Department of Thoracic Oncology, National Kyushu Cancer Center, Fukuoka.
Department of Biostatistics, Yokohama City University, Yokohama.
Ann Oncol. 2015 Feb;26(2):363-8. doi: 10.1093/annonc/mdu541. Epub 2014 Nov 17.
Thymic carcinoma (TC) is an exceptionally rare tumor, which has a very poor prognosis differing from thymoma. Till date, there has been no report of any results of clinical trials focusing on TC. The role of non-anthracycline-based chemotherapy has not been elucidated since the previous studies included a relatively small number of TC patients. This single-arm study evaluated carboplatin and paclitaxel (CbP) in chemotherapy-naive patients with advanced TC.
The study treatment consisted of carboplatin (area under the curve 6) and paclitaxel (200 mg/m(2)) every 3 weeks for a maximum of six cycles. The primary end point was objective response rate (ORR) by independent review. The secondary end points included overall survival (OS), progression-free survival (PFS), and safety. Based on the SWOG 2-stage design, the planned sample size of 40 patients was determined to reject the ORR of 20% under the expectation of 40% with a power of 0.85 and a type I error of 0.05.
Forty patients from 21 centers were enrolled for this study from May 2008 to November 2010. Of the 39 patients evaluable for analysis, 36 were pathologically diagnosed by independent review, and 97% patients were eventually TC. There was 1/13 complete/partial responses with an ORR of 36% (95% confidence interval 21%-53%; P = 0.031). The median PFS was 7.5 (6.2-12.3) months, while OS did not reach the median value. Major adverse event was grade 3-4 neutropenia in 34 patients (87%). There was no treatment-related death.
In this largest trial with TC, CbP showed promising efficacy in advanced TC when compared with anthracycline-based chemotherapy, which is the current standard treatment of thymic neoplasm. Our results established that CbP, one of the standard treatments for non-small-cell lung cancer, might be an option as a chemotherapy regimen for TC.
胸腺癌（TC）是一种非常罕见的肿瘤，其预后与胸腺瘤截然不同。迄今为止，尚无任何专门针对 TC 的临床试验结果报告。由于之前的研究纳入了相对较少的 TC 患者，因此非蒽环类药物化疗的作用尚未阐明。这项单臂研究评估了卡铂和紫杉醇（CbP）在未经化疗的晚期 TC 患者中的疗效。
研究治疗包括卡铂（曲线下面积 6）和紫杉醇（200mg/m²），每 3 周一次，最多 6 个周期。主要终点是独立评估的客观缓解率（ORR）。次要终点包括总生存期（OS）、无进展生存期（PFS）和安全性。根据 SWOG 2 期设计，计划纳入 40 例患者，以拒绝预期 40%的 ORR（20%），其功效为 0.85，I 型错误率为 0.05。
2008 年 5 月至 2010 年 11 月，来自 21 个中心的 40 例患者入组本研究。在可评估分析的 39 例患者中，36 例经独立评估确诊为病理，97%的患者最终被诊断为 TC。13 例患者中有 1 例达到完全/部分缓解，ORR 为 36%（95%置信区间 21%-53%；P=0.031）。中位 PFS 为 7.5 个月（6.2-12.3），OS 未达到中位值。主要不良事件是 34 例患者（87%）出现 3-4 级中性粒细胞减少。无治疗相关死亡。
在这项 TC 最大规模的试验中，与蒽环类药物化疗相比，CbP 在晚期 TC 中显示出有前景的疗效，蒽环类药物化疗是目前胸内肿瘤的标准治疗方法。我们的结果表明，CbP 作为非小细胞肺癌的标准治疗方法之一，可能是 TC 化疗方案的一种选择。
