摘要目的 探讨中低位直肠癌新辅助治疗后,评效为临床完全缓解(cCR)或近临床完全缓解(near-cCR)患者实施等待观察疗法或实施器官保留手术的预后.方法 收集自2011年3月至2017年8月期间,在北京大学肿瘤医院胃肠中心接受新辅助治疗后疗效评价为cCR或near-cCR、并实施等待观察疗法或器官保留手术(局部切除)的62例中低位直肠癌患者(cCR/near-cCR组)的临床资料进行回顾性病例分析.按照近似1∶2配对,选取同时间段在本中心进行新辅助治疗后,接受根治切除并获得病理完全缓解(ypCR)的123例患者(ypCR组)进行预后对比.研究主要终点为:3 年非肿瘤再生无病生存率(NR-DFS)和肿瘤特异性生存率(CSS);使用 Kaplan-Meier 曲线和Log-rank检验进行生存分析.研究次要终点为:3年器官保留率及括约肌保留率.结果 cCR/near-cCR组患者男性38例(61.3%)、女性24例(38.7%),中位年龄60(31~79)岁,基线状态肿瘤距肛缘中位距离4(1~8) cm.符合cCR和near-cCR诊断的比例分别为79.0%(49/62)和21.0%(13/62).全组患者的局部肿瘤再生率为24.2%(15/62).在15例肿瘤再生患者中,9例接受了补救性根治手术,术后无局部复发事件;4例接受了补救性局部切除,术后局部复发1例;另2例患者拒绝手术.总体转移率为8.1%(5/62),可根治性转移和不可根治性转移率分别为4.8%(3/62)和3.2%(2/62). 3年有效的器官和括约肌保留率分别为85.5%(53/62)和95.2%(59/62).中位随访36.2(8.6~89.0)月, cCR组和near-cCR组的3年NR-DFS分别为88.6%和83.1%,与ypCR组的3年NR-DFS(94.7%)差异无统计学意义(P＝0.217);cCR组和near-cCR组的3年CSS均为100%,与同期ypCR组的3年CSS(93.4%)差异也无统计学意义(P＝0.186).结论 新辅助治疗后评效为cCR或near-cCR的直肠癌患者,接受等待观察疗法或器官保留(局部切除)手术,远期预后良好,肿瘤局部再生和远隔转移率低,肿瘤学生存率与根治性手术后获得ypCR的患者相同.该治疗模式可能作为中低位直肠癌接受新辅助治疗后肿瘤退缩良好患者的一种有利于保留器官功能的治疗选择.

abstractsObjective To investigate the long-term outcome of organ preservation with local excision or "watch and wait" strategy for mid-low rectal cancer patients evaluated as clinical complete remission (cCR) or near-cCR following neoadjuvant chemoradiotherapy (NCRT). Methods Clinical data of 62 mid-low rectal cancer patients evaluated as cCR/near-cCR after NCRT undergoing organ preservation surgery with local excision or receiving "watch and wait" strategy at Department of Gastrointestinal Surgery, Peking University Cancer Hospital & Institute from March 2011 to August 2017 were retrospectively analyzed. According to the approximate 1∶2 pairing, 123 patients who underwent radical resection with complete pathological remission(ypCR) after neoadjuvant chemotherapy during the same period were selected for prognosis comparison. The primary endpoint of the study was 3-year non-regrowth disease-free survival (NR-DFS) and tumor specific survival (CSS). Survival analysis was performed using the Kaplan-Meier curve (Log-rank method). The secondary endpoint of the study was 3-year organ preservation and sphincter preservation. Results The retrospective study included 38 male and 24 female patients. The median age was 60 (31-79) years and the median distance from tumor to anal verge was 4 (1-8) cm. The ratio of cCR and near-cCR was 79.0%(49/62) and 21.0%(13/62) respectively. Local regrowth rate was 24.2%(15/62). Of 15 with tumor regrowth, 9 patients received salvage radical rectal resection and no local recurrence was found during follow-up; 4 patients received salvage local excision among whom one patient had a local recurrence occurred patient; 2 patients refused further surgery. The overall metastasis rate was 8.1% (5/62), including resectable metastasis (4.8%,3/62) and unresectable metastasis (3.2%,2/62). The valid 3-year organ preservation rate and sphincter preservation rate were 85.5% (53/62) and 95.2%(59/62) respectively. The median follow-up was 36.2 (8.6-89.0) months. The 3-year NR-DFS of patients with cCR and near-cCR was 88.6% and 83.1% respectively, which was not significantly different to that of patients with ypCR (94.7% , P＝0.217). The 3-year CSS of patients with cCR and near-cCR was both 100% , which was not significantly different to that of patients with ypCR (93.4% , P＝0.186). Conclusions Mid-low rectal cancer patients with cCR or near-cCR after NCRT undergoing organ preservation with local excision or receiving "watch and wait" strategy have good long-term prognosis with low rates of local tumor regrowth and distant metastasis, which is similar to those with ypCR after radical surgery. This treatment mode may be used as an option for organ preservation in mid-low rectal cancer patients with good tumor remission after NCRT.