Statistical aspects in adjuvant and neoadjuvant trials for gastrointestinal cancer in 2020: focus on time-to-event endpoints. Issue 4 (July 2020)

• Record Type: Journal Article
• Authors: Saad, Everardo D.; Buyse, Marc
• Is Part Of: Current opinion in oncology. Volume 32:Issue 4(2020:Jul.)
• Publication Date: 2020-07
• Subjects: colorectal neoplasms -- overall survival -- pancreatic neoplasms -- stomach neoplasms -- surrogate endpoints; Oncology -- Periodicals; 616.994
• DOI: 10.1097/CCO.0000000000000636
• Languages: English
• ISSNs: 1040-8746

Abstract

Abstract : Purpose of review: Clinical-trial design, analysis, and interpretation entails the use of efficient and reliable endpoints. Statistical issues related to endpoints warrant continued attention, as they may have a substantial impact on the conduct of clinical trials and on interpretation of their results. Recent findings: We review concepts and discuss recent developments related to the use of time-to-event endpoints in studies on adjuvant and neoadjuvant therapy for colon, pancreatic, and gastric adenocarcinomas. The definition of endpoints has varied to a considerable extent in these settings. Although these variations are relevant in interpreting results from individual trials, they probably have a small impact when considered in aggregate. In terms of surrogacy, most published reports so far have used aggregated data. A few studies based on the preferred method of a metaanalysis of individual-patient data have shown that disease-free survival (DFS) is a surrogate for overall survival in the adjuvant therapy of stage III colon cancer and in gastric cancer, whereas DFS with a landmark of six months is a surrogate for overall survival in the neoadjuvant therapy of adenocarcinoma of the esophagus, gastroesophageal junction, or stomach. Summary: Testing novel agents in gastrointestinal cancer requires continued attention to statistical issues related to endpoints.