Background: thymic basaloid carcinoma (BTC) is an extremely rare tumor, and very little data are available on BTC’s biology, clinical behavior, drug sensitivity, and patient outcomes. Methods: We performed a retrospective observational study on patients diagnosed with BTC in 11 referral centers of TYME. All BTC diagnoses were reviewed by the referring pathologist. Results: Twenty-eight patients were identified. A total of 22/28 patients were included. Eighteen patients had TNM stage I–III disease, and all underwent surgery; three patients received preoperative chemotherapy, and 10 patients received adjuvant radiotherapy. With a median follow-up of 46 (1–133) months, median overall survival (mOS) and median relapse-free survival were not reached. At 48 months, OS was 77% (95%CI 43–92), and DFS was 63% (95%CI 30–83). The median OS of the 4 patients diagnosed with metastatic disease was 7 months. Six patients received first-line systemic treatment for metastatic disease, and all showed tumor responses. Anti-tumor activity was also observed with an anti-VEGFR TKI and a multi-TKI inhibitor combined with an anti-PD1 antibody. Next-generation sequencing performed in three tumor samples did not identify actionable alterations or microsatellite instability. Conclusions: BTC is an extremely rare tumor that usually presents as a localized disease. Patients diagnosed with stage I–III disease can achieve long-term DFS, and efforts should be made to perform radical surgical resection combined with perioperative treatment whenever appropriate. Patients with advanced disease progression have a poor prognosis despite a high response rate to systemic treatments.
背景：胸腺基底样癌（BTC）是一种极为罕见的肿瘤，目前关于其生物学特性、临床表现、药物敏感性及患者预后的数据极为有限。方法：我们在TYME联盟的11家转诊中心对确诊为BTC的患者进行了一项回顾性观察研究。所有BTC诊断均由转诊病理学家复核确认。结果：共纳入28例患者中的22例进行分析。其中18例为TNM分期I–III期患者，均接受手术治疗；3例接受术前化疗，10例接受辅助放疗。中位随访时间为46个月（范围1-133个月），中位总生存期（mOS）和中位无复发生存期均未达到。48个月总生存率为77%（95%CI 43–92），无病生存率为63%（95%CI 30–83）。4例确诊为转移性疾病的患者中位总生存期为7个月。6例转移性疾病患者接受一线全身治疗均显示肿瘤应答。抗血管内皮生长因子受体酪氨酸激酶抑制剂及多靶点酪氨酸激酶抑制剂联合抗PD1抗体也观察到抗肿瘤活性。对三例肿瘤样本进行的新一代测序未发现可干预的基因变异或微卫星不稳定性。结论：BTC是极为罕见的肿瘤，通常表现为局限性病变。I–III期患者通过根治性手术切除联合围手术期治疗可获得长期无病生存，临床应积极争取在适宜条件下实施该治疗方案。尽管晚期疾病患者对全身治疗应答率较高，但疾病进展后预后仍然较差。
肿瘤(癌症)患者之家