Surrogacy of progression-free survival (PFS) for overall survival (OS) in esophageal cancer trials with preoperative therapy: Literature-based meta-analysis

Kataoka, Kozo; Nakamura, Kyoichi; Mizusawa, Junki; Kato, Ken; Eba, Junko; Katayama, Hokahiro; Shibata, Tomotaka; Fukuda, Haruhiko

doi:10.1016/j.ejso.2017.06.017

Cited by 14 publications

(21 citation statements)

References 37 publications

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“…Previous literature-based studies indicated that early efficacy endpoints of PFS or DFS were poorly associated with OS at the trial level. The treatment benefit of PFS or DFS was not likely to be converted to survival benefit in resectable esophageal or GEJ cancer patients receiving neoadjuvant therapy ( 4 , 5 ). In this study, the most recent trials were updated and only large-scale RCTs with ≥ 100 patients were included.…”

Section: Discussionmentioning

confidence: 99%

“…However, previous studies demonstrated a poor trial-level correlation between PFS/DFS and OS in resectable esophageal or GEJ cancer treated with neoadjuvant therapy, indicating PFS/ DFS as an unsuitable early efficacy endpoint (4,5). However, these studies may not be comprehensive because they did not exclude small-scale RCTs or perform quality control before statistical analysis.…”

Section: Introductionmentioning

confidence: 99%

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Progression-Free Survival as Early Efficacy Endpoint in Resectable Esophageal Cancer Treated With Neoadjuvant Therapy: A Systematic Review

et al. 2022

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To investigate literature-based evidence regarding progression-free survival (PFS) as an early efficacy endpoint in patients with resectable esophageal or gastroesophageal junction (GEJ) cancer receiving neoadjuvant therapy, this study identified large-scale randomized controlled trials (RCTs) with strict quality control. Twenty-four RCTs involving 7,514 patients were included. Trial-level correlation analysis was conducted to analyze the relationship between PFS hazard ratio (HR) and overall survival (OS) HR, Δ median PFS and Δ median OS. Correlation analysis at the neoadjuvant treatment arm level was performed between 1- to 5-year PFS and 5-year OS, median PFS and median OS. Subgroup analysis was performed in patients treated with standard neoadjuvant chemoradiotherapy (NCRT). The correlation was evaluated using the Pearson correlation coefficient r in weighted linear regression, with weight equal to patient size. In trial-level correlation, PFS were strongly associated with OS HR (r, 0.82 [95% confidence interval (CI), 0.42-0.97]) and Δ median survival (r, 0.83 [95% CI, 0.54-0.96]). In neoadjuvant treatment arms, there was a strong correlation between 1 to 5-year PFS rates and 5-year OS (r, 0.83-0.95), and median PFS and median OS (r, 0.97 [95% CI, 0.85-0.99]). NCRT subgroup analysis demonstrated acceptable consistency. In conclusion, we recommend PFS as an early efficacy endpoint in resected esophageal or GEJ cancer treated with neoadjuvant therapy.

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Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Progression-Free Survival as Early Efficacy Endpoint in Resectable Esophageal Cancer Treated With Neoadjuvant Therapy: A Systematic Review

et al. 2022

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“…OS was defined as the time interval between esophagectomy and the date of death from any cause or censor at final follow-up. PFS was defined as the time interval between esophagectomy and the date of radiological or histological detection of cancerous recurrence or metastasis (4,27). Another one secondary objective was to estimate the differences in postoperative complications, 30-day mortality and 90-day mortality between the two AAPR groups.…”

Section: Outcomes Of Interestmentioning

confidence: 99%

Albumin-To-Alkaline Phosphatase Ratio as a Novel and Promising Prognostic Biomarker in Patients Undergoing Esophagectomy for Carcinoma: A Propensity Score Matching Study

Zhu

Chen

et al. 2021

Front. Oncol.

