Marks Jennifer A, Ahn Jaeil, Reuss Joshua E, Barbie David, Altan Mehmet, Gutierrez Martin E, Garassino Marina C, Riely Gregory J, Wakelee Heather, Liu Stephen V, Kim Chul
Dana-Farber Cancer Institute, Boston, MA.
Georgetown University, Washington, DC.
Clin Lung Cancer. 2025 May;26(3):165-167. doi: 10.1016/j.cllc.2024.12.001. Epub 2024 Dec 4.
Thymic epithelial tumors (TETs), including thymoma and thymic carcinoma, are rare thoracic tumors of the anterior mediastinum. For those with advanced disease, platinum-based chemotherapy is used as first-line treatment. However, there is no standard regimen established for TET at progression after initial therapy, and treatment options for advanced/recurrent TETs are limited. Trop-2, a transmembrane glycoprotein, is overexpressed in solid tumors including thymomas and thymic carcinomas. Sacituzumab govitecan-hziy, a Trop-2-directed antibody-drug conjugate, has shown efficacy and safety in several tumors including breast cancer. The overexpression of Trop-2 in TETs and the clinical efficacy in other malignancies provide rationale for exploring its use in thymoma and thymic carcinoma.
This open-label, single-arm, parallel cohort, multi-center study assesses the safety and efficacy of sacituzumab govitecan-hziy in patients with advanced thymoma (cohort A) and thymic carcinoma (cohort B) who have received at least 1 prior line of systemic therapy (NCT06248515). The study employs a Simon optimal 2-stage design, enrolling patients with adequate performance status, measurable disease, and adequate organ function. Sacituzumab govitecan-hziy is administered at a fixed dose of 10 mg/kg weekly on days 1 and 8 of 21-day cycles until disease progression or unacceptable toxicity. Follow-up continues every 6 months for 2 years postdiscontinuation. Archival tissue is obtained prior to initiation of study treatment with an optional biopsy at the time of progression. In cases where archival tissue is not available, a fresh biopsy is obtained at baseline. The primary endpoint is investigator-assessed response rate using Response Evaluation Criteria in Solid Tumors v1.1 (RECIST) criteria, with tumor imaging assessments every 2 cycles during the first 3 months and every 3 cycles thereafter. Secondary endpoints comprise adverse events by Common Terminology Criteria for Adverse Events v5.0, median and 6-month progression-free survival, duration of response, and overall survival. For each cohort, 9 patients will be enrolled. If 0 of the 9 achieve a response, no further patients will be enrolled in that cohort. If 1 or more of the first 9 patients has a response, accrual will continue until a total of 17 patients have been enrolled in that cohort.
胸腺上皮肿瘤(TETs),包括胸腺瘤和胸腺癌,是前纵隔罕见的胸部肿瘤。对于晚期疾病患者,铂类化疗被用作一线治疗。然而,对于初始治疗后病情进展的TETs,尚未建立标准治疗方案,晚期/复发性TETs的治疗选择有限。Trop-2是一种跨膜糖蛋白,在包括胸腺瘤和胸腺癌在内的实体瘤中过表达。戈沙妥珠单抗(Sacituzumab govitecan-hziy)是一种靶向Trop-2的抗体药物偶联物,已在包括乳腺癌在内的多种肿瘤中显示出疗效和安全性。Trop-2在TETs中的过表达以及在其他恶性肿瘤中的临床疗效为探索其在胸腺瘤和胸腺癌中的应用提供了理论依据。
这项开放标签、单臂、平行队列、多中心研究评估了戈沙妥珠单抗在晚期胸腺瘤(队列A)和胸腺癌(队列B)患者中的安全性和有效性,这些患者之前至少接受过1线全身治疗(NCT06248515)。该研究采用西蒙最优两阶段设计,纳入体能状态良好、疾病可测量且器官功能良好的患者。戈沙妥珠单抗以固定剂量10mg/kg,在21天周期的第1天和第8天每周给药,直至疾病进展或出现不可接受的毒性。停药后每6个月随访2年。在研究治疗开始前获取存档组织,并在疾病进展时进行选择性活检。在没有存档组织的情况下,在基线时获取新鲜活检组织。主要终点是根据实体瘤疗效评价标准第1.1版(RECIST)由研究者评估的缓解率,在前3个月每2个周期进行一次肿瘤影像学评估,此后每3个周期进行一次。次要终点包括根据不良事件通用术语标准第5.0版评估的不良事件、中位无进展生存期和6个月无进展生存期、缓解持续时间和总生存期。每个队列将纳入9名患者。如果9名患者中无1例达到缓解,则该队列不再纳入更多患者。如果前9名患者中有1例或更多例有缓解,则继续入组,直至该队列共纳入17名患者。