尚无研究明确证明化疗可延长晚期食管癌的总生存期 (OS)。我们对一线晚期不可切除/转移性食管癌/胃食管结合部癌进行了 III 期随机研究。年龄为 18-70 岁、体能状态为 0-2 的患者被随机分配至单独最佳支持治疗 (BSC) 组或 BSC 组每周服用 80 mg/m2 紫杉醇组。如前所述，平衡计分卡包括教育、咨询、放射、支架置入、饲管放置、营养补充、镇痛药等药物以及转介至支持小组和姑息治疗。主要终点是 OS；次要终点包括无进展生存期 (PFS)、反应、毒性和生活质量。 2016 年 5 月至 2020 年 12 月期间，我们招募了 281 名患者：143 名接受化疗，138 名接受 BSC。 269 名 (95.7%) 患者的组织病理学结果为鳞状。紫杉醇剂量的中位数为 12（IQR，7-23）。 BSC 的中位 OS 为 4.2 个月（95% CI，3.42-5.32），化疗为 9.2 个月（95% CI，8.02-10.48）； HR，0.49（95% CI，0.39-0.64）； p < .001。与 BSC 相比，化疗提高了缓解率（2.9% 至 39%）、中位 PFS（2.1 至 4.2 个月）、1 年 OS（11% 至 32%）、2 年 OS（0 至 9%）、中位吞咽困难-无生存期（2.9至14.8个月）以及整体和食管特异性的生活质量，并且不会显着增加所有级别或≥3级的毒性。使用 ESMO 临床效益量表和 ASCO 价值框架，姑息化疗被评为具有“实质性价值”。我们的研究提供了第一个一级证据，证明化疗可以延长晚期食管/胃食管结合部癌的生存期。单独的平衡计分卡（BSC）已经不再合适。每周一次紫杉醇是一种有吸引力的选择，特别是在免疫治疗机会有限的中低收入国家。© 2024 作者。约翰·威利出版的《国际癌症杂志》

No study has unequivocally proven that chemotherapy prolongs overall survival (OS) in advanced esophageal cancer. We conducted a Phase III randomized study in first-line advanced unresectable/metastatic esophageal/GEJ cancer. Patients aged 18-70 years, with performance status 0-2, were randomized to best supportive care (BSC) alone, or BSC with weekly paclitaxel 80 mg/m2. BSC comprised, as indicated, education, counselling, radiation, stenting, feeding tube placement, nutritional supplementation, medications like analgesics, and referral to a support group and palliative care. The primary endpoint was OS; secondary endpoints included progression free survival (PFS), response, toxicity, and QoL. Between May 2016-December 2020, we recruited 281 patients: 143 to chemotherapy and 138 to BSC. Histopathology was squamous in 269 (95.7%) patients. Median number of paclitaxel doses was 12 (IQR, 7-23). Median OS was 4.2 months (95% CI, 3.42-5.32) in BSC, and 9.2 months (95% CI, 8.02-10.48) in chemotherapy; HR, 0.49 (95% CI, 0.39-0.64); p < .001. As compared to BSC, chemotherapy increased response (2.9% to 39%), median PFS (2.1 to 4.2 months), 1-year OS (11% to 32%), 2-year OS (0 to 9%), median dysphagia-free survival (2.9 to 14.8 months), and global and esophagus-specific QoL, without significantly increasing all-grade or grade ≥3 toxicities. Using ESMO clinical benefit scale and ASCO Value Framework, palliative chemotherapy scored as having "substantial value." Our study provides the first level 1 evidence that chemotherapy prolongs survival in advanced esophageal/GEJ carcinoma. BSC alone is no longer appropriate. Weekly paclitaxel is an attractive option, especially in LMICs with limited access to immunotherapy.© 2024 The Author(s). International Journal of Cancer published by John Wiley & Sons Ltd on behalf of UICC.