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Background: Rhabdomyosarcomas are the most common soft tissue sarcoma in children, and pediatric alveolar rhabdomyosarcoma (ARMS) prognosis has improved based on cooperative studies. However, in adults, ARMS is significantly rarer, has poorer outcomes, and currently lacks optimal treatment strategies. Objective: This study aimed to evaluate the clinical outcome of an adult ARMS population with different front-line systemic chemotherapies and determine if any chemotherapy regimen is associated with improved survival. Materials and methods: This is a retrospective study of histologically confirmed fusion-positive ARMS patients over 18 years of age, who were treated at MD Anderson Cancer Center (MDACC) from 2004 to 2021 and received systemic chemotherapy. Descriptive clinical statistics were performed, including staging, front-line chemotherapy, multimodal therapy usage, response rates, and survival analyses. Results: 49 ARMS patients who received upfront chemotherapy were identified. Locoregional treatments included radiotherapy (RT) alone (29%, n = 14), surgery alone (10%, n = 5), or both (45%, n = 22). Median overall survival (OS) for the entire cohort was 3.6 years, and the overall response rate to systemic therapy was 89%. No chemotherapy regimen showed OS benefit, specifically analyzing the pediatric-based vincristine, actinomycin-D, cyclophosphamide (VAC) or adult-based vincristine, doxorubicin, ifosfamide (VDI) regimens, even when controlled for other clinical risk factors. Conclusion: In this single-center contemporary series, adult ARMS patient outcomes remain poor. There was no statistically significant OS difference in patients who did or did not receive adult or pediatric based ARMS regimens, although a high overall response rate to chemotherapy was seen across the entire cohort. Based on these observations, further randomized prospective studies are necessary to delineate which frontline chemotherapy regimen is most beneficial in this rare adult cancer. [ABSTRACT FROM AUTHOR]

Purpose: To evaluate outcomes following percutaneous image‐guided ablation of soft tissue sarcoma metastases to the liver. Materials and Methods: A single‐institution retrospective analysis of patients with a diagnosis of metastatic soft tissue sarcoma who underwent percutaneous image‐guided ablation of hepatic metastases between January 2011 and December 2021 was performed. Patients with less than 60 days of follow‐up after ablation were excluded. The primary outcome was local tumor progression‐free survival (LPFS). Secondary outcomes included overall survival, liver‐specific progression‐free survival. and chemotherapy‐free survival. Results: Fifty‐five patients who underwent percutaneous ablation for 84 metastatic liver lesions were included. The most common histopathological subtypes were leiomyosarcoma (23/55), followed by gastrointestinal stromal tumor (22/55). The median treated liver lesions was 2 (range, 1–8), whereas the median size of metastases were 1.8 cm (0.3–8.7 cm). Complete response at 2 months was achieved in 90.5% of the treated lesions. LPFS was 83% at 1 year and 80% at 2 years. Liver‐specific progression‐free survival was 66% at 1 year and 40% at 2 years. The overall survival at 1 and 2 years was 98% and 94%. The chemotherapy‐free holiday from the start of ablation was 71.2% at 12 months. The complication rate was 3.6% (2/55); one of the complications was Common Terminology Criteria for Adverse Events grade 3 or higher. LPFS subgroup analysis for leiomyosarcoma versus gastrointestinal stromal tumor suggests histology‐agnostic outcomes (2 years, 89% vs 82%, p =.35). Conclusion: Percutaneous image‐guided liver ablation of soft tissue sarcoma metastases is safe and efficacious. Image‐guided ablation of sarcoma metastasis is a promising modality for treating sarcoma metastases to the liver, with a local tumor progression‐free survival of 83% and 80% at 1 and 2 years. Subgroup analysis demonstrated no significant differences between the local tumor progression‐free survival of gastrointestinal stromal tumor versus leiomyosarcoma, suggesting a histology‐agnostic treatment paradigm. [ABSTRACT FROM AUTHOR]

