients-A Position Paper from the National LeioMyoSarcoma Foundation (NLMSF) and Sarcoma Patients EuroNet (SPAEN).

Robin L Jones,Scott M Schuetze,Denise Reinke,Seth M Pollack,Kathrin Schuster,Rebecca A Gladdy,Scott Okuno,Breelyn Wilky,Bernd Kasper,Annie Achee,Neeta Somaiah,Brian A Van Tine,Gerard Van Oortmerssen,Matthew L Hemming,Paul Huang,Roberta Sanfilippo,Jonathan Trent,Matthew Ingham,Roger Wilson

doi:10.3390/cancers13040886

Abstract

Simple SummaryIn this position paper, we aim to summarize state-of-the-art treatments for patients with leiomyosarcomas in order to identify knowledge gaps and current unmet needs, thereby guiding the community to design innovative clinical trials and basic research and close these research gaps. This white paper arose from a leiomyosarcoma research meeting in October 2020 hosted by the National LeioMyoSarcoma Foundation (NLMSF) and Sarcoma Patients EuroNet (SPAEN).As leiomyosarcoma patients are challenged by the development of metastatic disease, effective systemic therapies are the cornerstone of outcome. However, the overall activity of the currently available conventional systemic treatments and the prognosis of patients with advanced or metastatic disease are still poor, making the treatment of this patient group challenging. Therefore, in a joint effort together with patient networks and organizations, namely Sarcoma Patients EuroNet (SPAEN), the international network of sarcoma patients organizations, and the National LeioMyoSarcoma Foundation (NLMSF) in the United States, we aim to summarize state-of-the-art treatments for leiomyosarcoma patients in order to identify knowledge gaps and current unmet needs, thereby guiding the community to design innovative clinical trials and basic research and close these research gaps. This position paper arose from a leiomyosarcoma research meeting in October 2020 hosted by the NLMSF and SPAEN.

Highlights

Soft-tissue sarcomas (STS) represent a highly heterogeneous group of mesenchymal malignancies comprising approximately 175 distinct histological subtypes
The trial demonstrated significantly longer progression-free survival (PFS) in the trabectedin arm compared to the dacarbazine arm, but no significant difference in overall survival (OS) [21]
In a randomized phase 3 trial in first-line advanced STS, no significant difference in response rate, PFS and OS was observed between single-agent doxorubicin and gemcitabine plus docetaxel, doxorubicin was better tolerated

Summary

Introduction

Soft-tissue sarcomas (STS) represent a highly heterogeneous group of mesenchymal malignancies comprising approximately 175 distinct histological subtypes. Leiomyosarcoma (LMS) is one of the most frequent subtypes, accounting for approximately 10–20% of all STS. Younger patients may be affected, especially in the context of LMS predisposition genetic syndromes such as Li-Fraumeni or Hereditary Leiomyomatosis and Renal Cell Cancer syndromes. LMS is thought to form from the smooth muscle or their precursor cells, and can arise anywhere in the body with a predilection for tumors in the uterus, the retroperitoneum and the extremities [1]. LMS can be divided into “extra-uterine” (retroperitoneal, inferior vena cava or renal vein, gastrointestinal, extremity, or subcutaneous) and “uterine” LMS, each with distinct clinicopathological characteristics [2]. Retrospective data suggest that there may be differential sensitivity to chemotherapy, but this still requires confirmation [4,5]

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