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 andp= 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.
背景：未分化多形性肉瘤是软组织肉瘤中最常见的亚型之一。目前关于免疫检查点抑制剂在未分化多形性肉瘤及其他高级别多形性软组织肉瘤患者中应用的真实世界数据较为有限。目的：本研究旨在描述本机构晚期未分化多形性肉瘤及其他高级别多形性肉瘤患者接受免疫检查点抑制剂治疗的疗效与毒性特征。方法：本研究为回顾性观察性研究，纳入2015年1月1日至2023年1月1日期间在MD安德森癌症中心接受美国食品药品监督管理局批准免疫检查点抑制剂治疗的转移性高级别多形性肉瘤患者，排除参与尚未公布结果的临床试验病例。结果：共纳入36例晚期/转移性多形性肉瘤患者，中位年龄52岁。其中未分化多形性肉瘤26例（72%），其他高级别多形性肉瘤10例（28%）。中位随访时间8.8个月，中位无进展生存期2.9个月，3个月与6个月无进展生存率分别为46%和32%。中位总生存期12.9个月，12个月与24个月总生存率分别为53%和29%。最佳治疗反应、既往放疗史及免疫检查点抑制剂治疗方案类型均与较短无进展生存期显著独立相关（p值分别为0.0012、0.0019和0.036）。未发现新的安全性信号，总体毒性可控，无毒性相关死亡病例，仅4例患者（11%）因毒性终止治疗。结论：真实世界回顾性数据与已发表文献结果一致，6个月无进展生存率达32%具有临床前景。相较于疾病进展者，免疫检查点抑制剂治疗达到部分缓解或疾病稳定的患者无进展生存期显著改善。
肿瘤(癌症)患者之家