Simple Summary: Undifferentiated pleomorphic sarcomas (UPSs) represent 10–20% of all soft tissue sarcomas (STSs) and have quickly emerged as one of the more immune-sensitive types. There are few real-world data on the use of immune checkpoint blockade (ICB) in UPS patients and those with other high-grade pleomorphic STSs. This is a retrospective, observational study of all patients with metastatic high-grade pleomorphic sarcomas treated with FDA-approved ICB at MD Anderson Cancer Center intended to describe the efficacy and toxicity of ICB in this particular group of patients. We find that our outcomes are comparable to those in the published literature and pose a question regarding the need to further evaluate the optimal sequencing of radiotherapy and prior lines of systemic therapy. Background: Undifferentiated pleomorphic sarcomas (UPSs) are amongst the most common subtypes of soft-tissue sarcomas. Few real-world data on the use of immune checkpoint blockade (ICB) in UPS patients and other high-grade pleomorphic STS patients are available. Purpose: The purpose of our study is to describe the efficacy and toxicity of ICB in patients with advanced UPSs and other high-grade pleomorphic sarcomas treated at our institution. Methods: This is a retrospective, observational study of all patients with metastatic high-grade pleomorphic sarcomas treated with FDA-approved ICB at MD Anderson Cancer Center between 1 January 2015 and 1 January 2023. Patients included in trials for which results are not yet published were excluded. Results: Thirty-six patients with advanced/metastatic pleomorphic sarcomas were included. The median age was 52 years. A total of 26 patients (72%) had UPSs and 10 patients (28%) had other high-grade pleomorphic sarcomas. The median follow-up time was 8.8 months. The median PFS was 2.9 months. The 3-month PFS and 6-month PFS were 46% and 32%, respectively. The median OS was 12.9 months. The 12-month OS and 24-month OS were 53% and 29%, respectively. The best response, previous RT, and type of ICB treatment were significantly and independently associated with shorter PFS (p = 0.0012, p = 0.0019 and p = 0.036, respectively). No new safety signal was identified, and the toxicity was overall manageable with no toxic deaths and only four patients (11%) stopping treatment due to toxicity. Conclusions: Real-world retrospective data are consistent with the published literature, with a promising 6-month PFS of 32%. Partial or stable responders to ICB treatment have significantly improved PFS compared to progressors. [ABSTRACT FROM AUTHOR]

Objective: Well‐differentiated (WDLPS) and dedifferentiated liposarcoma (DDLPS) account for the majority of liposarcomas. Although gemcitabine‐docetaxel is used as second‐line treatment in soft tissue sarcomas, its efficacy in WDLPS/DDLPS is not established. This study retrospectively analyzed the efficacy of gemcitabine regimens in WDLPS/DDLPS. Methods: All patients with WDLPS or DDLPS who received gemcitabine‐based chemotherapy at our institution between September 2002 and January 2021 were included. Response was evaluated by an independent radiologist using RECIST 1.1. The Kaplan–Meier method was used to estimate distributions of survival outcomes and log‐rank tests were used to compare survival outcomes between subgroups. Results: Sixty‐five WDLPS/DDLPS patients were included. Seven patients (10.8%) received a gemcitabine‐based regimen more than once, totaling 72 treatments. The median age at the start of treatment was 66 years (range 32–80 years). Sixty‐five (90.3%) regimens were gemcitabine‐docetaxel, and 7 (9.7%) were gemcitabine alone. Majorities of treatments were for disease that was recurrent/metastatic (86.1%), was abdominal/retroperitoneal (83.3%), and had DDLPS components (88.9%), while 25.0% of treatments were for multifocal disease. The overall response rate was 9.7% (7/72). All responses were in patients with documented DDLPS. The median time to progression was 9.2 months (95% CI 5.3–12.3 months). The median overall survival from the start of therapy was 18.8 months (95% CI 13.1–32.4 months). Conclusion: Gemcitabine‐docetaxel is an efficacious second‐line treatment for DDLPS. Though cross study comparisons are not advisable, response to gemcitabine‐docetaxel compares favorably to current standard options trabectedin and eribulin. This combination is a valid comparator arm for future second‐line trials in DDLPS. [ABSTRACT FROM AUTHOR]

