Department of Surgery, Leiden University Medical Center, Leiden, Netherlands; Department of Surgery, Netherlands Cancer institute, Amsterdam, Netherlands.
Department of Surgery, Leiden University Medical Center, Leiden, Netherlands; Department of Medical Oncology, Leiden University Medical Center, Leiden, Netherlands.
Lancet. 2018 Jun 23;391(10139):2537-2545. doi: 10.1016/S0140-6736(18)31078-X.
The strategy of watch and wait (W&W) in patients with rectal cancer who achieve a complete clinical response (cCR) after neoadjuvant therapy is new and offers an opportunity for patients to avoid major resection surgery. However, evidence is based on small-to-moderate sized series from specialist centres. The International Watch & Wait Database (IWWD) aims to describe the outcome of the W&W strategy in a large-scale registry of pooled individual patient data. We report the results of a descriptive analysis after inclusion of more than 1000 patients in the registry.
Participating centres entered data in the registry through an online, highly secured, and encrypted research data server. Data included baseline characteristics, neoadjuvant therapy, imaging protocols, incidence of local regrowth and distant metastasis, and survival status. All patients with rectal cancer in whom the standard of care (total mesorectal excision surgery) was omitted after neoadjuvant therapy were eligible to be included in the IWWD. For the present analysis, we only selected patients with no signs of residual tumour at reassessment (a cCR). We analysed the proportion of patients with local regrowth, proportion of patients with distant metastases, 5-year overall survival, and 5-year disease-specific survival.
Between April 14, 2015, and June 30, 2017, we identified 1009 patients who received neoadjuvant treatment and were managed by W&W in the database from 47 participating institutes (15 countries). We included 880 (87%) patients with a cCR. Median follow-up time was 3·3 years (95% CI 3·1-3·6). The 2-year cumulative incidence of local regrowth was 25·2% (95% CI 22·2-28·5%), 88% of all local regrowth was diagnosed in the first 2 years, and 97% of local regrowth was located in the bowel wall. Distant metastasis were diagnosed in 71 (8%) of 880 patients. 5-year overall survival was 85% (95% CI 80·9-87·7%), and 5-year disease-specific survival was 94% (91-96%).
This dataset has the largest series of patients with rectal cancer treated with a W&W approach, consisting of approximately 50% data from previous cohort series and 50% unpublished data. Local regrowth occurs mostly in the first 2 years and in the bowel wall, emphasising the importance of endoscopic surveillance to ensure the option of deferred curative surgery. Local unsalvageable disease after W&W was rare.
European Registration of Cancer Care financed by European Society of Surgical Oncology, Champalimaud Foundation Lisbon, Bas Mulder Award granted by the Alpe d'Huzes Foundation and Dutch Cancer Society, and European Research Council Advanced Grant.
在接受新辅助治疗后达到完全临床缓解（cCR）的直肠癌患者中采用观察等待（W&W）策略是一种新方法，为患者提供了避免接受重大切除术的机会。然而，该证据基于来自专科中心的中小规模系列研究。国际观察等待数据库（IWWD）旨在通过汇集个体患者数据的大型注册研究来描述 W&W 策略的结果。我们报告了在该注册中纳入 1000 多名患者后的描述性分析结果。
参与中心通过在线、高度安全和加密的研究数据服务器将数据输入到该注册中。数据包括基线特征、新辅助治疗、影像学方案、局部复发和远处转移的发生率以及生存状况。所有接受新辅助治疗后放弃标准治疗（直肠系膜全切除术）的直肠癌患者均有资格纳入 IWWD。对于本分析，我们仅选择在重新评估时无肿瘤残留迹象的患者（cCR）。我们分析了局部复发患者的比例、远处转移患者的比例、5 年总生存率和 5 年疾病特异性生存率。
2015 年 4 月 14 日至 2017 年 6 月 30 日，我们从 47 个参与机构（15 个国家）的数据库中确定了 1009 名接受新辅助治疗并采用 W&W 治疗的患者。我们纳入了 880 名（87%）达到 cCR 的患者。中位随访时间为 3.3 年（95%CI 3.1-3.6）。2 年局部复发累积发生率为 25.2%（95%CI 22.2-28.5%），88%的局部复发均发生在第 1-2 年内，97%的局部复发位于肠壁。880 名患者中有 71 名（8%）诊断为远处转移。5 年总生存率为 85%（95%CI 80.9-87.7%），5 年疾病特异性生存率为 94%（91-96%）。
该数据集是采用 W&W 方法治疗直肠癌患者的最大系列之一，约 50%的数据来自之前的队列系列研究，50%的数据来自未发表的数据。局部复发主要发生在第 1-2 年内和肠壁，强调了内镜监测的重要性，以确保选择延迟的根治性手术。W&W 后局部无法挽救的疾病很少见。
欧洲癌症护理注册由欧洲外科肿瘤学会资助，里斯本 Champalimaud 基金会、巴姆斯尔德尔奖由 Alpe d'Huzes 基金会和荷兰癌症协会授予，以及欧洲研究理事会高级拨款。
