To evaluate whether tegafur-uracil maintenance (UFTm) following postoperation adjuvant cisplatin-based concurrent chemoradiotherapy (CCRT) may reduce distant metastasis in patients with resected oral cavity squamous cell carcinoma (OSCC) with pathologic extranodal extension (pENE+).

A retrospective comparison was conducted between two cohorts of patients with resected pENE+ OSCC who completed adjuvant CCRT between March 2015 and December 2017, including one cohort of a phase II trial using UFTm and a trial-eligible but off-protocol cohort without using UFTm (non-UFTm) after their adjuvant CCRT. The UFTm trial enrolled patients without relapse within 2 months after the end of adjuvant CCRT and administered UFT 400 mg/day for 1 year. Kaplan–Meier methods estimated the actuarial rate of distant metastasis-free (DMF), locoregional control (LRC), event-free survival (EFS), and overall survival (OS).

A total of 103 patients were included in this study, 64 patients in UFTm and 39 patients in non-UFTm. Severe adverse events in UFTm included grade 3 anemia (n = 1, 1.6%) and grade 3 mucositis (n = 1, 1.6%). A total of 40 (62.5%) patients completed the full course of UFTm, while the remaining terminated UFTm earlier due to disease relapse (n = 14, 21.8%), poor compliance (n = 9, 14.1%), and adverse event (n = 1, 1.6%). The median (range) follow-up time of surviving patients was 43 (22–65) months. The outcomes compared between UFTm and non-UFTm were OS (hazard ratio [HR] 0.31 [95% CI: 0.17–0.57], p < 0·001), EFS (0.45 [0.25–0.82], 0.009), LRC (0.45 [0.19–1.05], 0.067), and DMF (0.47 [0.24–0.95], 0.035). Multivariable analysis, adjusted for UFTm, Charlson comorbidity index score 1–3, site of tongue, and number of ENE+ LN ≧4, confirmed better OS (0.29 [0.16–0.54], <0.001) and EFS (0.47 [0.26–0.85], 0.012) in favor of UFTm over non-UFTm. The 2-year DM rate was 25.8% in UFTm and 44.2% in non-UFTm. For relapsed patients in UFTm vs. non-UFTm, the rate of metastasectomy for oligometastasis was 53% vs. 6%, and the OS was 21.0 (95% CI: 17.8–24.1) months vs. 11.0 (9.1–12.8) months (p < 0.001), respectively.

UFTm may improve the dismal outcomes of the resected pENE+ OSCC. Further investigations are needed to confirm our observations.

旨在评估术后辅助顺铂同步放化疗 (CCRT) 后的替加氟尿嘧啶维持治疗 (UFTm) 是否可以减少伴有病理性结外扩展 (pENE+) 的口腔鳞状细胞癌 (OSCC) 患者的远处转移。

对 2015 年 3 月至 2017 年 12 月期间完成辅助 CCRT 的两组切除的 pENE+ OSCC 患者进行回顾性比较，其中一组使用 UFTm 进行 II 期试验，另一组符合试验资格但未使用 UFTm（非方案） -UFTm）在辅助 CCRT 后。 UFTm 试验招募了辅助 CCRT 结束后 2 个月内无复发的患者，并给予 UFT 400 mg/天，持续 1 年。 Kaplan-Meier 方法估计了无远处转移 (DMF)、局部区域控制 (LRC)、无事件生存 (EFS) 和总生存 (OS) 的精算率。

本研究共纳入 103 名患者，其中 64 名 UFTm 患者和 39 名非 UFTm 患者。 UFTm 中的严重不良事件包括 3 级贫血（n = 1，1.6%）和 3 级粘膜炎（n = 1，1.6%）。共有 40 名患者 (62.5%) 完成了 UFTm 的整个疗程，而其余患者由于疾病复发 (n = 14, 21.8%)、依从性差 (n = 9, 14.1%) 和不良事件 (n = 9, 14.1%) 提前终止了 UFTm。 n = 1, 1.6%）。存活患者的中位（范围）随访时间为 43（22-65）个月。 UFTm 和非 UFTm 之间比较的结果为 OS（风险比 [HR] 0.31 [95% CI：0.17–0.57]，p < 0·001）、EFS（0.45 [0.25–0.82]，0.009）、LRC（ 0.45 [0.19–1.05]、0.067) 和 DMF (0.47 [0.24–0.95]、0.035)。多变量分析，根据 UFTm、Charlson 合并症指数评分 1-3、舌头部位和 ENE+ LN ≥4 的数量进行调整，证实了更好的 OS (0.29 [0.16–0.54], <0.001) 和 EFS (0.47 [0.26– 0.85], 0.012) 相对于非 UFTm 有利于 UFTm。 UFTm 的 2 年 DM 率为 25.8%，非 UFTm 的 2 年 DM 率为 44.2%。对于 UFTm 与非 UFTm 的复发患者，寡转移的转移切除率为 53% vs. 6%，OS 分别为 21.0 (95% CI: 17.8–24.1) 个月 vs. 11.0 (9.1–12.8) 个月。 p < 0.001)，分别。

UFTm 可以改善切除 pENE+ OSCC 的惨淡结果。需要进一步调查来证实我们的观察。