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The present study was a retrospective single-center study. A total of 81 patients diagnosed with metastatic soft tissue sarcoma were included who received pazopanib therapy. Clinical data, including age at diagnosis, histological subtype, treatments received before pazopanib, number of metastatic sites at the time of initiation of treatment, progression-free survival and overall survival time under pazopanib treatment, side effects and response evaluation in follow-up imaging after initiation of pazopanib therapy, were recorded.

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Purpose: To characterize factors associated with response to immune checkpoint inhibitors (ICIs) in advanced sarcoma.

Experimental Design: This is a retrospective study with a cohort of 216 patients with advanced sarcoma treated with ICIs between 2016-2023 at Stanford Health Care. Overall survival (OS), progression free survival (PFS), objective response rates per RECIST criteria (ORR), and reason for ICI discontinuation were analyzed across histologic subtypes, ICI regimens, tumor mutational burden (TMB), and PD-L1 expression.

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Sarcomas are rare, mesenchymal tumors, representing about 10-15% of all childhood cancers. GD2 is a suitable target for chimeric antigen receptor (CAR) T-cell therapy due to its overexpression in several solid tumors. In this preclinical study, we investigated the potential use of iCasp9.

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Rhabdomyosarcoma in children and young adults.

Virchows Arch

December 2024

Department of Pathology and Laboratory Medicine, Indiana University, 350 W 11st St, Indianapolis, IN, 46202, USA.

Rhabdomyosarcoma (RMS) is the most common soft tissue malignancy in childhood, accounting for 3% of all pediatric malignancies and 50% of all pediatric soft tissue sarcomas. In adolescents and young adults (AYA) however, RMS comprises only 6.5% of all soft tissue sarcomas.

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Background: Rhabdomyosarcoma and other soft tissue sarcomas (STS) with high-risk features are still associated with an unsatisfactory outcome. We evaluated the efficacy of oral maintenance therapy added at the end of standard therapy in patients with high-risk rhabdomyosarcoma and STS.

Methods: CWS-2007-HR was a multicentre, open-label, randomised controlled, phase 3 trial done at 87 centers in 5 countries.

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