Palbociclib has been evaluated in early phase trials for well‐differentiated liposarcoma (WDLPS) and dedifferentiated liposarcoma (DDLPS) patients, with reported median progression‐free survival (PFS) of 18 weeks. Here, we report on real‐world use and surgical outcomes associated with palbociclib treatment. We retrospectively reviewed 61 consecutive patients with retroperitoneal WDLPS (n = 14) or DDLPS (n = 47) treated with palbociclib monotherapy between 1 March 2016 and 28 February 2021 at The University of Texas MD Anderson Cancer Center. At palbociclib initiation, median age was 64 (interquartile range [IQR] 56‐72). In WDLPS and DDLPS cohorts, the median number of prior systemic treatments was 0 (IQR 0‐0) and 2 (IQR 0‐4), respectively. Median number of prior surgeries was 2 (WDLPS IQR 1‐2.75) and 2 (DDLPS IQR 1‐3). Median PFS was 9.2 (WDLPS IQR 3.9‐21.9) and 2.6 months (DDLPS IQR 2.0‐6.1), with median time on treatment of 7.4 months (WDLPS IQR 3.5‐14.2) and 2.7 months (DDLPS IQR 2.0‐5.7). Twelve patients ultimately underwent surgical resection. Resections were macroscopically complete (R0/R1) in half (n = 6/12), among whom only one patient experienced relapse after resection (median follow‐up 7.5 months). All patients who underwent macroscopically incomplete resections progressed after surgery with median time to progression of 3.3 months (IQR 2.3‐4.4). Surgery after palbociclib treatment was not associated with improved overall survival. Efficacy of palbociclib monotherapy for patients with advanced WDLPS and DDLPS is disappointing. While palbociclib may have been used to delay surgery, there was no clear benefit from treatment and few patients achieved prolonged tumor control. [ABSTRACT FROM AUTHOR]

BACKGROUND. Treatment options are limited in patients with recurrent or metastatic disease after initial treatment of soft-tissue sarcoma (STS) by surgical resection, radiation, or systemic therapy. Percutaneous cryoablation may provide a complementary minimally invasive option in this setting. OBJECTIVE. The purpose of this article was to assess the safety and efficacy of percutaneous cryoablation performed for local control of treatment-refractory recurrent or metastatic STS. METHODS. This single-institution retrospective study included adult patients who underwent percutaneous cryoablation from January 2016 to April 2023 to achieve local control of recurrent or metastatic STS after earlier treatment (surgery, radiation, or chemotherapy). For each treated lesion, a single interventional radiologist rereviewed intraprocedural images to assess for adequate coverage by the ice ball of the entire lesion and a 5-mm or greater margin in all dimensions. Complications and outcomes were extracted from medical records. The primary end point for procedure efficacy was 1-year local progression-free survival. RESULTS. The study included 141 patients (median age, 66 years; 90 women, 51 men) who underwent 217 cryoablation procedures to treat 250 recurrent or metastatic STS lesions. The most common STS histologic types were leiomyosarcoma (56/141) and liposarcoma (39/141). Lesions had a mean long-axis diameter of 2.0 cm (range, 0.4-11.0 cm). Adequate ice-ball coverage was achieved for 82% (204/250) of lesions. The complication rate was 2% (4/217), including three major complications and one minor complication. Patients' median postablation follow-up was 25 months (range, 3-80 months). Local progression-free survival rate was 86% at 1 year and 80% at 2 years. The chemotherapy-free survival rate was 45% at 1 year and 31% at 2 years. The overall survival (OS) rate was 89% at 1 year and 80% at 2 years. In Kaplan-Meier analysis, leiomyosarcoma, in comparison with liposarcoma, had significantly higher local progression-free survival but no significant difference in OS. In multivariable analysis, factors independently associated with an increased risk for local progression included inadequate ice-ball coverage (HR = 7.34) and a lesion location of peritoneum (HR = 3.63) or retroperitoneum (HR = 3.71) relative to lung. CONCLUSION. Percutaneous cryoablation has a favorable safety and efficacy profile in patients with recurrent or metastatic STS after earlier treatments. CLINICAL IMPACT. Percutaneous cryoablation should be considered for local control of treatment-refractory STS.