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BackgroundAlbumin-to-alkaline phosphatase ratio (AAPR) has been reported as a novel prognostic predictor for numerous solid tumors. We aimed to assess the prognostic role of preoperative AAPR in surgically resectable esophageal squamous cell carcinoma (ESCC) by a propensity score matching (PSM) analysis with predictive nomograms.MethodsOur study was conducted in a single-center prospective database between June 2009 and December 2012. Kaplan-Meier analysis was used to distinguish the difference in survival outcomes between patients stratified by an AAPR threshold. Multivariable Cox proportional hazards regression model was finally generated to specify independent prognostic markers for the entire and PSM cohorts.ResultsA total of 497 patients with ESCC were included in this study. An AAPR of 0.50 was determined as the optimal cutoff point for prognostic outcome stratification. Patients with AAPR<0.50 had significantly worse overall survival (OS), and progression-free survival (PFS) compared to those with AAPR≥0.50 (Log-rank P<0.001). This significant difference remained stable in the PSM analysis. Multivariable analyses based on the entire and PSM cohorts consistently showed that AAPR<0.50 might be one of the most predominant prognostic factors resulting in unfavorable OS and PFS of ESCC patients undergoing esophagectomy (P<0.001). The nomograms consisting of AAPR and other independent prognostic factors further demonstrated a plausible predictive accuracy of postoperative OS and PFS.ConclusionAAPR can be considered as a simple, convenient and noninvasive biomarker with a significant prognostic effect in surgically resected ESCC.

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“…Moreover, recently, studies have demonstrated that a more immediate and more pronounced response is prognostic [ 16 , 17 , 18 , 19 ]. Progression-free survival (PFS) is considered a good surrogate endpoint for overall survival (OS) for some cancers [ 20 , 21 , 22 , 23 , 24 ], but not all situations [ 25 , 26 ]. The uncoupling of PFS and OS could be due to a number of factors, including treatment cross-over in clinical trials.…”

Section: “Response” As a Reflection Of Therapeutic Benefitmentioning

confidence: 99%

Monitoring for Response to Antineoplastic Drugs: The Potential of a Metabolomic Approach

Rattner

Bathe

2017

Metabolites

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For most cancers, chemotherapeutic options are rapidly expanding, providing the oncologist with substantial choices. Therefore, there is a growing need to select the best systemic therapy, for any individual, that effectively halts tumor progression with minimal toxicity. Having the capability to predict benefit and to anticipate toxicity would be ideal, but remains elusive at this time. An alternative approach is an adaptive approach that involves close observation for treatment response and emergence of resistance. Currently, response to systemic therapy is estimated using radiographic tests. Unfortunately, radiographic estimates of response are imperfect and radiographic signs of response can be delayed. This is particularly problematic for targeted agents, as tumor shrinkage is often not apparent with these drugs. As a result, patients are exposed to prolonged courses of toxic drugs that may ultimately be found to be ineffective. A biomarker-based adaptive strategy that involves the serial analysis of the metabolome is attractive. The metabolome changes rapidly with changes in physiology. Changes in the circulating metabolome associated with various antineoplastic agents have been described, but further work will be required to understand what changes signify clinical benefit. We present an investigative approach for the discovery and validation of metabolomic response biomarkers, which consists of serial analysis of the metabolome and linkage of changes in the metabolome to measurable therapeutic benefit. Potential pitfalls in the development of metabolomic biomarkers of response and loss of response are reviewed.

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Surrogacy of progression-free survival (PFS) for overall survival (OS) in esophageal cancer trials with preoperative therapy: Literature-based meta-analysis

Cited by 14 publications

References 37 publications

Progression-Free Survival as Early Efficacy Endpoint in Resectable Esophageal Cancer Treated With Neoadjuvant Therapy: A Systematic Review

Progression-Free Survival as Early Efficacy Endpoint in Resectable Esophageal Cancer Treated With Neoadjuvant Therapy: A Systematic Review

Albumin-To-Alkaline Phosphatase Ratio as a Novel and Promising Prognostic Biomarker in Patients Undergoing Esophagectomy for Carcinoma: A Propensity Score Matching Study

Monitoring for Response to Antineoplastic Drugs: The Potential of a Metabolomic Approach

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