@article{099aff0c422b46069d86479b3e64c211,
title = "What Clinical Trials Are Needed for Treatment of Leiomyosarcoma?",
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",
author = "Bernd Kasper and Lorenzo D{\textquoteright}Ambrosio and Davis, {Elizabeth J.} and Matthew Ingham and Broto, {Javier Martin} and Trent, {Jonathan C.} and {van Houdt}, {Winan J.} and {Van Tine}, {Brian A.}",
note = "Funding Information: Soft tissue sarcomas (STS) represent a highly heterogeneous group of mesenchymal malignancies comprising more than 150 histological subtypes. Leiomyosarcoma (LMS) is one of the most frequent subtypes accounting for approximately 20% of patients. LMS occurs in middle-aged or older adults with a female predominance. LMS originates from the smooth muscle or their precursor cells, and thus can arise anywhere in the body with a predilection for the retroperitoneum, the extremities, and the uterus []. LMS can be divided into “extra-uterine” (retroperitoneal, gastrointestinal, extremity, or subcutaneous) and “uterine” LMS, each with distinct clinicopathological characteristics [, ]. Diagnosis and staging of patients with LMS are in line with the general recommendations for STS and visceral sarcomas [] and overall management of LMS patients should be part of a multidisciplinary team in a high-volume sarcoma reference center. Despite complete resection of the primary tumor, LMS patients frequently develop metastatic disease; therefore, effective systemic therapies are needed. However, the overall activity of the currently available conventional systemic therapies and the prognosis of patients with advanced or metastatic disease are still poor, making the treatment of LMS patients challenging. Having clearly identified an unmet medical need, designing and performing meaningful clinical studies are of utmost importance to improve the prognosis of this patient population. Therefore, the aim of this review is to briefly summarize state-of-the-art treatments for LMS patients and to describe trial characteristics for the optimal design of clinical studies in this patient group. This work is based on a recent joint white paper from the National LeioMyoSarcoma Foundation (NLMSF) in collaboration with Sarcoma Patients EuroNet (SPAEN) and has been supported by the NLMSF [••]. Funding Information: 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. Publisher Copyright: {\textcopyright} 2022, The Author(s).",
year = "2022",
month = mar,
doi = "10.1007/s11864-021-00928-y",
language = "English",
volume = "23",
pages = "439--449",
journal = "Current Treatment Options in Oncology",
issn = "1527-2729",
number = "3",
}