Medical Oncology Department, Hospital de la Santa Creu i Sant Pau, Carrer de Sant Quintí, 89, 08041, Barcelona, Spain.
Department of Pathology, Hospital de la Santa Creu i Sant Pau, Barcelona, Spain.
Adv Ther. 2022 Apr;39(4):1596-1610. doi: 10.1007/s12325-022-02051-2. Epub 2022 Feb 7.
The effectiveness of trabectedin for the treatment of leiomyosarcoma and liposarcoma (commonly referred to as L-sarcomas) has been widely evidenced in clinical trials and real-world studies. Nevertheless, available literature on non-L-sarcomas is less abundant. The objective of the present study is to evaluate the effectiveness and safety of trabectedin in a cohort of patients with non-L-sarcomas in the real-world setting.
This retrospective, observational study included 34 patients who received trabectedin in the Hospital de la Santa Creu i Sant Pau (Barcelona, Spain) between October 2013 and July 2020.
The most frequent histologic subtypes were undifferentiated spindle cell/pleomorphic sarcoma (n = 11, 32.4%), synovial sarcoma (n = 6, 17.7%), myxofibrosarcoma (n = 5, 14.7%), and malignant peripheral nerve sheath tumor (n = 4, 11.8%). The mean number of cycles with trabectedin was 5.5 (range 2-28). Three patients achieved partial response (8.8%) and eight patients showed stable disease (23.5%). The objective response rate and disease control rate were 8.8% (95% confidence interval (CI), 95% CI 1.9-23.7) and 32.4% (95% CI 17.4-50.5), respectively. Overall, progression-free survival was 2.9 months (95% CI 2.1-3.4). The overall survival was 7.3 months (95% CI 4.7-12.8). The most common trabectedin-related grade 3 adverse events were observed in 10 patients (26.5%), mostly being neutropenia (14.7%) and elevated transaminases (5.9%), whereas one patient (2.9%) reported grade 4 febrile neutropenia that required hospitalization.
The findings of this real-life study consistently support that trabectedin is an effective and safe option for the treatment of patients with non-L-sarcoma after failure of anthracyclines and ifosfamide, or in patients who are unsuited to receive these agents.
在临床试验和真实世界研究中,已经广泛证明了 trabectedin 治疗平滑肌肉瘤和脂肪肉瘤(通常称为 L 肉瘤)的有效性。然而,关于非 L 肉瘤的可用文献则较少。本研究的目的是评估 trabectedin 在真实环境中治疗非 L 肉瘤患者的疗效和安全性。
这是一项回顾性、观察性研究,纳入了 2013 年 10 月至 2020 年 7 月期间在西班牙巴塞罗那圣十字和圣保罗医院接受 trabectedin 治疗的 34 名患者。
最常见的组织学亚型是未分化梭形细胞/多形性肉瘤(n=11,32.4%)、滑膜肉瘤(n=6,17.7%)、黏液纤维肉瘤(n=5,14.7%)和恶性外周神经鞘瘤(n=4,11.8%)。trabectedin 的平均治疗周期数为 5.5(范围 2-28)。3 名患者达到部分缓解(8.8%),8 名患者疾病稳定(23.5%)。客观缓解率和疾病控制率分别为 8.8%(95%置信区间(CI):95%CI 1.9-23.7)和 32.4%(95%CI 17.4-50.5)。总体而言,无进展生存期为 2.9 个月(95%CI 2.1-3.4)。总生存期为 7.3 个月(95%CI 4.7-12.8)。最常见的 trabectedin 相关 3 级不良事件发生在 10 名患者(26.5%)中,主要是中性粒细胞减少症(14.7%)和转氨酶升高(5.9%),而 1 名患者(2.9%)报告了 4 级发热性中性粒细胞减少症,需要住院治疗。
这项真实世界研究的结果一致表明,trabectedin 是蒽环类药物和异环磷酰胺治疗失败或不适合使用这些药物的非 L 肉瘤患者的有效和安全选择。