医脉通编译，转载请务必注明出处
2016年6月3-7日，一年一度的美国临床肿瘤学会（American Society of Clinical Oncology，ASCO）年会将在芝加哥举办。6月6日上午的消化系统（非结直肠）肿瘤口头报告专场上，一项摘要号为4002的随机II期试验，在转移性鳞状细胞食管癌（MSEC）患者中进行，医脉通整理如下：
尽管没有证据支持化疗（CT）在转移性鳞状细胞食管癌（MSEC）中的应用，很多临床医生为有良好ECOG体力状态（PS）的患者给予氟尿嘧啶（5-FU）/铂类为基础的化疗。因此，为了评估MSEC的总生存期（OS），研究人员在6周化疗后无进展（PD）的患者中设计了一项停药2期试验。
PS＜3的患者给予1线5-FU/铂类为基础的化疗，并在6周时接受肿瘤评估。6周的化疗后，无进展的患者被随机分配（1:1）接受CT继续（A组）或者CT停药联合BSC（B组）。在B组中，出现进展的患者被允许重新启动CT。主要终点是ITT 9个月时的OS率（从随机分组的日期计算）。计算样本数量用来在A组中提供ITT 9个月OS率±12.5的评估。B组作为内部对照，没有正式比较的意图。在第一次肿瘤评估时，出现58%的预期流失率，106例患者被需要以随机化62例患者。次要目标是耐受性，PFS，生活质量，和医疗费用。
105例患者被纳入，101例患者接受CT（FOLFOX：76/101；LV5FU2-CDDPq2w：18/101；TPF：4/101，FU-CDDPq3w：3/101）。67/101例患者在6周时没有进展被随机分配。基线患者特征如下：中位年龄：64岁；男性：54/67；PS≤1/2：61/67；转移性位置的数量：1/≥2：31/36；BMI≤18.5kg/m2：6/67；之前接受过化疗（联合放疗和/或手术）：37/67。64/67例患者符合要求和治疗（组A：31例，组B：33例）。化疗方案是LV5FU2-CDDPq2w 7/31，FOLFOX 24/31。ITT 9个月时的OS率分别为：组A，50%（85% CI：37-62）；组B，44%（85% CI，31-56）。随机分组后PFS分别为：组A，2.8个月（95% CI：1.7-5）；组B，1.4个月（95% CI：1.4-2.7）。耐受性良好，与我们应用化疗的预期一致。
综上所述，对于接受5-FU/铂类为基础化疗6周后无进展的转移性鳞状细胞食管癌患者来说，在随机分配接受化疗-停药的组A，9个月的OS率为50%。尽管OS和PFS趋势支持组A，但CT停药似乎没有提供更多的临床获益超过CT停药联合BSC。生活质量和医疗费用的结果目前在等待中。临床试验信息：NCT01248299。
会议专题》》》2016年ASCO年会专题报道
原文摘要：
Discontinuation of first-line chemotherapy (CT) after 6 weeks of CT in patients (pts) with metastatic squamous-cell esophageal cancer (MSEC): A randomized phase II trial.(Abstract 4002)
Authors：Antoine Adenis, Jaafar Bennouna,et al.
Session Type：Oral Abstract Session
Background: Even though there is no evidence to support the use of CT in MSEC, many physicians treat pts with good ECOG performance status (PS) with fluorouracil (5FU)/platinum-based CT. Therefore, in order to estimate overall survival (OS) in MSEC, we designed a discontinuation phase 2 trial in pts free from progression (PD) after 6 weeks (wks) of CT.
Methods: PS<3 MESC pts were treated with 1st-line 5FU/platinum-based CT, and underwent tumor assessment at 6 wks. Pts free from PD after 6 wks of CT were randomized (1:1) to receive CT continuation (arm A) or CT discontinuation plus BSC (arm B). In arm B, pts were allowed to restart CT in case of PD. Primary endpoint was ITT 9-month OS rate (calculated from randomization date). The sample size has been calculated to provide an estimate of ITT 9-month OS rate ± 12.5% in arm A. Arm B served as an internal control, without formal comparison intent.With an anticipated 58% dropout rate at 1st tumor assessment, 106 pts were needed to randomize 62 pts. Secondary objectives were tolerance, PFS, quality of life, and medical costs.
Results: 105 pts were included, and 101 received CT (FOLFOX: 76/101; LV5FU2-CDDPq2w: 18/101; TPF: 4/101, FU-CDDPq3w: 3/101). 67/101 pts free from PD at 6 wks were randomized. Baseline pts characteristics were as follows: median age: 64; male gender: 54/67; PS ≤1/2: 61/6; number of metastatic sites 1/≥2: 31/36; BMI<18.5kg/m²: 6/67; prior exposure to CT (combined to radiation therapy and/or surgery): 37/67. 64/67 pts were eligible and treated (arm A 31, arm B 33). CTs were LV5FU2-CDDPq2w 7/31, FOLFOX 24/31. ITT 9-months OS rate was 50% (85%-CI: 37-62) for arm A and 44% (85%-CI: 31-56) for arm B. PFS after randomization was 2.8 mo (95% CI: 1.7-5) for arm A, and 1.4 mo (95% CI: 1.4-2.7) for arm B. Tolerance was good, as expected with the CT we used.
Conclusions: In pts with MESC free from PD after 6 wks of 5FU/platinum-based CT who were randomized to the CT-continuation arm, 9-month OS rate in arm A was 50%. Despite a trend in OS and PFS favoring arm A, it does not appear that CT continuation provides much clinical benefit over CT discontinuation plus BSC, in such pts. Results of quality of life and medical costs are awaited. Clinical trial information: NCT01248299
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