Nassar Amin H, El-Am Edward, Denu Ryan, Abou Alaiwi Sarah, El Zarif Talal, Macaron Walid, Abdel-Wahab Noha, Desai Aakash, Smith Caleb, Parikh Kaushal, Abbasi Muhannad, Bou Farhat Elias, Williams James M, Collins Jeremy D, Al-Hader Ahmad, McKay Rana R, Malvar Carmel, Sabra Mohamad, Zhong Caiwei, El Alam Raquelle, Chehab Omar, Lima Joao, Phan Minh, Dalla Pria Hanna Ferreira, Trevino Alexandra, Neilan Tomas G, Kwan Jennifer M, Ravi Vinod, Deshpande Hari, Demetri George, Choueiri Toni K, Naqash Abdul Rafeh
Yale University School of Medicine, New Haven, Connecticut, USA.
Department of Cardiovascular Medicine, Mayo Clinic, Rochester, Minnesota, USA.
JACC CardioOncol. 2024 Jan 16;6(1):71-79. doi: 10.1016/j.jaccao.2023.11.007. eCollection 2024 Feb.
Primary cardiac soft tissue sarcomas (CSTS) affect young adults, with dismal outcomes.
The aim of this study was to investigate the clinical outcomes of patients with CSTS receiving immune checkpoint inhibitors (ICIs).
A retrospective, multi-institutional cohort study was conducted among patients with CSTS between 2015 and 2022. The patients were treated with ICI-based regimens. The Kaplan-Meier method was used to estimate overall survival (OS) and progression-free survival (PFS). Objective response rates were determined according to Response Evaluation Criteria in Solid Tumors version 1.1. Treatment-related adverse events were graded per the Common Terminology Criteria for Adverse Events version 5.0.
Among 24 patients with CSTS, 17 (70.8%) were White, and 13 (54.2%) were male. Eight patients (33.3%) had angiosarcoma. At the time of ICI treatment, 18 patients (75.0%) had metastatic CSTS, and 4 (16.7%) had locally advanced disease. ICIs were administered as the first-line therapy in 6 patients (25.0%) and as the second-line therapy or beyond in 18 patients (75.0%). For the 18 patients with available response data, objective response rate was 11.1% (n = 2 of 18). The median PFS and median OS in advanced and metastatic CSTS (n = 22) were 5.7 months (95% CI: 2.8-13.3 months) and 14.9 months (95% CI: 5.7-23.7 months), respectively. The median PFS and OS were significantly shorter in patients with cardiac angiosarcomas than in those with nonangiosarcoma CSTS: median PFS was 1.7 vs 11 months, respectively ( < 0.0001), and median OS was 3.0 vs 24.0 months, respectively ( = 0.008). Any grade treatment-related adverse events occurred exclusively in the 15 patients with nonangiosarcoma CSTS (n = 7 [46.7%]), of which 6 (40.0%) were grade ≥3.
Although ICIs demonstrate modest activity in CSTS, durable benefit was observed in a subset of patients with nonangiosarcoma, albeit with higher toxicity.
原发性心脏软组织肉瘤（CSTS）影响年轻成年人，预后不佳。
本研究旨在调查接受免疫检查点抑制剂（ICI）治疗的CSTS患者的临床结局。
对2015年至2022年间的CSTS患者进行了一项回顾性、多机构队列研究。患者接受基于ICI的治疗方案。采用Kaplan-Meier方法估计总生存期（OS）和无进展生存期（PFS）。根据实体瘤疗效评价标准第1.1版确定客观缓解率。根据不良事件通用术语标准第5.0版对治疗相关不良事件进行分级。
在24例CSTS患者中，17例（70.8%）为白人，13例（54.2%）为男性。8例（33.3%）患有血管肉瘤。在接受ICI治疗时，18例（75.0%）患者患有转移性CSTS，4例（16.7%）患有局部晚期疾病。6例（25.0%）患者将ICI作为一线治疗，18例（75.0%）患者将ICI作为二线或更晚期治疗。对于18例有可用缓解数据的患者，客观缓解率为11.1%（18例中的2例）。晚期和转移性CSTS（n = 22）的中位PFS和中位OS分别为5.7个月（95%CI：2.8 - 13.3个月）和14.9个月（95%CI：5.7 - 23.7个月）。心脏血管肉瘤患者的中位PFS和OS显著短于非血管肉瘤CSTS患者：中位PFS分别为1.7个月和11个月（<0.0001），中位OS分别为3.0个月和24.0个月（=0.008）。任何级别的治疗相关不良事件仅发生在非血管肉瘤CSTS的15例患者中（n = 7 [46.7%]），其中6例（40.0%）为≥3级。
尽管ICI在CSTS中显示出适度的活性，但在一部分非血管肉瘤患者中观察到了持久的获益，尽管毒性较高。
