Progression-free survival at 3 years is a reliable surrogate for 5-year overall survival for patients suffering from locally advanced esophageal squamous cell carcinoma.
Progression-free survival at 3 years is a reliable surrogate for 5-year overall survival for patients suffering from locally advanced esophageal squamous cell carcinoma.
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对于患有局部晚期食管鳞状细胞癌的患者，3 年无进展生存期是 5 年总生存期的可靠替代指标
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尽管在一些随机对照试验（RCT）中将3年生存期作为主要终点，但有限的证据支持使用中间终点来评价食管鳞状细胞癌（ESCC）新疗法的效果。本研究旨在系统评价ESCC患者的3年无进展生存期（3年PFS）和总生存期（OS）。我们确定了528例新诊断为局部晚期ESCC并接受明确放疗的患者。使用标准化死亡率比（SMR）将OS与年龄和性别匹配的一般中国人群进行比较。使用已发表的数据进行回归分析，以验证PFS和OS之间的相关性。3年后，年进展风险降至11.5%。未达到3年PFS的患者的中位进展后生存期（PPS）为7.3个月，5年OS率为9.6%，SMR为15.0（95%置信区间[CI]，12.9-17.5）。相反，达到3年PFS的患者的SMR为0.9（95% CI，0.6-1.3）。我们观察到试验水平的对数风险比（HR）（PFS）和对数HR（OS）之间存在显著相关性（r = 0.89; 95%CI，0.88-0.90）。在RCT和回顾性研究中，3年PFS和5年OS之间的相关性最强。3年内出现进展的患者生存率较差，而达到3年PFS的患者结局极佳。我们的研究支持3年PFS作为局部晚期ESCC研究设计和风险分层的可靠主要终点。1.对于局部晚期食管鳞状细胞癌患者，前3年的年进展和死亡风险降至20%以下。2.初始治疗后3年内无进展的患者经历了有利的长期结局，总生存时间与年龄和性别匹配的中国人群没有区别。相反，3年内发生疾病进展的患者预后非常差。3. 3年无进展生存期可用作未来研究中试验设计和风险分层的潜在终点。
Despite 3‐year survival being used as a primary endpoint in some randomized controlled trials (RCTs), limited evidence supports the use of intermediate endpoints to evaluate the effect of new therapies in esophageal squamous cell cancer (ESCC). This study aimed to systematically evaluate progression‐free survival at 3 years (3‐year PFS) and overall survival (OS) among patients with ESCC. We identified 528 patients newly diagnosed with locally advanced ESCC who received definitive radiotherapy. OS was compared with an age‐ and sex‐matched general Chinese population using the standardized mortality ratio (SMR). Regression analysis was used to validate the correlation between PFS and OS using published data. The annual risk of progression decreased to 11.5% after 3 years. Patients who did not achieve 3‐year PFS had a median postprogression survival (PPS) of 7.3 months, with a 5‐year OS rate of 9.6% and a SMR of 15.0 (95% confidence interval [CI], 12.9–17.5). Conversely, the SMR for patients who achieved 3‐year PFS was 0.9 (95% CI, 0.6–1.3). We observed a significant correlation between log hazard ratio (HR) (PFS) and log HR (OS) at the trial level (r = 0.89; 95% CI, 0.88–0.90). The strongest correlation was observed between 3‐year PFS and 5‐year OS in RCTs and retrospective studies. Patients exhibiting progression within 3 years experienced poor survival, whereas patients achieving 3‐year PFS had excellent outcomes. Our study supports 3‐year PFS as a reliable primary endpoint for study design and risk stratification in locally advanced ESCC. 1. The annual progression and death hazards decreased to less than 20% over the first 3 years for patients with locally advanced esophageal squamous cell carcinoma. 2. Patients who were progression‐free within 3 years after initial treatment experienced favorable long‐term outcomes, with overall survival times not distinguishable from that of age‐ and sex‐matched Chinese populations. Conversely, patients who experienced disease progression within 3 years had very poor prognosis. 3. 3‐year progression free survival can be used as a potential endpoint for trial design and risk stratification in future studies.
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