International Consensus-Driven Recommendations for Patient-Reported Outcome Research Objectives in Early Phase Dose-Finding Oncology Trials: OPTIMISE-ROR

作者：Emily Alger, Olalekan Lee Aiyegbusi, A. C. Dueck, Anna Minchom, madeline pe, John Devin Peipert, C. J. Snyder, Stefan N. Symeonides, Roger Wilson, Ethan Basch, Yu Qiao, Susan E. Bates, Helen Bulbeck, Lizzie Dean, Massimo Di Maïo, Aaron R. Hansen, Olga Kholmanskikh, Ken Kobayashi, Dónal Landers, Christophe Le Tourneau, J. Jack Lee, Brigette Ma, Lynley V. Marshall, Sheetal Patel, Joan Petrie, Gregory R. Pond, Kieran Prior, Khadija Rantell, John F. Reeve, O. Solovyeva, Nolan A. Wages, Harald A. Weber, Melanie Calvert, C. Yap · 发表于：Journal of Clinical Oncology · 年份：2026 · DOI：10.1200/jco-25-01625 · 被引用次数：5 · 研究领域：Cancer survivorship and care、Health Systems, Economic Evaluations, Quality of Life、Ethics in Clinical Research

PURPOSE: There is growing scientific interest in incorporating patient-reported outcomes (PROs) in early phase dose-finding oncology trials (DFOTs) to assess tolerability, inform dose selection, and guide later stage trial design. However, research indicates that PRO objectives in DFOTs are often unclear. The Incorporating Patient-Reported Outcomes in Dose-Finding Trials-Research Objectives Recommendations (OPTIMISE-ROR) project was established to support trialists to effectively incorporate PROs into DFOTs. METHODS: Using the Enhancing Quality and Transparency of Health Research (EQUATOR) Network's methodological framework, guideline development included the following: (1) a methodological review of published DFOTs incorporating PROs; (2) candidate item generation, refined through expert consultation; (3) a two-round international multistakeholder Delphi survey (N = 109 in Round 1 [October 2024]; N = 96 in Round 2 [December 2024]); and (4) an independently chaired virtual consensus meeting (N = 31; January 2025) where multidisciplinary, international experts reviewed and voted to finalize items for inclusion. RESULTS: Consensus was reached on six recommendations emphasizing three core PRO tolerability concepts: overall side effect impact, symptomatic adverse events, and overall health-related quality of life. The integration of PROs to inform final dose recommendations in dose escalation and optimization trials should be considered, regardless of trial design. The recommenda...