Chaigneau Loïc, Jary Marine, Nerich Virginie, Hervieu Alice, Aubry Sébastien, Charon Barra Céline, Meynard Guillaume, Neumann Florent, Kalbacher Elsa, Isambert Nicolas
Department of Oncology, University Hospital of Besançon, Besançon, France.
INSERM, EFS BFC, UMR1098, RIGHT Interactions Greffon-Hôte-Tumeur/Ingénierie Cellulaire Et Génique, Besançon, France.
Oncology. 2022;100(12):633-644. doi: 10.1159/000527602. Epub 2022 Oct 25.
Soft tissue sarcomas (STSs) are a rare and heterogenous group of tumors, with poor prognostic, judging from their frequency to relapse. Few drugs are available after the conventional first-line regimen. Since 2007, trabectedin got approval after failure of anthracyclines and ifosfamide, for advanced or metastatic STS. This led to a FDA approval in 2015, but real-world evidence is still required, complementary to the pivotal phase II and III trials.
One hundred twenty-six patients with STS, treated by trabectedin between 2002 and 2019, were analyzed in this retrospective study, in two French centers. The effects of trabectedin on survival, response, and toxicity were described. All patients were tested for toxicities, and efficacy was assessed in patients exposed to at least 2 cycles of trabectedin.
Three median cycles were administered per patient (1-79). Among the 113 patients analyzed for efficacy, the median progression-free survival was 3.0 months (95% CI: 2.3-4.8), with an overall survival of 12.3 months (95% CI: 10.2-16.9). The rate of disease control was 46% at the end of treatment. Myxoid liposarcoma (n = 11) was the histology subtype that benefited most from this chemotherapy with median progression-free survival and overall survival of 13.3 months (95% CI: 2.3-18.7) and 27.8 months (95% CI: 3.2-64.7), respectively. Adverse events were manageable.
Efficacy of trabectedin is confirmed in terms of clinical benefit and low toxicity, especially for myxoid liposarcoma. Combinatory regimens are under clinical trials to optimize the place of this chemotherapy.
软组织肉瘤（STS）是一类罕见且异质性的肿瘤，从其复发频率来看预后较差。传统一线治疗方案后可用的药物很少。自2007年以来，曲贝替定在蒽环类药物和异环磷酰胺治疗失败后获批用于晚期或转移性STS。这导致其在2015年获得美国食品药品监督管理局（FDA）批准，但仍需要真实世界证据来补充关键的II期和III期试验。
在这项回顾性研究中，对2002年至2019年间在法国两个中心接受曲贝替定治疗的126例STS患者进行了分析。描述了曲贝替定对生存、反应和毒性的影响。对所有患者进行毒性检测，并在接受至少2个周期曲贝替定治疗的患者中评估疗效。
每位患者接受的中位周期数为3个（1 - 79个）。在分析疗效的113例患者中，中位无进展生存期为3.0个月（95%置信区间：2.3 - 4.8），总生存期为12.3个月（95%置信区间：10.2 - 16.9）。治疗结束时疾病控制率为46%。黏液样脂肪肉瘤（n = 11）是从这种化疗中获益最大的组织学亚型，中位无进展生存期和总生存期分别为13.3个月（95%置信区间：2.3 - 18.7）和27.8个月（95%置信区间：3.2 - 64.7）。不良事件可控。
曲贝替定在临床获益和低毒性方面的疗效得到证实，尤其是对黏液样脂肪肉瘤。联合治疗方案正在临床试验中，以优化这种化疗的地位。
