Meta-analysis to Investigate the Correlation Between Event-Free Survival and Overall Survival in Patients with Resectable Locally Advanced Head and Neck Squamous Cell Carcinoma.

IF 4 3区医学 Q2 MEDICINE, RESEARCH & EXPERIMENTAL

Advances in Therapy Pub Date : 2025-09-25 DOI:10.1007/s12325-025-03351-z

Dandan Zheng, Ali Mojebi, Yuexin Tang, Braden Hale, Sanjay Merchant, Sichen Liu, Sam Keeping

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引用次数: 0

Abstract

Introduction: While overall survival (OS) is a widely accepted trial endpoint in oncology, survival data are often immature in early-stage populations. Alternative time-to-event outcomes have been considered by regulatory and reimbursement agencies to allow for early patient access. Event-free survival (EFS) has shown strong correlations with OS in patients with locally advanced (LA) head and neck squamous cell carcinoma (HNSCC) and within the subgroup with unresectable tumors. With novel neoadjuvant and adjuvant immunotherapies being investigated in resectable LA-HNSCC, this study aimed to assess the trial-level correlation of EFS and OS in patients with resectable LA-HNSCC.

Methods: A systematic review (October 29, 2024) identified randomized controlled trials evaluating surgery with adjuvant therapy, neoadjuvant therapy, or both in patients with resectable LA-HNSCC. Trials reporting hazard ratios (HRs) or Kaplan-Meier curves for OS (time from randomization to death) and EFS (time from randomization to disease progression/recurrence or death) were eligible for the analysis. Base case included trials comparing neoadjuvant therapy + surgery + adjuvant therapy versus surgery + adjuvant therapy. Sensitivity analyses included models with trials comparing broader regimens (e.g., adjuvant therapy versus adjuvant therapy), excluding an outlier trial and restricting to publications after 2004. Correlations were measured between log(EFS HR) and log(OS HR) using regression models, with their strength measured using Pearson's correlation coefficient (R).

Results: The review included 45 trials, with 20 trials qualifying for correlation analysis. R (95% confidence interval) was 0.91 (0.36, 0.99) in the base case (n = 5 trials) and 0.41 (-0.01, 0.71; n = 20), 0.78 (0.52, 0.91; n = 19), and 0.76 (0.41, 0.92; n = 13) in the three sensitivity analyses, respectively.

Conclusion: Strong trial-level correlations were observed between EFS and OS, suggesting EFS is a valid surrogate for OS in patients with resectable LA-HNSCC, particularly in trials investigating neoadjuvant therapy + surgery + adjuvant therapy.

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研究可切除的局部晚期头颈部鳞状细胞癌患者无事件生存率与总生存率相关性的荟萃分析。

虽然总生存期（OS）是肿瘤学中被广泛接受的试验终点，但早期人群的生存数据往往不成熟。监管机构和报销机构已经考虑了可替代的事件发生时间结局，以允许患者早期获得治疗。在局部晚期（LA）头颈部鳞状细胞癌（HNSCC）患者和不可切除肿瘤亚组中，无事件生存期（EFS）与OS有很强的相关性。随着可切除的LA-HNSCC中新的辅助和辅助免疫疗法的研究，本研究旨在评估可切除的LA-HNSCC患者的EFS和OS的试验水平相关性。方法：一项系统综述（2024年10月29日）确定了随机对照试验，评估可切除的LA-HNSCC患者手术辅助治疗、新辅助治疗或两者兼用。报告OS（从随机化到死亡的时间）和EFS（从随机化到疾病进展/复发或死亡的时间）的风险比（hr）或Kaplan-Meier曲线的试验符合分析条件。基础病例包括比较新辅助治疗+手术+辅助治疗与手术+辅助治疗的试验。敏感性分析包括比较更广泛方案的试验模型（例如，辅助治疗与辅助治疗），排除异常试验并限制2004年以后的出版物。使用回归模型测量log（EFS HR）和log（OS HR）之间的相关性，使用Pearson相关系数(R)测量其强度。结果：纳入45项试验，其中20项符合相关分析。在基本情况（n = 5个试验）中，R（95%置信区间）为0.91(0.36,0.99)，在三个敏感性分析中分别为0.41 （-0.01,0.71;n = 20）、0.78 （0.52,0.91;n = 19）和0.76 （0.41,0.92;n = 13）。结论：试验水平上观察到EFS和OS之间存在很强的相关性，表明EFS是可切除的LA-HNSCC患者OS的有效替代指标，特别是在研究新辅助治疗+手术+辅助治疗的试验中。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Advances in Therapy 医学-药学

CiteScore

7.20

自引率

2.60%

发文量

353

审稿时长

6-12 weeks

期刊介绍： Advances in Therapy is an international, peer reviewed, rapid-publication (peer review in 2 weeks, published 3–4 weeks from acceptance) journal dedicated to the publication of high-quality clinical (all phases), observational, real-world, and health outcomes research around the discovery, development, and use of therapeutics and interventions (including devices) across all therapeutic areas. Studies relating to diagnostics and diagnosis, pharmacoeconomics, public health, epidemiology, quality of life, and patient care, management, and education are also encouraged. The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, communications and letters. The journal is read by a global audience and receives submissions from all over the world. Advances in Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of all scientifically and ethically sound research.